Regeneron gene therapy restores hearing in 10 kids

Today’s Big News

Feb 25, 2025

Takeda fronts $46M for 2nd small molecule collab with BridGene


Repare unveils scale of layoffs with 75% of staff and chief medical officer heading for exit


Lava, smoldering from dropping lead asset, lays off 30% of staff to save cash


Polish biotech Ryvu waves goodbye to 30% of staff in focus on phase 3 blood cancer trials


Regeneron gene therapy improves hearing in 10 children with rare form of hearing loss


Kiniksa adds to Sjögren’s exodus with trial termination, axes AstraZeneca asset 


Deerfield sues to stop Alcon's 'unrelenting campaign' to take over Aurion


BioCity's ETA antagonist reduces proteinuria in phase 2 trial of diabetic kidney disease 


FDA works to reverse layoffs, rehire lost staff: reports

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Featured

Takeda fronts $46M for 2nd small molecule collab with BridGene

Takeda has returned to BridGene Biosciences for another small-molecule collaboration—this time focused on immunology as well as neurology.
 

Top Stories

Repare unveils scale of layoffs with 75% of staff and chief medical officer heading for exit

For the last six weeks the shadow of potential layoffs has hung over Repare Therapeutics’ employees. Monday, staff learned that three-quarters of them are heading for the exits.

Lava, smoldering from dropping lead asset, lays off 30% of staff to save cash

With Lava Therapeutics still smoldering from the decision to dump its lead blood cancer drug, the biotech is now laying off 30% of its staff in an effort to conserve cash.

5 Market Access Mistakes That Can Derail Your EU Orphan Drug Launch

Avoid common pitfalls in EU orphan drug market access—learn how local expertise can help navigate pricing, reimbursement, and regulatory hurdles.

Polish biotech Ryvu waves goodbye to 30% of staff in focus on phase 3 blood cancer trials

Ryvu Therapeutics is shrinking its head count by 30% as the Polish biotech focuses its funds on taking its lead blood cancer drug through phase 3 trials.

Fortrea's Alaric Jackson Talks AI in Clinical Trials

Fortrea's CTO discusses AI's expanding role in clinical trials, from discovery to execution, and its potential to improve patient outcomes and diversity.

Regeneron gene therapy improves hearing in 10 children with rare form of hearing loss

Regeneron’s investigational gene therapy has been tied to notable hearing improvements in 10 of 11 children who were treated for a rare genetic condition that causes hearing loss.

Kiniksa adds to Sjögren’s exodus with trial termination, axes AstraZeneca asset

Kiniksa Pharmaceuticals is stopping development of a midphase autoimmune asset and returning a drug candidate to AstraZeneca as it doubles down on its cardiovascular disease pipeline.

Deerfield sues to stop Alcon's 'unrelenting campaign' to take over Aurion

As Alcon Research seeks to block investee Aurion Biotech from listing on the New York Stock Exchange, another investor has come to Aurion’s defense.

BioCity's ETA antagonist reduces proteinuria in phase 2 trial of diabetic kidney disease

BioCity Biopharma’s selective endothelin receptor type A antagonist has reduced excess proteins in the urine of patients with diabetic kidney disease, hitting a primary endpoint of a phase 2 trial.

FDA works to reverse layoffs, rehire lost staff: reports

After hundreds of FDA employees were suddenly laid off last weekend, including dozens at the agency’s device center, the government has begun rescinding some of those cuts and attempting to rehire lost workers.

Antibodies stop tissue damage and reduce virus levels in mice with deadly Crimean-Congo hemorrhagic fever

Researchers studying a deadly virus discovered a key protein behind its lethality and successfully used antibodies to stop that protein from causing dangerous fluid leakage in mice.

In letter to RFK Jr., Democrats flag threats to drug review process from FDA firings

HHS secretary Robert F. Kennedy Jr. has been on the receiving end of two heated letters from lawmakers outlining the threats to innovation posed by mass firings at U.S. health agencies.

Eli Lilly, Novo Nordisk lead revenue boom in the 'always tricky' Q4

Revenues in the biopharma industry continued to boom in the fourth quarter of last year, following a pattern seen increasingly throughout 2024. Of the world’s 22 biopharma companies that have reported quarterly revenues of at least $2 billion, only one posted a year-over-year decline. And that decline came with a caveat.

HHS makes Elon Musk-directed email demands voluntary, warns of foreign actor threat

Elon Musk is facing a lawsuit from federal workers over a mass email requiring government employees to list productive tasks from the prior week or risk termination. HHS leadership instructed staff a response is voluntary, with a new set of guidelines to follow.
 
Fierce podcasts

Don’t miss an episode

What were the biggest clinical trial flops of 2024?

This week on "The Top Line," we dive into several of the biggest failures in clinical-stage drug development last year and discuss what the biopharma industry can learn from these setbacks.
 

Resources

Whitepaper

The Oncology Market: 2024 Year in Review

This paper reviews key developments in the oncology market during 2024 and outlines what they mean for the future.

 

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