11 blind children able to see after MeiraGTx gene therapy treatment

Today’s Big News

Feb 21, 2025

As BioMarin searches for 'clever science,' its R&D chief warns innovation could be hit by NIH fund drought

Procedural ‘loophole’ leaves NIH grants frozen, despite orders from federal judge: reports

MeiraGTx gene therapy allows 11 blind children to see as biotech eyes accelerated approval

Concentra curveball offer throws Acelyrin merger with Alumis into doubt

US biotech Hookipa retracts merger plans with UK company

Chutes & Ladders—NIH leaders join health agency exodus

What were the biggest clinical trial flops of 2024?

The Top Line Podcast: Don’t miss out on the newest episode. Listen now.

 

Featured

As BioMarin searches for 'clever science,' its R&D chief warns innovation could be hit by NIH fund drought

By Darren Incorvaia

A slimmed-down BioMarin started the new year open for business, seeking to leverage its unique skillset to strike deals that bolster its pipeline of medicines for genetic diseases.
 

Top Stories

Procedural ‘loophole’ leaves NIH grants frozen, despite orders from federal judge: reports

By Gabrielle Masson

The Trump administration is exploiting an administrative loophole to keep federal research funding frozen, despite a federal judge ordering for a halt to the action, according to Nature.

MeiraGTx gene therapy allows 11 blind children to see as biotech eyes accelerated approval

By Darren Incorvaia

Eleven children who were legally blind at birth all gained visual acuity after receiving MeiraGTx's investigational gene therapy, data that is prompting the biotech to seek accelerated approval in AIPL1-associated severe retinal dystrophy.

Grifols, through pioneering plasma research, looks to reveal early warning signs of Parkinson's

Sponsored by Grifols

Nearly one million people in the U.S. and more than six million people worldwide have Parkinson's disease (PD), a debilitating neurological condition characterized by progressive motor and non-motor symptoms that significantly affects patients and their loved ones.

Concentra curveball offer throws Acelyrin merger with Alumis into doubt

By James Waldron

Acelyrin had been all set to merge into Alumis—but a surprise offer by Concentra Biosciences has thrown the deal into doubt.

UPDATE: US biotech Hookipa retracts merger plans with UK company

By Gabrielle Masson

After announcing acquisition plans at the beginning of 2025, immunotherapeutics biotech Hookipa Pharma has decided not to buy rare disease company Poolbeg Pharma.

Chutes & Ladders—NIH leaders join health agency exodus

By Darren Incorvaia,Zoey Becker

The NIH has lost two distinguished leaders as mass layoffs directed by the Trump administration hit federal health agencies.

What were the biggest clinical trial flops of 2024?

By Andrea Park,James Waldron,Gabrielle Masson

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.

Pfizer discontinues hemophilia treatment Beqvez, emptying its gene therapy portfolio

By Angus Liu

Pfizer is pulling further away from the gene therapy field with its decision to discontinue hemophilia B product Beqvez. The move leaves no active gene therapy programs in Pfizer's portfolio.

Regulatory tracker: Gilead's seladelpar nabs EU approval in primary biliary cholangitis

By Fraiser Kansteiner,Eric Sagonowsky,Angus Liu,Zoey Becker,Kevin Dunleavy

In this tracker, Fierce Pharma is recording the regulatory progress of in-market products, including expansions into key geographies and new indications. Some of these updates may not meet the bar for standalone stories, but we think they are still worth mentioning.

Fierce Pharma Asia—Novo Nordisk's fraud accusation; AstraZeneca's China deal; Ono's FDA nod

By Angus Liu

Novo Nordisk alleges Singaporean biotech KBP Biosciences made misleading statements before the two firms inked a licensing deal worth up to $1.3 billion. AstraZeneca is buying out its roxadustat partner FibroGen in China. Ono Pharmaceutical's $2.4 billion Deciphera acquisition has borne fruit. And more.
 
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.

How do certified community behavioral health clinics work?

A heartwarming roundup of cardiovascular news and most-loved stories

 

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.

Presented by Blue Matter, strategic consultants in the life sciences

 

Upcoming Fierce Events

29-1
Apr-May
Fierce Pharma Engage
San Diego, CA
7-9
May
Fierce Pharma Marketing Innovation Week
Free Virtual Event
14
May
Fierce Pharma Forward: Drug Development and Delivery Insights for 2025
Free Virtual Event
15-15
Aug-Nov
Fierce Life Sciences Innovation Awards
Submissions Open Early August
8-11
Sep
Fierce Pharma Week
Philadelphia, PA

View all events