Contact Us
Menu
Contact Us
Gray haired woman in blue shirt smiles into the camera.

Blood Clotting Therapies At
THE HEART OF HUMAN RESILIENCE.

Hemab is pioneering the first targeted preventative treatments and functional cures for underserved bleeding and thrombotic disorders—reimagining the standard of care for patients everywhere.

Our Approach

No Patient With A
Clotting Disorder

SHOULD BE
LEFT BEHIND.
While patients with common bleeding and thrombotic disorders have had access to preventative treatments for more than a half-century, no such measures exist for less-common disorders.
Young girl with glasses in a yellow sweater smiles into the camera.
For these tens of thousands of men, women, and children, life is paralyzingly unpredictable. A bleeding or thrombotic event can strike any moment, from a simple menstrual period to a routine dental procedure, and cause symptoms so severe that by the time they reach a hospital, it may be too late.
It’s time to ensure that every clotting disorder patient has the security and peace of mind they deserve.

The First-Ever Preventative Treatments For

RARE CLOTTING DISORDERS.
Hemab is leapfrogging technological advancements to deliver the first ever prophylactic treatments and functional cures for the full spectrum of genetically driven bleeding and thrombotic disorders to prevent life-threatening complications and offer patients a new lifeline. 
Essentially, the body’s bleeding and thrombosis pathway is fine-tuned, and there are numerous points within it that can fail, causing disease. Our novel therapies are designed to accumulate, modulate/balance, and at times recruit endogenous clotting factors already present in the blood to the site of injury to enable patients to form hemostatic plugs (or, clots) at the right times and in the right places.
Man with glasses in a gray sweater smiles into the camera.
Our therapeutic candidates* bind the body’s native clotting factor(s) with high specificity and affinity. This binding can stabilize the native clotting factor(s), preventing breakdown and allowing accumulation to therapeutic levels. Furthermore, the binding can be neutral (preserving the activity of the native clotting factor) or inhibitory (inactivating the clotting factor), giving us the versatility to address challenges across the clotting pathway. Our state-of-the-art technology also allows us, when necessary, to recruit native clotting factors directly to the site of injury.  
Man in green shirt hugs woman in gray sweater, both smiling into camera.

*Currently using monovalent and bispecific antibodies, with intention of expanding to other modalities

Our Pipeline

Preventing The Full Spectrum Of

BLEEDING & THROMBOTIC EVENTS.

Leveraging validated advanced technologies and key insights into the biology of clotting, Hemab’s approach is prime for widespread expansion into a range of underserved bleeding and thrombotic disorders, including Glanzmann Thrombasthenia, Factor VII Deficiency, Hemophilia B with inhibitors, Bernard Soulier Syndrome, Von Willebrand Disease, Hereditary Hemorrhagic Telangiectasia (or Osler-Weber-Rendu disease), Congenital Antithrombin III Deficiency, and others.

Man in a tan shirt smiles into the camera.

We are committed to finding and serving patients that have been left behind despite recent innovative therapies. Our strategic guidance, Hemab 1-2-5TM is targeting development of five clinical assets by 2025 to transform treatment for these rare disorders and enable patients everywhere to live healthy, active lives.

Hemab 1-2-5TM: 5 Clinical Assets by 2025
Program
Discovery
Preclinical
PI
PII
Program
Discovery
Preclinical
Phase I
Phase II
HMB-001 (Bleeding)
Lead candidate HMB-001 is a bispecific antibody that binds, stabilizes, and recruits endogenous factor VIIa (FVIIa) to the site of vascular injury to overcome the body’s inability to form healthy clots—building on the longstanding efficacy of recombinant FVIIa with the benefit of prophylaxis. Indicated for Glanzmann Thrombasthenia and Factor VII Deficiency.
HMB-002 (Bleeding)
HMB-002 is designed as a prophylactic treatment for Von Willebrand Disease Type 1 and 2
HMB-003
To be disclosed soon
HMB-004
To be disclosed soon
HMB-005
To be disclosed soon

If you have questions about our ongoing clinical trials, please contact clinicaltrials@hemab.com.

Our expanded access policy can be found here.
Our Team

Building The Ultimate
CLOTTING COMPANY.

Bringing together international experts in clotting biology, patient care, and drug development, the Hemab team is committed to changing the lives of patients with rare clotting disorders all around the world.

Young girl in a cheek-to-cheek hug with her mother, both smiling into the camera.
Benny Sorensen, MD, PhD
CEO
Mads Behrndt​, MSc
CFO & General Manager
Cécile Bonvoisin, MSc
Senior Vice President, CMC and Manufacturing
Tara O’Meara
Senior Vice President, Development Operations
John Maraganore​, PhD
Board Chair
Linda Bain​
Director
Dan Becker, MD, PhD
Director
Uya Chuluunbaatar, PhD
Director
Jørgen Søberg Petersen, MD, PhD, DMSc, MBA
Director
Benny Sorensen, MD, PhD
Director
Mårten Steen, MD, PhD
Director
Laura Tadvalkar​, PhD, MSc
Director
Akshay Vaishnaw, MD, PhD
Director
Christine Borowski, PhD
Observer
Camilla Petrycer Hansen, PhD, Cand. Pharm
Observer
Shan Wang, PhD
Observer
Jigar Amin, PharmD
Senior Director, Medical Affairs
Annika Anthonsen
Senior Laboratory Technician
Theresa Helene Bak-Thomsen, PhD
Vice President, Research
Erik Bjornson
Vice President, Regulatory Affairs
Amalie Carnbring Bonde, PhD
Research Scientist
Leyna Cho
Senior Clinical Trial Associate
Jennifer Dupee
Executive Assistant/Office Manager
Heidi Dyreborg
Senior Accountant
Samira Farboodmanesh, PhD
Senior Director, Regulatory Affairs
Michelle Fisher
Director, Data Management
Jacob Fredsted, MSc
Senior Scientist, Drug Substance Development
Prafull Gandhi, PhD, MS
Senior Scientific Director
Shea Golden
Senior Clinical Research Associate
Ashley Gosnell, MPAS, PA-C
Senior Manager, Clinical Research
Christina Grosse
Office/HR Manager and Executive Assistant to the Head of CMC & Manufacturing
Mattias Häger, PhD, MSc
Scientific Director
Rane Harrison, PhD
Director, Analytical Development
Klaus Krainer Jønsson, MSc
Finance Director
Louise Kempf-Amkær
Senior Laboratory Technician
Andrew Law
Senior Director, Medical Sciences
Ruthvik Malladi, PharmD
Director, Medical Affairs
Shivangi Mathy
Associate Director, Clinical Operations
Pruthvi S R Nagilla, MSc
Senior Director, Bioanalysis and DMPK
Rikke Lykke Nielsen
Senior Laboratory Technician
Helle Elisabeth Gluver Nørgaard
Senior Laboratory Technician
Henrik Østergaard, PhD, MSc
Scientific Vice President
Kristen Pappas, MBA
Vice President, Clinical Operations
Tara Parsons
Senior Clinical Trial Manager
Emil Poulsen, MSc
Director, Drug Substance Development, CMC
Caroline Rasmussen, DVM, PhD
Senior Director, Nonclinical Development
Catherine Rea, MD, PhD
Senior Director, Medical Sciences
Corrina Roman-Kreuze, BSc
Director, Quality
Anja R. H. Skands, MSc
Vice President, Drug Product and Device Development
Thanh-Hue Tran, MSc
Finance Manager
Mette Aaskov Ulbak, MCPHS
Director, Regulatory Affairs
Joe Vogel
Senior Director, Clinical and Program Leadership
Kathryn Zwetchkenbaum
Manager, Human Resources and Executive Assistant

Benny Sorensen, MD, PhD CEO

Benny Sorensen, MD, PhD, joined as CEO of Hemab Therapeutics from Codiak Biosciences where he served as Senior Vice President, Head of Clinical Development and Strategic Projects. During his tenure, he was involved with building the company and leading non-clinical, clinical, and regulatory affairs and advancing the first-ever engineered exosome therapeutics from idea into clinical proof of concept. Dr. Sorensen remains a member of the Scientific Advisory Board at Codiak. Before Codiak, he was Senior Director of Clinical Research at Alnylam Pharmaceuticals, where he led development of fitusiran and other hematology products. Previously, he was a Global Medical Director at Baxter Healthcare Corporation advancing pivotal development of Adynovate® and Vonvendi®. He has more than 15 years of experience in clinical and translational research and management. Prior to industry, he was the Director of the Haemostasis Research Unit and Honorary Lecturer at Guy’s and St. Thomas’ Hospital & King’s College London School of Medicine, where he led basic translational research and advancement of several clinical trials across Phases I, II, and III. Dr. Sorensen earned his MD and PhD in Medicine degrees from Aarhus University, Denmark, and has published extensively in hematology translational and clinical research.

