A recent study conducted by researchers from the TIDU GENOV at the Institut du Cerveau in Paris, France, presents promising results for the treatment of Metachromatic Leukodystrophy (MLD), a severe neurodegenerative disease. Led by Emilie Audouard with key...
After hitting a speed bump in its first attempt at developing a gene therapy for Friedreich’s ataxia, Astellas is ready to put a new version in the clinic. On Thursday, the Japanese drugmaker announced the FDA has cleared its IND for ASP2016. The company is hoping to...
The FDA’s Center for Biologics Evaluation and Research is planning to release some crucial guidance soon for CRISPR and gene therapy developers on platform technologies, accelerated approvals for rare diseases, and potentially in the “not-too-distant future,” a...
The U.S Food and Drug Administration (FDA) has granted approval to ImmunityBio, Inc. (NASDAQ: IBRX) for their immunotherapy treatment, ANKTIVA® (N-803), in combination with Bacillus Calmette-Guérin (BCG), for patients with BCG-unresponsive non-muscle invasive bladder...
Company unveils Parkinson’s disease associated with GBA mutations (PD-GBA) program; two oral presentations feature new data from Capsida’s wholly owned programs in PD-GBA and genetic epilepsy due to STXBP1 mutations; both programs in IND-enabling studies...