| 05.07.25 | Vertex suspends Moderna-partnered CF trial; Neurocrine passes 2 gene therapy programs back to Voyager

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Market Access Insights from AXS25

Explore dynamic forces reshaping how therapies reach patients and gain actionable insights on market access and patient support from industry experts in this podcast series from Asembia's AXS25 Summit. Listen now.

Today’s Big News

May 7, 2025

Vertex pauses Moderna-partnered cystic fibrosis trial, takes $379M hit tied to separate program

Neurocrine hands back 2 CNS gene therapy programs to Voyager

FDA Commissioner Marty Makary taps Vinay Prasad to head up CBER

C4 Therapeutics halts BRAF degrader work to save cash for cemsidomide

Trump's new executive order targets gain-of-function research, citing unsupported 'lab leak' theory

Marea rises toward phase 2b after showing antibody cuts remnant cholesterol

Beacon's rare eye disease gene therapy signals vision function improvements in early phase 2 data

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Featured

Vertex pauses Moderna-partnered cystic fibrosis trial, takes $379M hit tied to separate program

By Gabrielle Masson

Vertex Pharmaceuticals is pressing pause on a phase 1/2 cystic fibrosis trial over a tolerability issue, while reporting a separate impairment charge of $379 million tied to an earlier discontinuation.

Neurocrine hands back 2 CNS gene therapy programs to Voyager

By James Waldron

Neurocrine has handed back two discovery-stage gene therapy programs to Voyager Therapeutics, although the companies’ wider central nervous system-focused collaboration remains on track.

FDA Commissioner Marty Makary taps Vinay Prasad to head up CBER

By Eric Sagonowsky

In the wake of the high-profile departure of Peter Marks, M.D., Ph.D., the FDA has a new leader for its Center for Biologics Evaluation and Research.

Pushing for innovation amid a ‘silent surge’ of rising MASH rates

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C4 Therapeutics halts BRAF degrader work to save cash for cemsidomide

By James Waldron

C4 Therapeutics is continuing to saw off parts of its pipeline, this time ending work on its clinical-stage BRAF degraded program in order to focus on its IKZF1/3 degrader cemsidomide.

Trump's new executive order targets gain-of-function research, citing unsupported 'lab leak' theory

By Darren Incorvaia

President Donald Trump is continuing to promote the unsupported “lab leak” COVID-19 theory, using it now to rationalize a new executive order designed to prohibit federal funding of certain research on pathogens abroad and severely limit such research in the U.S.

Marea rises toward phase 2b after showing antibody cuts remnant cholesterol

By Nick Paul Taylor

Marea Therapeutics has linked its lead candidate to a 53% placebo-adjusted mean reduction in remnant cholesterol, positioning the biotech to start a phase 2b trial imminently.

Beacon's rare eye disease gene therapy signals vision function improvements in early phase 2 data

By Zoey Becker

The company's gene therapy made for improvements in visual function for patients with X-linked retinitis pigmentosa at a six month interim of its phase 2 study.

Masimo reports cyberattack slowing manufacturing, sales

By Conor Hale

Masimo has disclosed that a cyberattack on its systems has hit its manufacturing facilities, slowing production at the remote patient monitoring company.

Sarepta tries to quell concerns over DMD gene therapy Elevidys as patients delay treatment after death report

By Angus Liu

Sarepta Therapeutics CEO Doug Ingram opened the company’s first-quarter earnings call Tuesday by acknowledging that, like the broader biopharma industry, the genetic medicine biotech is facing a challenging time.

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