Mads Behrndt​, MSc CFO & General Manager

Mads Behrndt, MSc, is CFO and General Manager of Hemab. He brings more than 14 years of experience in the international capital markets as well as within strategy and business development. He joined Hemab from PwC, where he was a Partner at PwC’s Corporate Finance practice. At PwC, he led and executed private and public company financings and M&A transactions. He earned an MSc in Finance & Strategic Management from the Copenhagen Business School, Denmark.

Cécile Bonvoisin, MSc Senior Vice President, CMC and Manufacturing

Cécile Bonvoisin, MSc, serves as Senior Vice President, CMC and Manufacturing of Hemab. She brings more than 20 years of strong operational experience in the pharmaceutical industry, especially in drug substance and drug product development for small molecules, biochemical and biologic products. She started her career at GlaxoSmithKline and joined from Sanofi, where she was the Global Head of Clinical Injectables Manufacturing within Biologics CMC. She also held positions in manufacturing and commercial organizations, leading international teams and strengthened her strategic thinking skills as the “attaché” of the Sanofi ExCom member in charge of Manufacturing and Quality, Supply Chain and EHS global functions. She earned a Master’s Degree in Engineering and an MSc in Process Engineering from the National Engineering School of Chemical Engineering in Toulouse, France.

Tara O’Meara Senior Vice President, Development Operations

Tara O’Meara serves as Senior Vice President, Development Operations at Hemab. She brings more than 25 years of biotech operations and clinical development experience, especially in rare disease, across small molecules, large molecules, and gene/cell therapy. Prior to Hemab, Tara worked at several growing biotechnology companies where she built the development operations teams and capabilities, including bluebird bio and Relay Therapeutics. Tara has been involved in multiple product approvals throughout her career, including Myozyme®, Skysona™, Zynteglo®, and Abecma®. Tara has a Bachelor of Science in Biology from Tufts University.

John Maraganore​, PhD Board Chair

John Maraganore, PhD, serves as the Chair of Hemab’s Board of Directors. In his nearly 20-year tenure as the founding CEO of Alnylam, he steered the company’s efforts to pioneer RNA interference (RNAi) therapeutics, raising over $7 billion to fund its programs and establishing partnerships with more than 25 of the leading pharmaceutical and biotechnology companies. As inventor of bivalirudin, a direct-acting thrombin inhibitor later commercialized as ANGIOMAX™, and formative contributor to Fitusiran, an RNAi therapeutic targeting antithrombin for the treatment of hemophilia A or B, he has deep knowledge of thrombosis and hemostasis research and development. He has a PhD in Biochemistry and Molecular Biology from the University of Chicago, United States.

Linda Bain​ Director

Linda Bain is Chief Operating Officer and Chief Financial Officer of Mariana Oncology. She most recently served as CFO of Codiak BioSciences. Prior to joining Codiak, she held senior roles at Adverum Biotechnologies (previously Avalanche Biotechnologies), bluebird bio, Genzyme Corporation, Fidelity Investments, and AstraZeneca. Her dedication to health equity will be key to advancing Hemab’s mission to aid underserved patients around the world. She earned a BS and an Honors degree in Accounting and Business Administration from the University of the Free State, South Africa.

Dan Becker, MD, PhD Director

Dan Becker, MD, PhD, is a Managing Director at Access Biotechnology. Prior to Access, Dan was a Principal at New Leaf Venture Partners, where he played a key role in multiple private and public biopharma investments, and a Principal in the Health Care practice at the Boston Consulting Group, where he led projects across the health care sector with an emphasis on biopharma R&D. Dan trained clinically in internal medicine and nephrology at Brigham and Women’s Hospital and Massachusetts General Hospital, and was a Research Fellow at Harvard Medical School. He obtained both his M.D. and Ph.D. (Cellular and Molecular Biology) degrees from the University of Michigan, and received his B.S. in Physiology from the University of Illinois at Urbana-Champaign.

Uya Chuluunbaatar, PhD Director

Uya Chuluunbaatar, PhD, is a Partner at Avoro Ventures. Prior to joining Avoro Ventures, Uya was a Principal at Amzak Health where she led private and public biotech investments. Prior to Amzak, Uya was a biotech equity research analyst at Goldman Sachs, covering large and mid-cap companies. Uya completed her Ph.D in Microbiology at New York University School of Medicine and holds a B.S. in Biochemistry from Cornell University, United States.

Jørgen Søberg Petersen, MD, PhD, DMSc, MBA Director

Jørgen Søberg Petersen, MD, PhD, DMSc, MBA, is Senior Partner at Novo Holdings and previously served as the Hemab Board Chair. Dr. Pedersen trained in medicine in Denmark and in the United States. During his 20 years in the pharmaceutical industry, he served in executive R&D roles at Lundbeck, Zealand Pharma, Merck, and Novo Nordisk. He co-founded Zealand Pharma and built the company to exit. He has extensive experience in rare disease development and financing of rare disease companies. He earned an MD, PhD in Pharmacology, and DMSc from the University of Copenhagen, Denmark, and an MBA from the Technical University of Denmark.

Benny Sorensen, MD, PhD Director

Benny Sorensen, MD, PhD, joined as CEO of Hemab Therapeutics from Codiak Biosciences where he served as Senior Vice President, Head of Clinical Development and Strategic Projects. During his tenure, he was involved with building the company and leading non-clinical, clinical, and regulatory affairs and advancing the first-ever engineered exosome therapeutics from idea into clinical proof of concept. Dr. Sorensen remains a member of the Scientific Advisory Board at Codiak. Before Codiak, he was Senior Director of Clinical Research at Alnylam Pharmaceuticals, where he led development of fitusiran and other hematology products. Previously, he was a Global Medical Director at Baxter Healthcare Corporation advancing pivotal development of Adynovate® and Vonvendi®. He has more than 15 years of experience in clinical and translational research and management. Prior to industry, he was the Director of the Haemostasis Research Unit and Honorary Lecturer at Guy’s and St. Thomas’ Hospital & King’s College London School of Medicine, where he led basic translational research and advancement of several clinical trials across Phases I, II, and III. Dr. Sorensen earned his MD and PhD in Medicine degrees from Aarhus University, Denmark, and has published extensively in hematology translational and clinical research.

Mårten Steen, MD, PhD Director

Mårten Steen, MD, PhD, is a partner of HealthCap. Previously, he worked as a Director of business development at Merck Serono, focusing on both product and technology licensing. Dr. Steen has pursued research in the field of protein chemistry and coagulation disorders at Lund University and Novo Nordisk. He is the author of numerous scientific papers published in peer-reviewed journals. He earned an MD and PhD in Clinical Chemistry from Lund University, Sweden.

Laura Tadvalkar​, PhD, MSc Director

Laura Tadvalkar, PhD, MSc, is a Managing Director at RA Capital Management. Her primary responsibility at RA Capital is to identify compelling assets to help facilitate new company creation or investments in emerging early-stage companies. Prior to RA, she was a Principal at MP Healthcare Venture Management. Prior to MP, she was a Consultant at Clarion Healthcare. She has a PhD in Chemical Biology from Harvard University, United States.

