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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

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

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.

Fierce podcasts

Don’t miss an episode

ElevAAte and the push for East Asian American leaders in biopharma

This week on "The Top Line," hear how the founders of ElevAAte are turning shared East Asian American experiences into collective power for the next generation of biopharma leaders.

Angus on Angus: A Fierce oncology discussion

Managing diabetes with cultural foods

Resources

Whitepaper

Accelerating Clinical Trial Excellence: A Seamless IRB & IBC Collaboration

Don’t delay your gene transfer trial’s start-up timeline by gaining the latest insights from WCG’s case study on the power of a seamless Institutional Review Board (IRB) and Institutional Biosafety Committee (IBC) review process.

SpyLock Technology: A cutting-Edge Solution for Rapid Bispecific Antibody Screening

Learn how the SpyLock service can offer fast bispecific generation, reduce workload, and accelerate candidate selection.

Best Practice Medical Affairs Organization Study Report

We interviewed 24 Medical Affairs leaders on the critical success factors for building a Best Practice Medical Affairs organization. This report shares what we learned.

Presented by Blue Matter, strategic consultants in the life sciences

eBook

Through a CRO's perspective: Innovative, Agile Solutions In Vaccine Efficacy Testing

Typical vaccine development timelines have averaged 10 years, but the time from the SARS-CoV-2 genetic sequence’s release to the submission of the first vaccine clinical trial data for regulatory review was a mere 314 days. In IQVIA Laboratories’ Vaccine eBook we focus on how contract research organizations (CROs) can best leverage strategic partnerships with sponsors to prepare for the next pandemic.