FiercePharma1d
PTC wins FDA approval for first brain-delivered gene therapy Kebilidi
The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The gene therapy ...
GEN1d
AI Collaboration Between Sanofi and Healx Will Target Novel Rare Diseases Indications
Healx will use its AI-based drug discovery tech to analyze proprietary Sanofi compound data and identify potential rare ...
Investing2d
PTC Therapeutics's SWOT analysis: rare disease biotech stock faces pivotal year
However, analysts note potential flexibility from the FDA regarding rare diseases, which could benefit PTC Therapeutics' approval strategies. PTC Therapeutics is well-positioned in the rare ...
CAMP4 Analyst Highlights Rare Disease Platform Potential
CAMP4 Therapeutics raises $75 million in IPO, advances RNA therapies for rare diseases, and partners with BioMarin. Analysts ...
JD Supra9d
FDA Amplifies Cross-Center Collaboration with the Creation of the Rare Disease Innovation Hub
and the Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program. Unifying the Centers’s efforts is poised to bring greater advances in identifying promising drug ...
Investing2d
Zevra Therapeutics' SWOT analysis: rare disease drug maker's stock poised for growth
Zevra Therapeutics, Inc. (NASDAQ:ZVRA), a biopharmaceutical company focused on developing and commercializing novel medicines for rare diseases, has recently achieved a significant milestone with ...
Actuate announces FDA rare pediatric disease designation to elraglusib
Actuate Therapeutics (ACTU) announced that the U.S. Food and Drug Administration, FDA, has granted rare pediatric disease designation to ...
PTC Therapeutics wins FDA nod for gene therapy
PTC Therapeutics (NASDAQ:PTCT) has received FDA approval for its gene replacement therapy Kebilidi for the treatment of ...
Business Wire4d
Oranda Therapeutics Announces Series A Funding Round to support its mission: to improve the lives of patients living with rare disease
DUBLIN--(BUSINESS WIRE)--Oranda Therapeutics, a new Irish headquartered rare disease company with an innovative, fast to market, commercial approach, today announces the launch of their Series A ...
Actuate Therapeutics: FDA Grants Pediatric-Disease Status to Ewing Sarcoma Treatment
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 ...
Investing2d
PTC Therapeutics's SWOT analysis: rare disease biotech stock faces pivotal year
However, analysts note potential flexibility from the FDA regarding rare diseases, which could benefit PTC Therapeutics' approval strategies. PTC Therapeutics is well-positioned in the rare disease ...
Investing2d
Zevra Therapeutics' SWOT analysis: rare disease drug maker's stock poised for growth
Zevra Therapeutics, Inc. (NASDAQ:ZVRA), a biopharmaceutical company focused on developing and commercializing novel medicines for rare diseases, has recently achieved a significant milestone with the ...