Akshay Vaishnaw, MD, PhD

Dr. Vaishnaw is the Chief Innovation Officer at Alnylam Pharmaceuticals. Since joining the company in 2006, he has served in positions of increasing responsibility across Research and Development (R&D), and most recently as President of Alnylam. Prior to Alnylam, Dr. Vaishnaw was Senior Director, Translational Medicine at Biogen. Dr. Vaishnaw earned his bachelor’s degree from University College Cardiff, his MD from the University of Wales College of Medicine, and his PhD in Molecular Immunology from the University of London.

Christine Borowski, PhD Observer

Christine Borowski, PhD, is a Vice President at Access Biotechnology. Before joining Access, she worked on therapeutics company creation at Apple Tree Partners. Prior to becoming an investor, Christine was an editor at several high-impact scientific journals, most recently Chief Editor of Nature Medicine. She earned a Ph.D. in Immunology at Harvard University and did her postdoctoral work in immunology at the University of Chicago.

Camilla Petrycer Hansen, PhD, Cand. Pharm Observer

Camilla Petrycer Hansen, PhD, Cand. Pharm, is a Principal at Novo Holdings and a Hemab Board Observer. Previously, she served as Senior Manager, Business Development at Novozymes and Business Development Manager at Statens Serum Institut. She earned a PhD in Medicinal Chemistry and a Cand. Pharm. in Pharmaceutical Sciences from the University of Copenhagen, Denmark.

Shan Wang, PhD Observer

Shan Wang is an Associate Director at RA Capital Management and a Hemab Board Observer. She earned a PhD in Neuroscience from Harvard Medical School, United States.

Jigar Amin, PharmD Senior Director, Medical Affairs

Jigar Amin, Pharm.D., currently serves as Director, Medical Affairs and Global External Engagement at Hemab. He has worked in the bleeding disorder space for more than a decade including roles at Novo Nordisk and Alnylam and his experience includes hemophilia, acute hepatic porphyria, primary hyperoxaluria. Jigar holds a Doctorate of Pharmacy from the University of Houston – College of Pharmacy.

Annika Anthonsen Senior Laboratory Technician

Annika Anthonsen is a Senior Laboratory Technician at Hemab. Annikas has a wealth of experience from 25 years within bleeding disorders spanning from gene therapy to expression and purification of proteins and antibodies to biochemical and plasma-based assays. Annika has held various lab technician roles at biotech and big pharma: Novozymes, AGC Biologics, Maxygen and latest Novo Nordisk.

Theresa Helene Bak-Thomsen, PhD Vice President, Research

Theresa Bak-Thomsen serves as Vice President, Research at Hemab, bringing 15 years of expertise in early drug discovery. In her current role, Theresa leads Hemab’s research initiatives with the research team, driving innovation and the new pipeline initiatives. With a career that includes leadership and project management roles at Novo Nordisk, Theresa has demonstrated a keen ability to direct research teams and complex projects in the early research phase. Theresa brings technological experience from leading antibody and proteins technology departments in Denmark and US with a variety of antibody platforms for antibody discovery and screening, research projects with diverse MoA profiles and within different therapeutic areas. Theresa holds a Ph.D. in Immunology from the University of Southern Denmark.

Erik Bjornson Vice President, Regulatory Affairs

Erik Bjornson serves as Vice President, Regulatory Affairs at Hemab. He brings more than 25 years of experience in the pharmaceutical industry with a focus on the development of products for rare bleeding disorders.  He started his career at Baxter Healthcare and has been involved with the development and licensure of numerous products, including ADVATE, ADYNOVATE, VONVENDI, RIXUBIS and OBIZUR. Erik’s experience includes roles at Baxter Healthcare, Baxalta, Shire, Takeda and most recently Galderma, where he was the Head of Biologics Regulatory Strategy.  He earned his Bachelor of Science in Chemistry from Texas Christian University, Fort Worth.

Amalie Carnbring Bonde, PhD Research Scientist

Amalie Carnbring Bonde, PhD, serves as a Research Scientist at Hemab. Prior to joining the company, she worked as an industrial PhD student at Novo Nordisk. She holds a PhD in Molecular Biology from Aarhaus University Denmark.

Leyna Cho Senior Clinical Trial Associate

Leyna Cho is a Senior Clinical Trial Associate at Hemab. She brings 6 years of clinical operations experience and has held a variety of positions on the sponsor side and CRO side. Leyna has worked on clinical studies in ophthalmology, rare disease, and gene therapy. Leyna earned her Bachelor of Science degree in Microbiology from University of Massachusetts Amherst.

Jennifer Dupee Executive Assistant/Office Manager

Jen comes to Hemab with 20+ years of diverse experience in operations and administration in biotech and venture capital, including space planning, buildouts/facilities, human resources, benefits administration, event planning, and employee wellness. Prior to joining Hemab, she served as Sr. Manager of Operations and Administration at Visterra, Inc. and as Office Manager at PureTech Ventures (now PureTech Health). Jennifer holds a Bachelor of Arts from Brown University where she studied English and American Literature and received her honors in Creative Writing.

Heidi Dyreborg Senior Accountant

Heidi Dyreborg is a Senior Accountant at Hemab. Heidi has more than 20 years of experience in accounting and financial assistance, with positions held at KemPharm. Inc., Orphazyme, and ORIGIO. She received her degree from Ballerup Handelsskole in Ballerup, Denmark.

Samira Farboodmanesh, PhD Senior Director, Regulatory Affairs

Samira Farboodmanesh

Samira Farboodmanesh, PhD, serves as Senior Director, Regulatory Affairs at Hemab, with over 18 years of expertise in product development and regulatory affairs. She began her career in R&D at the medical device startup Intrinsic Therapeutics and later joined EMD Millipore. Her path then led to leadership positions in both R&D and regulatory affairs across the pharmaceutical industry, including positions at AstraZeneca, Shire, Takeda, and Lyra Therapeutics. Her expertise spans various therapeutics areas –i.e., rare disease, respiratory, infectious, and endocrine disorders. Samira holds a PhD in Mechanical Engineering from the University of Massachusetts Lowell.

Michelle Fisher Director, Data Management

Michelle Fisher serves as Director, Data Management at Hemab. She brings over 15+ years of experience in Clinical Data Management within the biotechnology industry. Michelle has worked across several therapeutic areas, including Oncology, Auto-Immune Disorders, and Hematology. She has also worked in various stages of drug development from Phase I-IV study start-up, study conduct, through post-approval. Michelle earned her Bachelor of Science degree from Penn State University.

Jacob Fredsted, MSc Senior Scientist, Drug Substance Development

Jacob Fredsted, MSc, serves as Senior Scientist, Drug Substance Development, CMC at Hemab and has 10 years of experience in the biotechnology industry across early- and late-stage process development as well as commercial manufacturing. With experience in monoclonal -and bispecific antibodies, antibody-drug conjugates, and recombinant proteins, he has been involved in Process Characterization, Performance Qualification, submission- and approval by EMA and FDA. Prior to joining Hemab, he worked at Novo Nordisk, AGC Biologics, and most recently as CMC Specialist at Genmab. He earned a Master of Science in Biochemistry from the University of Copenhagen, Denmark.

Prafull Gandhi, PhD, MS Senior Scientific Director

Prafull S. Gandhi, PhD, MS, serves as Scientific Director at Hemab. He has 10+ years of experience within pre-clinical innovation, research, and project management. He specializes in protein structure, function, and engineering for drug discovery. Most recently, he worked as a Principal Scientist at Novo Nordisk, where he managed pre-clinical projects, characterized protein-protein interactions, and gained insight into the field of bleeding disorders. He has authored more than 15 articles in scientific journals and is an inventor of several patents. He holds an MS in Chemical Engineering from Missouri University of Science and Technology, United States and a PhD in Biochemistry and Molecular Biophysics from Washington University School of Medicine in St. Louis, United States.

Shea Golden Senior Clinical Research Associate

Shea Golden is a Senior Clinical Research Associate at Hemab. She brings experience from her work on multiple clinical trials for amyotrophic lateral sclerosis (ALS)  and as a clinical research coordinator at Massachusetts General Hospital in Boston. Shea received her Bachelor of Arts degree from Middlebury College in Vermont.

