CB 2679-GT / GC Biopharma - LARVOL DELTA

Using Recombinant Adeno-Associated Viral Vectors for Long-Term Expression of a Hyperactive Human Factor IX Mutant in Hemophilic Mice and Comparison of AAV-LK03 and AAV-KP1 in Nonhuman Primates (ASGCT 2022) - "Our study demonstrates that combining a next generation AAV vector with the potency enhanced FIX variant CB 2679d-GT has the potential to improve transgene expression and effectively lower the viral dose needed to achieve therapeutically relevant FIX activity levels when administered systemically. This would substantially reduce the cost of rAAV based FIX gene therapy, the potential for liver toxicity and the risk of adverse immune responses with high doses."

Preclinical • Gene Therapies • Hematological Disorders • Hemophilia • Hepatology • Rare Diseases • APOE

CATALYST BIOSCIENCES : Management's Discussion and Analysis of Financial Condition and Results of Operations (form 10-Q) (Market Screener) - "We have licensed AAV technology from The Board of Trustees of The Leland Stanford Junior University ('Stanford') and are currently optimizing the vector under a sponsored research agreement with Stanford. Data presented at the European Association for Haemophilia and Allied Disorders ('EAHAD') showed that the combination of our proprietary potency enhanced CB 2679d-GT Factor IX construct with a novel chimeric AAV capsid may reduce the vector dose required in gene therapy while maintaining high Factor IX levels."

Licensing / partnership • Preclinical • Genetic Disorders • Hemophilia

Gene Therapy For Hemophilia B Using CB 2679d-GT: A Novel Factor IX Variant With Higher Potency Than Factor IX Padua. (PubMed, Blood) - "FIX expression was sustained for at least 20 weeks with both CB 2679d-GT and R338L-Padua while immunogenicity was not significantly increased. This is a novel gene therapy study demonstrating the superiority of CB 2679d-GT highlighting its potential to obtain higher FIX activity levels and superior hemostatic efficacy following AAV directed gene therapy in hemophilia B patients than what is currently achievable with the R338L-Padua variant."

Journal • Gene Therapies • Hematological Disorders • Hemophilia • Hepatology • Immunology • Inflammation • Rare Diseases

Catalyst Biosciences Reports Second Quarter 2020 Operating & Financial Results and Provides a Corporate Update (GlobeNewswire) - "'We are currently on track to achieve additional important milestones in the second half of 2020...We closed the quarter with approximately $117 million in cash putting us in a strong position to deliver on these upcoming milestones'....MarzAA: Enroll the first patient in a Phase 3 open-label trial in late 2020, evaluating the efficacy of SQ MarzAA to treat episodic bleeding in individuals with hemophilia A or B with inhibitors. Initiate a Phase 1/2 trial in FVII Deficiency, Glanzmann Thrombastenia, and patients using Hemlibra, in late 2020. Timelines for both trials are subject to the readiness of trial sites and potential effects of COVID-19 on clinical trial execution. CB 2679d-GT (FIX Gene Therapy): Announce a development candidate in late 2020."

Clinical • Enrollment status • Financing • New P1/2 trial • Hemophilia

Catalyst Biosciences Presents Preclinical FIX Gene Therapy Data in an Oral Presentation at the World Federation of Hemophilia Virtual Summit 2020 (Catalyst Biosciences Press Release) - "Catalyst Biosciences, Inc....today presented data from preclinical studies of its hemophilia B gene therapy CB 2679d-GT at the World Federation of Hemophilia Virtual Summit, taking place from June 14 -19, 2020. The oral presentation, entitled: 'Combination of a Novel Chimeric AAV Capsid and Potency Enhanced FIX Variant for Hemophilia B Gene Therapy,' given by Dr. Grant Blouse, senior vice president of translational research, provided preclinical results of CB 2679d-GT...The study demonstrated that CB 2679d-GT was well tolerated with high FIX expression that stabilized to approximately 25% to 50% FIX above baseline levels at the 6-week interim data cutoff."

Preclinical • Hemophilia

Catalyst Biosciences announces oral and poster presentations at the World Federation of Hemophilia Virtual Summit 2020 (Catalyst Biosciences Press Release) - "Catalyst Biosciences, Inc....today announced oral and poster presentations at the upcoming World Foundation of Hemophilia Virtual Summit, taking place from June 14-19, 2020....The open-label Phase 2b study of dalcinonacog alfa (DalcA), a next-generation subcutaneously (SQ) administered Factor IX (FIX) for the treatment of hemophilia B was designed to evaluate daily SQ dosing and the ability to maintain protective steady state FIX levels above 12% in six individuals with severe hemophilia B....Dr. Blouse will present data from preclinical studies of Catalyst’s hemophilia B gene therapy CB 2679d-GT..."

P2b data • Preclinical • Hemophilia