Market News
Global Fanconi Anemia Drug Market- Recent Developments
Research and Development Activities
- On October 10, 2023, according to the Thalassaemia International Federation (TIF),a non-profit, non-governmental organization, a study found 53.8% disease-free survival and 66.8% five-year overall survival at a median follow-up of 8.7 years associated with allogeneic hematopoietic stem cell transplantation for patients with myelodysplastic syndrome or acute myeloid leukemia resulting from Fanconi anemia. Two-thirds of patients who received allogeneic hematopoietic stem cell transplantation (allo-HSCT) to treat myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) resulting from Fanconi anemia (FA).
- On September 21, 2023, Jasper Therapeutics, Inc., a biotechnology company that focuses on developing novel antibody therapies targeting c-Kit (CD117) to address diseases such as chronic spontaneous urticaria and lower to intermediate risk myelodysplastic syndromes (MDS) as well as novel stem cell transplant conditioning regimes, announced new positive Phase 1b data on briquilimab as a conditioning agent in the treatment of Fanconi Anemia (FA)
- On April 27, 2023, The California Institute for Regenerative Medicine (CIRM) approved investing nearly US$ 89 million in projects from its Clinical and Translation programs which includes project Reduced intensity conditioning with JSP191 prior to TCRαβ+ T-cell/CD19+ B-cell depleted hematopoietic stem cell transplant for Fanconi Anemia patients from Stanford University, California, U.S.
- On May 16, 2022, Rocket Pharmaceuticals, Inc., a late-stage, clinical biotechnology company, announced positive clinical updates from its Phase 2 pivotal trial for Fanconi Anemia (FA). The Phase 2 pivotal study in Fanconi Anemia, five of nine patients sustained increasing bone marrow cell resistance to mitomycin-C (MMC) confirmed over two consecutive time points.
- On December 12, 2022, Rocket Pharmaceuticals, Inc., a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, announced positive clinical data from its lentiviral (LV)-based gene therapy programs such as Lentiviral-mediated Gene Therapy for Patients with Fanconi Anemia [Group A]: Updated Results from Global RP-L102 Clinical Trials
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