Ashley Gosnell, MPAS, PA-C Senior Manager, Clinical Research

Ashley Gosnell, MPAS, PA-C, serves as Senior Manager, Clinical Research, where she brings over a decade of unique healthcare and biotech experiences, ranging from clinical research and trial management to surgical procedures and diagnostics, to the Hemab team. She received a MSc in Physician Assistant Studies from South College in Knoxville, TN.

Christina Grosse Office/HR Manager and Executive Assistant to the Head of CMC & Manufacturing

Christina Grosse acts as Office/HR Manager, Europe,  and Executive Assistant to the Head of CMC & Manufacturing. She brings 20+ years of experience, including HR administration and Executive Assistant support to C-suite executives from international companies. Prior to joining Hemab, she served for the past 15 years as Strategic Project Lead and Senior Global Executive Assistant at the IT company SAP. Christina received her degree from the Higher Commercial Examination Programme (hhx) at Aarhus Business College and has an associate degree in banking from the Danske Bank, Copenhagen.

Mattias Häger, PhD, MSc Scientific Director

Mattias Häger, PhD, MSc, serves as a Scientific Director at Hemab. He has more than a decade of experience within early drug discovery with a focus on translational in vivo pharmacology and experience in animal model generation, gene expression and editing, and assay development. Most recently, Mattias served as a Principal Scientist and Project Manager at LEO Pharma, with previous experience at Zealand Pharma and Novo Nordisk, where he worked in the Hemophilia Pharmacology department. He is the author of more than 10 scientific publications and received his MSc from the University of Gothenburg and PhD from Lund University.

Rane Harrison, PhD Director, Analytical Development

Rane Harrison, PhD, serves as Director, Analytical Development at Hemab. He has over 15 years of experience and has worked on a diverse array of therapeutics ranging from small molecules to extracellular vesicles. Most recently, Rane was Director and Head of Analytical Development at Codiak BioSciences, where he helped develop and mature a novel platform for exosome-based therapeutics. He is an expert in liquid chromatography and mass spectrometry and has authored various scientific publications leveraging these techniques in different ways, and he is an inventor on several patents/patent applications. Rane earned a PhD in Chemistry from Northeastern University in Boston, Massachusetts, where he has since taught graduate students as an adjunct faculty member.

Klaus Krainer Jønsson, MSc Finance Director

Klaus Krainer Jønsson, MSc, is Finance Director of Hemab. He has more than 20 years of experience in the finance sector, and he has held finance leadership positions at Y-mAbs Therapeutics, Novo Nordisk, Roche and Nycomed, among others. He received an MSc in Business Economics and Auditing from Copenhagen Business School, Denmark.

Louise Kempf-Amkær Senior Laboratory Technician

Louise Kempf-Amkær is a senior Laboratory Technician at Hemab. She has vast laboratory experience in DNA techniques, protein purification and classification, and expression systems of E. Coli and mammalian cells through her tenure at Novo Nordisk as a Senior Lab Technician. Prior to joining Novo Nordisk, she served as a Sergeant in the Royal Danish Navy.

Andrew Law Senior Director, Medical Sciences

Andrew Law serves as Senior Director, Medical Sciences at Hemab. Andrew has over a decade of experience across clinical development, business development and medical affairs. His focus has been mainly within hematology therapeutic areas including haemostasis and thrombosis. He has held leadership responsibilities across small to large pharma. Andrew studied at the University of Bristol, is a Qualified General Practitioner and Member of the Faculty of Pharmaceutical Medicine in the UK.

Ruthvik Malladi, PharmD Director, Medical Affairs

Ruthvik Malladi, PharmD, serves as Director, Medical Affairs at Hemab. He brings over seven years of biotech experience with a strong focus on rare diseases. Most recently, Ruthvik worked at Blueprint Medicines in field roles in both Commercial and Medical Affairs. Prior to that, Ruthvik worked at Alnylam Pharmaceuticals in various capacities, from scientific communications to diagnostics. Ruthvik supported the launch of five medicines and aims to advance more treatments for patients. Ruthvik holds a Doctor of Pharmacy from Northeastern University.

Shivangi Mathy Associate Director, Clinical Operations

Shivangi Mathy is an Associate Director, Clinical Operations at Hemab. She has more than 14 years of experience in the biotechnology industry. She started her career as a bench scientist working in cell and molecular biology research. Over the course of her career, she transitioned into clinical operations and has managed clinical studies in rare disease, oncology and auto-immune indications.

Shivangi earned a Master’s degree in Pharmaceutical Sciences from Northeastern University, Boston, MA and a Bachelor of Science in Pharmacy from Poona College of Pharmacy, Pune, India.

Pruthvi S R Nagilla, MSc Senior Director, Bioanalysis and DMPK

Pruthvi Nagilla serves as Senior Director, Bioanalysis and DMPK at Hemab. He has over 15 years of progressive experience as a scientist and as an operational leader in biotech/ biopharma (Asher Bio, Passage Bio, AskBio), bioanalytical CRO (BioAgilytix), and in academic research (Pitt Infectious Diseases). Pruthvi is a cell and molecular biologist by training, with recent experience leading non-clinical and global clinical phase 1/2 bioanalysis for developing immunotherapies (oncology) and gene therapies (rare diseases in neurological, metabolic and cardiovascular therapeutic areas). He earned Master of Science degree in Biotechnology from Georgetown University in Washington, DC.

Rikke Lykke Nielsen Senior Laboratory Technician

Rikke Lykke Nielsen brings more than 25 years of experience from hospitals and small and big pharma, such as BioImage, Genzyme Biosurgery, Agilent Technologies. Most recently, she held various positions at Novo Nordisk in both CMC and in R&D. Rikke has solid experience in bioanalysis and robust set-up of bioassays and validation.

Helle Elisabeth Gluver Nørgaard Senior Laboratory Technician

Helle Elisabeth Gluver Nørgaard is a Senior Laboratory Technician at Hemab. She brings more than 20 years of laboratory experience from a number of companies and an expertise in analytical chemistry. Prior to Hemab, she worked at NeuroSearch as a Lab Technician and at Novo Nordisk as a Laboratory Technician in Protein Interaction and then in Development (ADME). She was also a Product Specialist at RAMCON.

Henrik Østergaard, PhD, MSc Scientific Vice President

Henrik Østergaard, PhD, MSc, serves as Scientific Vice President at Hemab. He has 18 years of experience within early biopharmaceutical drug discovery and development, most recently in the role as Scientific Director at Novo Nordisk A/S. With a particular focus on early innovation, protein engineering, and enzymology, Henrik has led several drug development projects within the hemophilia space, of which Refixia® is currently on the market. He is the author of numerous scientific publications and an inventor of patents covering new technologies and therapeutic molecules. He earned an MSc and a PhD from the Technical University of Denmark.

Kristen Pappas, MBA Vice President, Clinical Operations

Kristen Pappas serves as Vice President, Clinical Operations at Hemab. She brings over 20 years of clinical operations experience in the pharmaceutical/biotechnologyindustry. Kristen began her career as a CRA at PRA Int’l and continued working in clinical operations at Wyeth/Genetics Institute, Acceleron Pharma, Biogen, and bluebird bio where she served as Senior Director in the Clinical Trial Strategy and Execution Department. Her work has spanned a broad range of therapeutic areas including diabetes, bone repair, hemophilia, thalassemia, oncology and adrenoleukodystrophy (ALD). She has participated in multiple EMA/FDA filings and inspections and was a key contributor to the approval of SkysonaTM for boys with ALD. Kristen holds a bachelor’s degree in biology from Union College and earned her MBA from Babson College.

Tara Parsons Senior Clinical Trial Manager

Tara Parsons is a Senior Clinical Trial Manager at Hemab. Over her 15-year career in the biotech industry, Tara has held a variety of positions in both Clinical Operations and Clinical Compliance. Tara has worked across several therapeutic areas for drugs in various stages of development, including early phase through post-approval. Tara received her Bachelor of Arts degree from Assumption University in Worcester, MA.

