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Aug 23, 2026
Gene therapies for Fabry and Gaucher disease showed sustained benefits in early trials: all 11 Fabry patients stopped enzyme replacement therapy, and four of six Gaucher patients stayed off standard treatment for up to two years.
Feb 24, 2026
Myriad Genetics, uniQure, and Legend Biotech have scheduled Q4 and full year 2025 earnings calls for Feb 23, Mar 2, and Mar 10, 2026, respectively. Details include webcast access, dial-in numbers, and company background.
Jun 09, 2026
FDA granted priority review to Sanofi's venglustat for type 3 Gaucher disease (action date Nov 25, 2026). Phase 3 LEAP2MONO data showed venglustat met both primary endpoints and three of four key secondary endpoints.
Jun 08, 2026
Sangamo Therapeutics has retained Raymond James to evaluate strategic alternatives to advance its pipeline and maximize stakeholder value. Key assets include the BLA-ready Fabry disease gene therapy ST-920, the STAC-BBB capsid platform generating $88M in fees to date, and multiple neurology programs. No transaction has been agreed and no timetable has been set.
Mar 21, 2026
uniQure disclosed the FDA will not accept Phase I/II data alone for AMT-130 approval, causing shares to drop 49%. The company held regulatory meetings and reported $622.5 million in cash while investors face an April 13 class action deadline.
Mar 03, 2026
Relay Therapeutics reported full-year 2025 revenue of $15.36 million and a narrowed net loss of $276.48 million, while securing FDA Breakthrough Therapy designation for lead asset zovegalisib. The company filed a $96.18 million shelf registration.
Mar 02, 2026
uniQure received FDA feedback stating current AMT-130 data unlikely to support accelerated approval for Huntington's disease, while securities fraud lawsuits challenge prior disclosures. Fresh Fabry disease trial data showed elevated enzyme activity but prompted a dosing pause for safety review.
Mar 02, 2026
Over 100 children with rare diseases in India have exhausted the Rs 50 lakh financial assistance limit under the National Policy for Rare Diseases 2021, forcing treatment halts and prompting urgent appeals for continued support.
