Von Willebrand Diseases

SOUTH SAN FRANCISCO, Calif.--( BUSINESS WIRE )-- Star Therapeutics , a clinical stage biotechnology company discovering and developing best-in-class antibodies, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for VGA039 in von Willebrand disease (VWD). VGA039 is an investigational monoclonal antibody that targets Protein S to restore balance in blood clotting, as a universal hemostatic therapy for numerous bleeding disorders. It is potentially the first subcutaneous therapy that addresses all types of VWD and has a convenient dosing regimen. “VGA039 is the first drug candidate to receive Fast Track designation for VWD, and we are committed to advancing drug innovation for this debilitating disease that has lagged behind other bleeding disorders,” said Adam Rosenthal, Ph.D., CEO and Founder of Star Therapeutics. “Fast Track designation enables us to potentially accelerate the development path for VGA039 as a therapy that can transform the way VWD is treated with a convenient, subcutaneous therapy for patients with all types of VWD.” The FDA grants investigational medicines Fast Track designation to facilitate the development and expedite the review of medicines that demonstrate the potential to treat serious conditions and fill an unmet medical need. Programs with Fast Track designation can benefit from early and more frequent interactions with the FDA to discuss the product candidate’s development plan in addition to a rolling submission of the marketing application. Product candidates with Fast Track designation may also be eligible for priority review and accelerated approval. About VGA039 VGA039 is a monoclonal antibody therapy with a novel mechanism of action that targets Protein S, a key component in restoring balance to the blood clotting process. VGA039 has potential to be a universal hemostatic therapy that can treat numerous bleeding disorders, with the first disease indication of von Willebrand disease (VWD). As a subcutaneously self-administered antibody therapy with a convenient dosing regimen, VGA039 has the potential to dramatically reduce treatment burden for patients. About Star Therapeutics Star Therapeutics is a clinical stage biotechnology company discovering and developing best-in-class antibodies to create life-changing therapies for patients, initially addressing unmet needs in hematology and immunology. The company applies its expertise in antibody innovation to interrogate areas of biology that have been overlooked and have the potential for addressing multiple diseases with a single therapy. Star is backed by leading life sciences investors and located in South San Francisco. For more information, please visit Star Therapeutics and follow us on LinkedIn .

In the current global pharmaceutical market, gene therapy drugs occupy a significant portion of the most expensive drug rankings, with five listed in the top ten. Pfizer's recently approved treatment for moderate to severe Hemophilia B, Beqvez, is priced at a staggering $3.5 million, making it one of the highest-priced drugs in history. Hemophilia is a hereditary bleeding disorder primarily divided into Hemophilia A and B, caused by mutations in the genes for clotting factors VIII and IX, respectively. Patients typically suffer from lifelong coagulation dysfunction, leading to joint bleeding and other severe complications. Currently, replacement therapies with clotting factors are the main treatment method, but frequent long-term injections can lead to poor patient compliance and may produce inhibitory antibodies, affecting treatment efficacy. Although new clotting factor drugs, humanized bispecific antibodies, and monoclonal antibodies that inhibit the tissue factor pathway have made advancements in administration methods and duration of action, these methods still cannot cure hemophilia. For hemophilia patients, gene therapy offers a glimmer of hope for a cure. Through gene therapy, clotting factors can be safely delivered into the patient's cells for long-term stable expression, thereby improving bleeding conditions and coagulation function, which is the ultimate goal of hemophilia treatment. Currently, several gene therapy drugs for hemophilia have been approved, including: HYMPAVZI™ (Matizumab): An anti-TFPI antibody suitable for patients with Hemophilia A or B, developed by Pfizer, approved by the European Commission in November 2024. Vonvendi (Veyinzh®): A recombinant von Willebrand factor (rVWF) for adult patients with von Willebrand disease (VWD), developed by Takeda, approved by China's National Medical Products Administration in August 2024. Beqvez: A neutralizing antibody for adult patients with moderate to severe Hemophilia B, developed by Pfizer, priced at $3.5 million. Hemgenix (AMT060): A gene therapy based on adeno-associated virus 5 (AAV5) for patients with Hemophilia B, developed by UniQure, approved by the U.S. FDA in November 2022. Roctavian: Also based on the AAV5 vector, this gene therapy is for patients with Hemophilia A (HA), priced at $2.9 million per treatment, developed by BioMarin, and received conditional marketing authorization from the European Commission in August 2022. As the hemophilia market continues to grow, it’s expected to reach a scale of billions by 2028. Currently, over 40 gene therapies for hemophilia are in research, with multinational companies like Pfizer, Sanofi, Bayer, and Takeda, as well as domestic firms like China National Pharmaceutical Group and Hualan Biologicals, actively conducting clinical studies. Despite the enormous potential of gene therapy in treating hemophilia, the high costs remain a significant barrier to its widespread adoption.

SOUTH SAN FRANCISCO, Calif.--( BUSINESS WIRE )-- SonoThera ™, a biotechnology company dedicated to treating the root cause of human diseases through genetic therapy, today announced it will present new data on its novel gene delivery platform and therapeutic programs at the 66 th American Society of Hematology (ASH) Annual Meeting and Exposition , being held December 7-10 th in San Diego, California. The preclinical data highlights the ability of ultrasound-mediated gene delivery (UMGD) to deliver nonviral genetic payloads in a durable, titratable, redosable manner while achieving therapeutic FVIII and FIX gene expression levels in murine models. SonoThera’s novel UMGD platform is being developed to noninvasively deliver nucleic acid payloads of diverse formats and sizes safely, selectively targeting a wide range of organs and tissues within the body. “A major challenge to creating safe and effective genetic treatments for hematological diseases such as Hemophilia A and B, and von Willebrand disease remains the ability to efficiently deliver oversized, nonviral DNA payloads to the liver in a safe and redosable manner,” said Kenneth Greenberg , PhD, CEO of SonoThera. “Ultrasound-mediated gene delivery has the ability to address these key issues, and we look forward to presenting our latest data demonstrating its potential.” Presentation Details: Publication Number: 2232 Title: Targeted Non-Viral Gene Delivery through Transcutaneous Ultrasound Enables Robust, Redosable, Titratable, and Durable FVIII and FIX Gene Expression in the Liver in Murine Models. Session: 803. Emerging Tools, Techniques, and Artificial Intelligence in Hematology: Poster I Date: Saturday, December 7, 2024 Time: 5:30 pm – 7:30 pm PST Location: San Diego Convention Center, Halls G-H About SonoThera Founded by Drs. Kenneth Greenberg, Michael Davidson, and Steve Feinstein, SonoThera, Inc. is a biotechnology company dedicated to treating the root cause of human disease through genetic therapy. SonoThera is developing a nonviral, ultrasound-mediated gene delivery platform designed to provide patients with the next generation of safe and effective genetic medicines. The novel platform utilizes sonoporation, a microbubble-mediated biophysical process to noninvasively deliver genetic payloads selectively targeting a wide range of organs within the body. SonoThera is headquartered in South San Francisco, California. Visit www.sonothera.com to learn more.