Emil Poulsen, MSc Director, Drug Substance Development, CMC

Emil Poulsen, MSc, serves as Director, Drug Substance Development, CMC at Hemab and has more than 20 years of experience in the biotechnology industry across early- and late-stage process development. With experience in monoclonal antibodies, bispecific antibodies, antibody-drug conjugates and fusion proteins, he has successfully seen programs through Process Performance Qualification, submission and approval by EMA and FDA. Prior to joining Hemab, he worked at Biogen, Zymenex, CMC Biologics, Novozymes, Alpharma and was most recently an Associate Director of CMC Late-Stage Development Manufacturing at Genmab. He earned a Master of Science in Chemical Engineering and Biotechnology from the Technical University of Denmark.

Caroline Rasmussen, DVM, PhD Senior Director, Nonclinical Development

Caroline Rasmussen, DVM, PhD, is Senior Director, Nonclinical Development at Hemab. She brings over a decade of experience within the area of nonclinical development – ranging from early drug development to marketing authorization. She started her career at Novo Nordisk A/S working mainly within the hemostasis portfolio. Most recently, Caroline was employed at Ascendis Pharma A/S as Director of Toxicology and Safety Pharmacology, from where she brings a strong focus on unmet medical needs, science, and animal welfare. She earned her DVM and PhD at the Faculty of Life Sciences, Copenhagen University. As her doctorial research focused on the cardiovascular field, she has numerous publications within this area.

Catherine Rea, MD, PhD Senior Director, Medical Sciences

Catherine Rea, MD, PhD, serves as Senior Director, Medical Sciences at Hemab, bringing nearly two decades of clinical experience in hematology. She is specialized in the diagnosis and management of coagulation disorders. Allied with her clinical practice, she has expertise in translational research and study design. She has collaborated and led on projects covering all stages of novel therapy development, from pre-clinical drug evaluation through to phase 3 clinical trials. Dr. Rea studied at Cambridge University, England and was awarded a PhD in Medical Sciences from Kings College London, England. She has numerous publications covering basic science, translational research, and clinical hematology.

Corrina Roman-Kreuze, BSc Director, Quality

Corrina Roman Kreuze serves as Director, Quality at Hemab. She has 30 years of experience in the pharmaceutical, biotech and gene therapy industry. She started her career as a Medical Technologist in Hematology. Over the course of her career, she transitioned from hospital laboratory into industry, and has worked on enzyme replacement therapeutics (Replagal®) at Transkaryotic Therapies , viral cancer therapeutics at BioVex, small molecule therapeutics including Cubicin®, Cubicin RF®,at Cubist, and the Gardasil®9 vaccine at Merck. She most recently served as Head of Quality at Freeline Therapeutics where she worked on gene therapy treatments for Hemophilia A, Fabry’s Disease and Gaucher’s Disease. Corrina earned a BSc in Medical Laboratory Science from the University of New Hampshire.

Anja R. H. Skands, MSc Vice President, Drug Product and Device Development

Anja R. H. Skands, MSc, serves as Vice President, Drug Product and Device Development, CMC at Hemab. She has more than 20 years of experience in the biotechnology industry across early- and late-stage process development with a focus on protein and peptide development. Prior to joining Hemab, she worked at Biofac, Novo Nordisk and was most recently Vice President of Pharmaceutical Development at Ascendis Pharma.


Anja has successfully led programs and people through early development, Process Performance Qualification, submission and regulatory approval by EMA, FDA and PMDA. She earned a Master of Science in Chemical Engineering from the Technical University of Denmark.

Thanh-Hue Tran, MSc Finance Manager

Thanh-Hue Tran, M.Sc., is Finance Manager of Hemab. She brings more than 15 years of experience within the finance sector and life science companies including her role as Senior Auditor at Deloitte and Danske Bank; Group Financial Controller at Origio; Finance Manager at Orphazyme and most recently as Senior Finance Manager at BioInnovation Institute. Thanh-Hue holds a Master of Science in Business Administration and Auditing from Copenhagen Business School, Denmark. 

Mette Aaskov Ulbak, MCPHS Director, Regulatory Affairs

Mette Aaskov Ulbak serves as Director, Regulatory Affairs at Hemab. She possess a wealth of experience developing products for rare diseases with multiple therapeutic modalities including gene therapy. Her work spans early phase to submission of marketing authorization applications leading to approval. She has worked at both small and large organizations, including Lundbeck, Ovid Therapeutics, Prevail Therapeutics, Ascendis Pharma and most recently at Adrenomed AG, where she was the Head of Regulatory Affairs. Mette holds a Master in Pharmaceutical Sciences from the University of Copenhagen, Denmark.

Joe Vogel Senior Director, Clinical and Program Leadership

Joe Vogel serves as Senior Director, Clinical and Program Leadership at Hemab, and is the Program Team Lead for HMB-001. In his 15 years in the biotech industry, he has held a variety of roles spanning clinical operations and program management. Joe’s focus has been in rare disease including lysosomal storage disorders, hemophilia, TTR amyloidosis and hereditary angioedema. He worked across a broad range of treatment modalities from small molecule, ERT, siRNA, and CRISPR, across all phases of development. Joe is a lifelong DE&I practitioner and volunteers on the Board of OUTbio, the biotech industry’s largest LGBTQ+ professionals group. Joe holds a Bachelor degree in Psychology from Syracuse University and a Master of Business Administration from University of Massachusetts Amherst.

Kathryn Zwetchkenbaum Manager, Human Resources and Executive Assistant

Kathryn Zwetchkenbaum serves as Hemab’s Manager, Human Resources and Executive Assistant to the CEO. In her role she also oversees management of the Hemab Cambridge office. Prior to joining Hemab, Kathryn was the Executive Assistant for several C-suite executives at Relay Therapeutics including its CEO. While at Relay she founded the Women’s Employee Resource Group. Kathryn received a Bachelor of Music from the Royal Conservatoire of Scotland, Glasgow, where she studied Classical and Jazz Vocal Performance.

In the News

Learn About The Latest
HEMAB NEWS & RESEARCH.

February 9, 2024
Hemab Therapeutics Presents Positive Phase 1 Results for HMB-001 in Glanzmann Thrombasthenia at 2024 EAHAD Annual Congress
Read More
News: Press Release
Hemab Therapeutics Presents Positive Phase 1 Results for HMB-001 in Glanzmann Thrombasthenia at 2024 EAHAD Annual Congress

HMB-001 was well tolerated with no drug-related adverse or thromboembolic events; dose-dependent improvements in blood-clotting activity support the potential of HMB-001 as a first-in-class prophylactic treatment for Glanzmann Thrombasthenia and other blood clotting disorders

Preclinical HMB-001 findings were published in the February 2024 issue of Nature Cardiovascular Research

COPENHAGEN, DENMARK AND CAMBRIDGE, MASS., US – February 9, 2024 – Hemab Therapeutics, a clinical-stage biotechnology company developing novel prophylactic therapeutics for serious, underserved bleeding and thrombotic disorders, today presented initial results from Phase 1 of its ongoing evaluation of HMB-001. A novel bispecific antibody, HMB-001 is in development as a prophylactic treatment for the bleeding disorder Glanzmann Thrombasthenia (Glanzmann). The data were presented as one of 10 abstracts selected for the SLAM oral presentation session at the 17th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD) held this week in Frankfurt, Germany.

“The first clinical data for HMB-001 in Glanzmann suggest that the demonstrated mechanism is suitable as a new prophylactic treatment for people with neglected blood clotting disorders who face severe, potentially life-threatening bleeds every day,” said Joe Vogel, Senior Director and Program Lead for HMB-001 at Hemab. “With Phase 2 already underway, we are committed to advancing clinical evaluation of HMB-001 toward bringing life-changing preventative treatments to improve patients’ quality of life.”

