Summary Astellas has declined to exercise its option on AviadoBio’s AAV-based gene therapy program targeting frontotemporal dementia (FTD), but the program will continue through nonprofit funding support. The development highlights an alternative pathway for sustaining high-risk, rare-disease programs despite pharmaceutical…
Summary The RNA therapeutics field is approaching a key inflection point with the anticipated initiation of the first clinical trial of engineered transfer RNA (tRNA) therapy, led by emerging players such as Alltrna. This modality targets nonsense mutations, representing a…
Summary Tempest Therapeutics reported its year-end 2025 business update, highlighting two key developments: the acquisition of dual-targeting CAR-T assets from Factor Bioscience and positive interim data from the ongoing REDEEM-1 Phase 1/2a trial of TPST-2003 in relapsed/refractory multiple myeloma. While…
Summary Fortress Biotech, through its subsidiary Cyprium Therapeutics, has completed the sale of a Rare Pediatric Disease Priority Review Voucher (PRV) for $205 million following the FDA approval of ZYCUBO (copper histidinate) for Menkes disease. The transaction represents a significant…