The US FDA has unveiled draft guidance designed to assist rare disease therapy developers by providing regulatory clarity in ...
In a phase 2 study, SAR402663 gene therapy for wet age-related macular degeneration showed positive efficacy and safety ...
Sujith Ramachandran, PhD, associate director of Trinity Life Sciences, grouped access barriers into 3 buckets: upfront ...
Cell and Gene Therapies (CGTs) present unique development challenges, from novel manufacturing processes and delivery methods ...
Cardiologists who study gene therapy explain its promise, risks and the safeguards needed for responsible use.
AviadoBio Presents Preclinical Data Validating the vMiX™ Platform for AVB-406 in Alzheimer’s Disease and for AVB-SNCA in Parkinson’s Disease at ESGCT ...
FDA proposes streamlined approvals for personalized gene editing as CMS expands therapy access model
FDA draft guidance lets sponsors add patient-specific variants to one BLA for personalized genome editing therapies. Read on for CMS access details.
Orphan Therapeutics Accelerator licenses OTXL-101, an investigational AAV liver-targeted gene therapy for ornithine transcarbamylase deficiency. Read more.
REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will host a conference call on Thursday, November 5, at 8:00 a.m. ET to ...
The number of approvals is really only 1 piece of the pie,” said Nicole A. Caffiero, PharmD, MBA, BCACP, senior clinical ...
Adjuvia Therapeutics, a biotechnology company developing novel therapeutics to treat mitochondrial dysfunction and disease, ...
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