The Phase 1/2 first-in-human open-label study in patients with Glanzmann is evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of HMB-001. As part of the UK-based Phase 1 single-ascending dose, seven patients received subcutaneous HMB-001 at 0.2 mg/kg, (n=1), 0.5 mg/kg (n=3) or 1.25 mg/kg (n=3). Over a 56-day observation period, HMB-001 was well tolerated; the most common adverse events (AEs) were mild or moderate in severity. No AEs or serious adverse events were deemed related to HMB-001. There were no dose-limiting toxicities and no thromboembolic events reported.

Treatment with HMB-001 resulted in a dose-dependent pharmacodynamic effect of endogenous Factor VIIa accumulation that was associated with decreases in prothrombin time and improvement in exploratory thrombin generation analyses. The pharmacokinetic profile and half-life of HMB-001 support dosing every two weeks or a less-frequent dosing regimen.

“People living with bleeding disorders like Glanzmann face devastating bleed events and considerable psychosocial impacts—from severe pain and social stigmatization to depression and isolation—but have no approved preventative treatments to turn to,” said Suthesh Sivapalaratnam, MD, PhD, Queen Mary University of London, Barts Health NHS Trust. “These Phase 1 data support further investigation into the promise of HMB-001 for patients and providers across the globe who are awaiting innovation to improve the standard of care.”

Hemab has begun dosing and is actively enrolling in Phase 2, the multiple-ascending dose of the study, which will be expanded to sites across Europe and the U.S. For more information, visit clinicaltrials.gov (NCT06211634).

In addition, Hemab announced that preclinical research findings of HMB-001 are now available online as part of the February 2024 issue of Nature Cardiovascular Research. The study results demonstrate the ability of HMB-001 to accumulate and potentiate the activity of endogenous Factor VIIa selectively on activated platelets, providing a sustained and localized procoagulant activity that may support prophylactic treatment of Glanzmann or other bleeding disorders. Read the paper here.

About Glanzmann Thrombasthenia

Glanzmann Thrombasthenia (Glanzmann) is a rare and severe bleeding disorder associated with debilitating and sometimes life-threatening bleeding episodes. Initial findings from the international Glanzmann’s 360 (GT360) natural history study—Hemab’s research initiative in partnership with UK specialist research consultancy Haemnet—found that 87% of the 104 respondents reported experiencing at least one bleed in the previous week, with 37% of those bleeds requiring medical treatment.

These bleeding episodes significantly impact the mental health and quality of life of people living with Glanzmann. Low mood, emotional problems, and social isolation were reported by participants (66%, 50%, and 44% respectively), and 80% reported that they missed school or work due to bruising or bleeding. To date, there are no effective prophylactic treatment options for Glanzmann.

About HMB-001

HMB-001 is bispecific antibody that binds and stabilizes endogenous Factor VIIa with one antibody arm and binds to TLT-1 on activated platelets with the other arm. This allows for accumulation of endogenous Factor VIIa in the body, recruitment of Factor VIIa directly to the surface of the activated platelets where it is known to facilitate clotting, and avoidance of clotting activity in the absence of tissue damage. HMB-001 is designed to be a first-in-class prophylactic treatment for Glanzmann Thrombasthenia with the potential to treat other debilitating rare bleeding disorders. The U.S. Food and Drug Administration granted Fast Track Designation to HMB-001 for the treatment of Glanzmann.

About Hemab Therapeutics

Hemab is a clinical-stage biotech company developing novel prophylactic therapeutics for serious, underserved bleeding and thrombotic disorders. Based in Cambridge, MA and Copenhagen, Denmark, Hemab is progressing a pipeline of monoclonal and bispecific antibody-based therapeutics to transform the treatment paradigm for patients with high unmet need. The company’s strategic guidance, Hemab 1-2-5TM, targets the development of 5 assets by 2025 to deliver long-awaited innovation for patients with high unmet need blood-clotting disorders like Glanzmann Thrombasthenia, Factor VII Deficiency, Von Willebrand Disease, and others. Learn more at hemab.com. Follow us on LinkedIn and X (formerly known as Twitter).

# # #

Media Contact:
Lia Dangelico
lia.dangelico@vergescientific.com
540-303-0180

January 4, 2024
Hemab Therapeutics Appoints Dr. Akshay Vaishnaw to Board of Directors and Announces Presentation at the 42nd Annual J.P. Morgan Healthcare Conference
Read More
News: Press Release
Hemab Therapeutics Appoints Dr. Akshay Vaishnaw to Board of Directors and Announces Presentation at the 42nd Annual J.P. Morgan Healthcare Conference

COPENHAGEN, DENMARK AND CAMBRIDGE, MASS., US – January 4, 2024 – Hemab Therapeutics, a clinical-stage biotechnology company developing novel prophylactic therapeutics for serious, underserved bleeding and thrombotic disorders, today announced the appointment of Akshay Vaishnaw, MD, PhD, to its Board of Directors.

The company also announced CEO Benny Sorensen, MD, PhD, will present an update on Hemab’s progress and outlook for 2024 at the 42nd Annual J.P. Morgan Healthcare Conference on Wednesday, January 10, at 4:30 PM PST/7:30 PM EST.

“In the last year, Hemab has achieved several important milestones, including raising a substantial Series B round, initiated its first Phase 2 clinical study and continued to build its pipeline of development candidates,” said John Maraganore, PhD, Board Chair. “With the appointment of Akshay to the Board, we add a biotech veteran who brings deep R&D experience at a time when Hemab will be advancing multiple clinical programs across a range of blood-clotting disorders. We welcome Akshay and the expertise he brings to Hemab and its mission.”

Dr. Vaishnaw is the Chief Innovation Officer at Alnylam Pharmaceuticals. Since joining the company in 2006 he has served in positions of increasing responsibility across Research and Development (R&D), and most recently as President of Alnylam. Prior to Alnylam, Dr. Vaishnaw was Senior Director, Translational Medicine at Biogen. Dr. Vaishnaw received a bachelor’s degree from University College Cardiff, UK, he received his MD from the University of Wales College of Medicine, UK, and a PhD from the University of London, UK, in Molecular Immunology. He is a Fellow of the Royal College of Physicians, UK. Dr. Vaishnaw is a member of the Board of Directors for Editas Medicine Inc. and Scholar Rock Inc.

“Hemab’s product development approach allows for a spectrum of modalities to be utilized, which holds immense promise for transforming the treatment of multiple blood-clotting disorders like Glanzmann Thrombasthenia, Factor VII Deficiency, and Von Willebrand Disease that remain urgently in need of novel prophylactic treatments,” said Dr. Vaishnaw. “I am excited to join Hemab’s Board of Directors and work with the leadership team who are clearly driven by patient need in their mission to deliver life-changing treatments for many people.”

“Akshay joins our Board at a time when his experience leading translational research, clinical development and delivering regulatory success will be invaluable,” said Dr. Sorensen. “I am thrilled to have his partnership and to bring his passion for science and commitment to building people-focused cultures to Hemab.”  

About Hemab Therapeutics

Hemab is a clinical-stage biotech company developing novel prophylactic therapeutics for serious, underserved bleeding and thrombotic disorders. Based in Cambridge, MA and Copenhagen, Denmark, Hemab is progressing a pipeline of monoclonal and bispecific antibody-based therapeutics to transform the treatment paradigm for patients with high unmet need. The company’s strategic guidance, Hemab 1-2-5TM, targets the development of 5 independent assets by 2025 to deliver long-awaited innovation for patients with high unmet need blood-clotting disorders like Glanzmann Thrombasthenia, Factor VII Deficiency, Von Willebrand Disease and other serious disorders. Learn more at hemab.com.

