Advanced Therapies
Regenxbio and Solid Biosciences believe they have a next-generation approach to AAV-based gene therapies – but must distance themselves from Sarepta’s safety and efficacy problems.
The company had planned to keep its DMD gene therapy available for ambulatory patients, but now says pausing shipments may enable a better working relationship with the US FDA while safety labeling is updated.
Sarepta knew about the death in a Phase I trial of SRP-9004 for LGMD when it recently revealed a restructuring plan, but did not disclose the event. The now-discontinued gene therapy candidate uses the same AAV vector as Elevidys, which has been linked to two DMD patient deaths.
CMS said that 33 US states plus the District of Columbia and Puerto Rico had joined a program whereby the agency negotiates outcomes-based contracts on their behalf.
Nanoscope’s experimental gene therapy, MCO-010, is designed to target the broader retinitis pigmentosa population, regardless of the underlying gene mutation, with a completed US regulatory submission expected in early 2026.
Seven weeks before its action date, Capricor got an FDA complete response for cell therapy deramiocel in DMD-related cardiomyopathy. It has a Phase III study nearing readout, though.
The Kevin Tang-led firm will pay $4.38 per share for Cargo, which laid off most of its workforce following the failure of a Phase II CAR-T trial.
Relaxing CAR-T restrictions may not be an access panacea because of patient fragility and lack of payer incentive. Conversely, the ACIP’s recent recommendation on Thiomersal-containing flu vaccines is probably the thin edge of a more restrictive wedge.
The gene therapy developer appointed a new president, chief medical officer and chief of staff following the closing of its acquisition by private equity firms Carlysle and SK Capital.
The drugmaker will spend up to $2.1bn to acquire the privately held biotech firm and its targeted lipid nanoparticle technology platform, with an anti-CD19 asset in the clinic.
The FDA eliminated REMS requirements for approved CAR-T cell therapies that have limited their availability, a move that could enable increased use in the community setting.
With financial help from the founder of Uniqlo, Japan’s leading facility for induced pluripotent stem cells is aiming to automate production to slash costs and kick-start R&D in the field.
The biotech announced results from the pivotal trial of isaralgagene civoparvovec and plans to file for approval in 2026 as it looks for a commercialization partner.
A partnership with Germfree Laboratories will bring a new facility to boost Saudi Arabia’s biotech sector as the world’s largest oil exporter tries to establish itself as a global biotech hub by 2040.
The off-the-shelf stem cell therapy shows sustained glycemic control and a high rate of insulin independence.
While some are calling for Syncona to be wound up, the UK-based company believes many existing and new investors will back the creation of new private fund.
Oral RNA splicing modulator has gone into a Phase II/III trial
The country’s life sciences investment head tells Scrip that it is business as usual for the sector despite the rumblings from across the Atlantic.
Instead of opting in on Phase II-ready PCSK9-targeted VERVE-102, Lilly will buy its partner for up to $1.3bn. Analysts call the deal a bargain for Lilly, but fair to Verve.
With two deaths in non-ambulatory DMD patients, Sarepta is attempting damage control and will ask the US FDA to advise, setting up a possible confrontation with CBER director Prasad.