Regenxbio

Suspicious activity violating TipRanks' Terms of Use detected, including excessive page views or use of scraping tools. Account usually reactivates within 24 hours; contact support if still disabled.

ABBV-RGX-314 shows 97% reduction in treatment burden at nine months, with zero cases of intraocular inflammation and potential for bilateral wet AMD treatment.

REGENXBIO to present ABBV-RGX-314 data for wet AMD at AAO 2024: subretinal delivery results on Oct 19, suprachoroidal delivery for DR on Oct 21, and long-term follow-up for nAMD on Oct 21.

The FDA approved 55 new drugs last year, emphasizing the importance of selecting appropriate clinical outcome assessments (COAs) in drug development. COAs are crucial for collecting patient data and securing regulatory approval, ensuring outcomes are meaningful to patients. Effective COAs must be relevant, psychometrically sound, and meet regulatory standards. Poor COA selection risks misleading results, regulatory delays, and patient burden. Pearson assessments, used in successful drug development for conditions like schizophrenia and spinal muscular atrophy, highlight the integral role of well-validated COAs in advancing drug development and securing approval.

Andrew Miller's invention, KarXT, secures FDA approval, prompting his first strategic move.

Tagworks Pharmaceuticals appoints Ken Mills as CEO, with founding CEO Marc Robillard transitioning to Chief Scientific Officer. Mills, a 25-year biotech veteran, previously led REGENXBIO through IPO and oversaw multiple clinical trials. He aims to expand Tagworks' ADC platform for cancer treatment.

John Brandsema, MD, discusses ongoing research for Duchenne muscular dystrophy (DMD), including exon skipping agents, PPMOs, sevasemten, cell-based therapies, and gene transfer therapies. He emphasizes the need for further optimization and the importance of newborn screening for early diagnosis.

Kyverna appoints Warner Biddle as CEO, Christi Shaw to board. Regenxbio welcomes Mitchell Chan as CFO. Mirador Therapeutics appoints Gregg Gilbert as CFO.

RGX-121, an investigational gene therapy, showed an 85% median reduction in cerebrospinal fluid (CSF) levels of heparan sulfate (HS) D2S6 in patients with mucopolysaccharidosis type II (MPS II), supporting its potential as the first gene therapy for this condition. The therapy demonstrated sustained reductions for up to 2 years and was well-tolerated, with REGENXBIO planning to initiate a rolling biologics license application (BLA) in Q3 2024. The BLA will use CSF D2S6 as a surrogate end point to predict clinical benefit.