# # #

Media Contact:
Lia Dangelico
lia.dangelico@vergescientific.com
540-303-0180

December 11, 2023
Hemab Therapeutics Announces First Patient Dosed in Phase 2 Clinical Study Investigating HMB-001 for the Treatment of Glanzmann Thrombasthenia
Read More
News: Press Release
Hemab Therapeutics Announces First Patient Dosed in Phase 2 Clinical Study Investigating HMB-001 for the Treatment of Glanzmann Thrombasthenia

HMB-001 is a novel bispecific antibody designed to be the first prophylactic treatment for Glanzmann Thrombasthenia (GT) and other debilitating bleeding disorders

Phase 1 was successfully completed in the UK; Hemab plans additional sites in Europe and the U.S. for Phase 2

The U.S. FDA has cleared the Investigational New Drug (IND) application and granted Fast Track Designation to HMB-001 for the treatment of GT

COPENHAGEN, DENMARK AND CAMBRIDGE, MASS., US – December 11, 2023 – Hemab Therapeutics, a clinical-stage biotechnology company developing novel prophylactic therapeutics for serious, underserved bleeding and thrombotic disorders, announced today that it has completed Phase 1, the single ascending dose part, and transitioned to Phase 2, the multiple ascending dose part, of its Phase 1/2 clinical study of HMB-001 in Glanzmann Thrombasthenia (GT), a platelet disorder that causes severe, potentially life-threatening bleeding episodes.

The company also announced that the U.S. Food and Drug Administration (FDA) has cleared Hemab’s investigational new drug application (IND) for HMB-001 in GT, enabling enrollment in the U.S. Phase 1 of the clinical study was completed in the UK, and Phase 2 will include additional sites in Europe as well as the U.S.

In addition, the FDA granted Fast Track designation to HMB-001, emphasizing the seriousness and high unmet need for treatments for GT. The Fast Track program enables Hemab to have more frequent interactions with the FDA to facilitate the development of HMB-001.

“People living with Glanzmann Thrombasthenia can experience frequent, sometimes severe bleeds that can be life-threatening and compromise their quality of life. Currently, there are no prophylactic treatment options that would reduce or prevent these bleeding episodes,” said Benny Sorensen, MD, PhD, CEO of Hemab. “The completion of Phase 1 on time and transitioning to Phase 2 is an important milestone for HMB-001 and Hemab. Furthermore, expanding our clinical study into the U.S., following clearance of the IND, and Fast Track designation are a testament to the importance of advancing prophylactic treatment for people living with Glanzmann Thrombasthenia.”  

The Phase 1/2 clinical study evaluates the safety, tolerability, pharmacodynamics, and pharmacokinetics of HMB-001. Initial efficacy signals based on an assessment of changes in bleeding frequency will also be measured. The study is composed of three parts: Part A, single ascending dose, Part B, multiple ascending dose, and Part C, extended dosing. Hemab plans to report data from the Phase 1, single ascending dose portion, at an international scientific conference in early 2024.

The Phase 1/2 study design was detailed in the company’s poster presentation (number 1225), “A Phase 1/2, First-in-Human, Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of HMB-001 in Participants with Glanzmann Thrombasthenia,” at the 65th American Society of Hematology Annual Meeting and Exposition.

About Glanzmann Thrombasthenia

Glanzmann Thrombasthenia (GT) is a rare and severe bleeding disorder associated with debilitating and sometimes life-threatening bleeding episodes. Initial findings from the international Glanzmann’s 360 (GT360) natural history study, Hemab’s research initiative in partnership with UK specialist research consultancy Haemnet, found that 87% of the 104 respondents reported experiencing at least one bleed in the previous week, and 37% of those bleeds required medical treatment.

These bleeding episodes have a significant impact on the mental health and quality of life of people living with GT. Low mood, emotional problems, and social isolation were reported by participants (66%, 50%, and 44% respectively) and 80% reported that they missed school or work due to bruising or bleeding. To date, there are no effective prophylactic treatment options for people living with GT.

About HMB-001

HMB-001 is bispecific antibody that binds and stabilizes endogenous factor VIIa (FVIIa) with one antibody arm and binds to TLT-1 on activated platelets with the other arm. This allows for accumulation of endogenous FVIIa in the body, recruitment of FVIIa directly to the surface of the activated platelets where it is known to facilitate clotting, and avoidance of clotting activity in the absence of tissue damage. HMB-001 is designed to be a first-in-class prophylactic treatment for Glanzmann Thrombasthenia with the potential to treat other debilitating rare bleeding disorders.

About Hemab Therapeutics

Hemab is a clinical-stage biotech company developing novel prophylactic therapeutics for serious, underserved bleeding and thrombotic disorders. Based in Cambridge, MA and Copenhagen, Denmark, Hemab is progressing a pipeline of monoclonal and bispecific antibody-based therapeutics to transform the treatment paradigm for patients with high unmet need. The company’s strategic guidance, Hemab 1-2-5TM, targets the development of 5 independent assets by 2025 to deliver long-awaited innovation for patients with high unmet need blood-clotting disorders like Glanzmann Thrombasthenia, Factor VII Deficiency, Bernard Soulier Syndrome, Von Willebrand Disease, and other serious disorders. Learn more at hemab.com.

Media Contact

Lia Dangelico
lia.dangelico@vergescientific.com
540-303-0180

June 24, 2023
Hemab Therapeutics Presents New Preclinical Research Demonstrating Effects of Its Bispecific Antibody HMB-001 in Factor VII Deficiency
Read More
News: Press Release
Hemab Therapeutics Presents New Preclinical Research Demonstrating Effects of Its Bispecific Antibody HMB-001 in Factor VII Deficiency

HMB-001 recognizes and binds to factor VII (FVII) protein variants associated with moderate/severe FVII deficiency and drives accumulation of endogenous FVII/FVIIa to normal ranges in in-vivo models

COPENHAGEN, DENMARK AND BOSTON, MASS., US – June 24, 2023 – Hemab Therapeutics, a clinical-stage biotechnology company developing the first prophylactic therapeutics for serious, underserved bleeding and thrombotic disorders, announced results today from preclinical research of HMB-001 in models of factor VII (FVII) deficiency at the International Society on Thrombosis and Haemostasis (ISTH) 2023 Congress in Montreal.

“We believe that HMB-001 has the potential to transform treatment in several serious bleeding disorders and have already initiated a Phase 1/2 study in Glanzmann Thrombasthenia,” said Benny Sorensen, MD, PhD, CEO and President of Hemab. “The new preclinical data presented today show HMB-001 successfully targeted and accumulated endogenous FVIIa to levels that would be expected to provide clinical benefit in FVII deficiency, supporting the potential for HMB-001 in an additional underserved bleeding disorder.”

FVII is a protein necessary in the formation of hemostatic plugs to control bleeding. FVII deficiency can cause spontaneous or excessive and prolonged bleeding after injury or surgery; heavy or prolonged menstrual bleeding in women; and in very severe cases, life-threatening bleeding inside the skull or digestive tract.

HMB-001, Hemab’s lead candidate, is a bispecific antibody that binds to and stabilizes endogenous activated FVII (FVIIa) with one antibody arm and localizes FVIIa to the surface of activated platelets by binding to TLT-1 with the other arm. This allows for accumulation of FVIIa in the body and recruitment of FVIIa directly to the surface of activated platelets at the site of vascular injury where FVIIa is known to facilitate the formation of protective hemostatic plugs to stop bleeding.

The preclinical research presented at ISTH, “HMB-001, a Bispecific anti-FVIIa/anti-TLT-1 Antibody Demonstrates Effect in Models of FVII Deficiency,”assessed key requirements for HMB-001 to function in FVII deficiency, specifically its ability to bind with FVII protein variants associated with moderate/severe deficiency and the potential of HMB-001 to accumulate FVIIa in an in-vivo non-human primate model of FVII deficiency.

A panel of 12 FVII variants from the European Association for Haemophilia and Allied Disorders (EAHAD) database was produced based on high prevalence and association with moderate/severe FVII deficiency phenotype as well as proximity to the HMB-001 binding site. In subsequent binding studies, HMB-001 was shown to bind to all FVII variants at clinically relevant concentrations.

The ability of HMB-001 to accumulate endogenous FVIIa in FVII deficiency was assessed using small interfering RNA (siRNA) to knock down FVII/FVIIa to levels between 10 to 30 percent of normal in animal models (n=3). After continuous, stable knock down, HMB-001 (5 mg/kg) was administered. The total accumulation of FVIIa observed with HMB-001 was comparable to the normal range seen in healthy animals. These initial results suggest HMB-001 may have potential application as a treatment for FVII deficiency.

