Phase I
Results from a Phase I study presented Friday at the ASGCT 2024 annual meeting showed that despite a good safety profile, Excision BioTherapeutics’ HIV gene editor failed to suppress viral activity.
Regeneron Pharmaceuticals on Wednesday revealed that its investigational gene therapy DB-OTO restored hearing in two young children, according to an oral presentation at the American Society of Gene & Cell Therapy annual meeting.
The biotech touted its prime editing technology at ASGCT on Tuesday after receiving FDA clearance last week for a clinical study of a drug candidate based on the platform.
The FDA has cleared a clinical trial of an ex vivo prime editing candidate in patients with a rare disease, Prime Medicine announced Monday. The technique taps CRISPR technology to rewrite defective genes without breaking DNA double helix strands.
Several companies—including Roche, Curemark and Yamo Pharmaceuticals—are running clinical trials of treatments that aim to offset the myriad challenges of autism.
While the San Diego-based biotech awaits a Phase IIa data readout for its lead lung disease candidate, Endeavor BioMedicines’ next-generation HER3-targeted antibody-drug conjugate will start a Phase I/II trial later this year.
A one-time treatment for Parkinson’s disease could be a ‘market changer,’ experts told BioSpace, adding that cell therapies could limit the adverse effects seen with current drugs.
Drugmakers, including the genetic testing company 23andMe, are using novel approaches to stimulate NK cells’ intrinsic ability to recognize and kill tumor cells.
In a Phase I/II study, Ultragenyx’s antisense oligonucleotide GTX-102 led to significant cognitive, behavioral, sleep and gross motor improvements in patients with the rare genetic disorder Angelman syndrome.
Following cases of convulsions in rabbits in a preclinical study, the FDA has placed a clinical hold on Neumora Therapeutics’ Phase I schizophrenia drug candidate NMRA-266.
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