ProQR Announces Positive Phase 1/2 Study of Sepofarsen
The company announced topline results for its PQ-110-001 study in patients with Leber's congenital amaurosis 10 (LCA10).
Prevail Therapeutics and Lonza Enter Strategic Collaboration
Through the collaboration, the companies will focus on Prevail's gene therapy programs for patients with neurodegenerative diseases.
BrainStorm Receives US Patent Allowance for NurOwn Platform
The company received a notice of allowance for its US Patent Application called 'Method of Qualifying Cells.'
Applied Genetic Technologies Announces Strategic Collaboration with Otonomy
The companies will develop a gene therapy for patients with a sensorineural hearing loss, which is a form of congenital...
Ziopharm Oncology Receives FDA Clearance of Rapid Personalized Manufacture of CD19-specific CAR-T
The company received clearance from the FDA for a Phase 1 clinical trial for patients with relapsed CD19 leukemias and...
NewLink Genetics Receives FDA Acceptance for Partnered Biologics License Application
NewLink Genetics (NASDAQ:NLNK) has announced that the US Food and Drug Administration has accepted Merck’s (NYSE:MRK)’s Biologics License Application and...
Wave Life Sciences Gets Fast Track Designation from FDA
The designation from the FDA has been granted for the company's treatment suvodirsen to treat Duchenne muscular dystrophy.