About HMB-001

HMB-001 is bispecific antibody that binds and stabilizes endogenous factor VIIa (FVIIa) with one antibody arm and TLT-1 on activated platelets with the other arm. This allows for accumulation of FVIIa in the body, recruitment of FVIIa directly to the surface of the activated platelets where it is known to facilitate clotting, and avoidance of clotting activity in the absence of tissue damage. HMB-001 was designed to be a first-in-class prophylactic treatment for Glanzmann Thrombasthenia (GT) with potential for other debilitating rare bleeding disorders, including factor VII deficiency.

About Hemab Therapeutics

Hemab is a clinical-stage biotech company developing the first prophylactic therapeutics for serious, underserved bleeding and thrombotic disorders. Based in the US and Denmark, Hemab is progressing a pipeline of monoclonal and bispecific antibody-based therapeutics to transform the treatment paradigm for patients with high unmet need. The company’s strategic guidance, Hemab 1-2-5TM, targets the development of 5 clinical assets by 2025 to deliver long-awaited innovation for patients with high unmet need blood clotting disorders like Glanzmann Thrombasthenia, factor VII deficiency, Bernard Soulier Syndrome, Von Willebrand Disease and other serious disorders. Learn more at hemab.com.

# # #

Media Contact
Lia Dangelico
ldangelico@vergescientific.com
540-303-0180

June 15, 2023
Hemab Therapeutics to Present New Preclinical Data on HMB-001 in Factor VII Deficiency and Initial Findings from Glanzmann Thrombasthenia Natural History Study at the International Society on Thrombosis and Haemostasis (ISTH) 2023 Congress
Read More
News: Press Release
Hemab Therapeutics to Present New Preclinical Data on HMB-001 in Factor VII Deficiency and Initial Findings from Glanzmann Thrombasthenia Natural History Study at the International Society on Thrombosis and Haemostasis (ISTH) 2023 Congress

HMB-001 is currently in Phase 1/2 clinical study for Glanzmann Thrombasthenia—early research supports its potential expansion into Factor VII Deficiency

COPENHAGEN, DENMARK AND BOSTON, MASS., US – June 15, 2023 – Hemab Therapeutics, a clinical-stage biotechnology company developing the first prophylactic therapeutics for serious, underserved bleeding and thrombotic disorders, announced today it will present data from preclinical research of HMB-001 in Factor VII (FVII) deficiency at the upcoming International Society on Thrombosis and Haemostasis (ISTH) 2023 Congress, June 24 – 28, in Montreal.

FVII deficiency can cause spontaneous or excessive and prolonged bleeding after injury or surgery; heavy or prolonged menstrual bleeding in women; and in very severe cases, life-threatening bleeding inside the skull or digestive tract.i Hemab’s lead candidate, HMB-001, is a bispecific antibody that binds and stabilizes endogenous Factor VIIa (FVIIa) with one antibody arm and TLT-1 on activated platelets with the other arm. This allows for accumulation of FVIIa in the body and recruitment of FVIIa directly to the surface of the activated platelets where it is known to facilitate the formation of protective hemostatic plugs to stop bleeding.

In addition, initial findings from the Glanzmann’s 360 natural history study, Hemab’s research initiative in partnership with UK specialist research consultancy Haemnet, will be presented at ISTH 2023. This first-of-its-kind study was designed to better define the real-life social, economic and clinical burdens experienced by people living with Glanzmann Thrombasthenia (GT), a rare platelet disorder that causes severe, potentially life-threatening bleeding episodes.

Hemab’s Presentations at ISTH 2023

HMB-001 in Factor VII Deficiency:

  • Oral Presentation 07.4:HMB-001, a Bispecific anti-FVIIa/anti-TLT-1 Antibody Demonstrates Effect in Models of FVII Deficiency
    • Presenting Author: Henrik Østergaard, PhD, MSc, Vice President of Research, Hemab Therapeutics
    • Date/Time: Saturday, June 24, 2023, 1:45-2:00 p.m. ET

First-of-Its-Kind Study of Glanzmann Thrombasthenia:

  • Poster0228: A Prospective, Observational Study of Bleeding and Quality of Life in Patients with Glanzmann Thrombasthenia in the United Kingdom: A First Report
    • Presenting Author: Catherine Rea, MD, PhD, Senior Director of Clinical Research, Hemab Therapeutics
    • Date/Time: Sunday, June 25, 2023, 6:30-7:30 p.m. ET

  • Poster 1375: Menstrual Bleeding in Women with Glanzmann’s Thrombasthenia – Results from the Glanzmann’s 360 Study (GT360)
    • Presenting Author: Kate Khair, RN, RSCN, PhD, Director of Research, Haemnet
    • Date/Time: Tuesday, June 27, 2023, 6:30-7:30 p.m. ET

  • Poster 1377: Living with Glanzmann’s Thrombasthenia: An Interim Report from the Glanzmann’s 360 International Patient Survey
    • Presenting Author: Kate Khair, RN, RSCN, PhD, Director of Research, Haemnet
    • Date/Time: Tuesday, June 27, 2023, 6:30-7:30 p.m. ET

  • Poster 1393: Living with Glanzmann’s Thrombasthenia: An Interim Report on 14 Qualitative Interviews from the Glanzmann’s 360 Study
    • Presenting Author: Kate Khair, RN, RSCN, PhD, Director of Research, Haemnet
    • Date/Time: Tuesday, June 27, 2023, 6:30-7:30 p.m. ET

About HMB-001

HMB-001 is bispecific antibody that binds and stabilizes endogenous factor VIIa (FVIIa) with one antibody arm and TLT-1 on activated platelets with the other arm. This allows for accumulation of FVIIa in the body, recruitment of FVIIa directly to the surface of the activated platelets where it is known to facilitate clotting, and avoidance of clotting activity in the absence of tissue damage. HMB-001 was designed to be a first-in-class prophylactic treatment for Glanzmann Thrombasthenia (GT) with potential for other debilitating rare bleeding disorders, including Factor VII deficiency.

HMB-001 entered Phase 1/2 clinical evaluation in late 2022 for GT, with initial data expected 2H 2023.

About Hemab Therapeutics

Hemab is a clinical-stage biotech company developing the first prophylactic therapeutics for serious, underserved bleeding and thrombotic disorders. Based in the US and Denmark, Hemab is progressing a pipeline of monoclonal and bispecific antibody-based therapeutics to transform the treatment paradigm for patients with high unmet need. The company’s strategic guidance, Hemab 1-2-5TM, targets the development of 5 clinical assets by 2025 to deliver long-awaited innovation for patients with high unmet need blood clotting disorders like Glanzmann Thrombasthenia, Factor VII Deficiency, Bernard Soulier Syndrome, Von Willebrand Disease and other serious disorders. Learn more at hemab.com.

# # #

iFactor VII deficiency – About the Disease – Genetic and Rare Diseases Information Center (nih.gov), accessed June 12, 2023.


Media Contact
Lia Dangelico
Verge Scientific Communications
540-303-0180

VIEW ALL NEWS
Careers
Redheaded woman in a black tank top smiles into the camera.

Resilient
TOGETHER.

Join us as we work together to give patients with rare clotting disorder a new lifeline and improve health equity around the globe.

APPLY NOW
Woman and man embrace, both smiling into the camera. Young girl in a cheek-to-cheek hug with her mother, both smiling into the camera. Woman with glasses and a pink headscarf smiles into the camera. Man in a tan shirt smiles into the camera. Man with glasses and a black sweater smiles into the camera. Young girl with glasses in a yellow sweater smiles into the camera. Man in green shirt hugs woman in gray sweater, both smiling into camera. Redheaded woman in a black tank top smiles into the camera. Man with glasses in a gray sweater smiles into the camera. Young boy in a black shirt smiles into the camera with an adult’s arms around him. Redheaded man in a doctor’s coat with stethoscope folding his arms and smiling into the camera. Woman with white hair and a gray shirt smiles into the camera.