PTC Therapeutics' Kebilidi gains FDA accelerated approval as the first brain-administered gene therapy in the U.S. for AADC ...

The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The gene therapy ...

PTC Therapeutics (NASDAQ:PTCT) has received FDA approval for its gene replacement therapy Kebilidi for the treatment of ...

Enrollment and dosing complete in single ascending dose clinical trial of anti-tau antibody VY7523 for Alzheimer's; on track to generate top-line data H1 2025 - - Recent third-party clinical data esta ...

Elraglusib is undergoing investigation in an open-label, multicenter, phase 1/2 trial for the treatment of patients with ...

Actuate Therapeutics said its treatment for Ewing sarcoma was granted rare pediatric-disease designation from the Food and Drug Administration. Chief Executive Daniel Schmitt said early clinical data ...

Rare Pediatric Disease Designation is granted by the FDA for serious ... that could be utilized by the Company or potentially sold to another company for its use. About Actuate Therapeutics, Inc.

Oak Hill Bio, Chiesi announce first European patient enrolled in phase 2b study of OHB-607 for prevention of BPD in premature infants: Parma, Italy Tuesday, November 5, 2024, 14:0 ...

According to analysts' consensus estimate, Intellia Therapeutics ( NTLA -6.14%), a clinical-stage biotech, boasts a substantial upside. Its shares could jump by 354% if we go by their average price ...

GENEVA, SWITZERLAND / ACCESSWIRE / November 4, 2024 /RELIEF THERAPEUTICS (SIX:RLF)(OTCQB:RLFTF)(OTCQB:RLFTY) (Relief, or the ...

Oct. 30, 2024 (GLOBE NEWSWIRE) -- October 30, 2024 – Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare ...