Sarepta Snaps Up Gene Therapy Approaches to DMD

Sarepta may have launched its first therapy for Duchenne muscular dystrophy in the US, but it is facing potential competition from a plethora of late-stage DMD therapies; it is backing research that could lead to new gene therapies able to treat most affected patients, rather than a subgroup of individuals with a particular gene mutation.

Sarepta Therapeutics Inc. has licensed rights to two gene therapy research programs underway at the Nationwide Children’s Hospital, Columbus, Ohio, adding new projects to its growing Duchenne muscular dystrophy pipeline to complement its recently launched lead DMD product Exondys 51 (eteplirsen).

Marketing a range of therapies for a specific disease or condition is a well-established strategy for pharmaceutical companies, particularly in...

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on Scrip for daily insights

  • Start your 7-day free trial
  • Explore trusted news, analysis, and insights
  • Access comprehensive global coverage
  • Enjoy instant access – no credit card required

More from Business

Activist Investor Vs. Japan Pharma: ASKA Plans Poison Pill As Dalton Pushes For MBO

 
• By 

While ASKA plans to dilute the holding of largest investor Dalton by issuing new shares, the US investment firm continues to push a management buyout option to the mid-sized Japanese pharma company.

Finance Watch: Karyopharm Cuts Jobs In Cash Crunch; Pacira’s Layoffs Attributed To Efficiency

 
• By 

Restructuring Edition: Karyopharm is reducing its workforce by 20% as it seeks alternatives to extend its cash runway, but Pacira’s production improvements created efficiency that necessitate an 8% layoff in manufacturing. Jasper, Century, Sage, Leap and Prothena also cut jobs.

Kailera Eyes Global Obesity Market With Hengrui’s Phase III Dual Agonist

 
• By 

With China’s Hengrui reporting successful Phase III data for its dual GLP-1/GIP agonist, licensee Kailera is readying plans to demonstrate better efficacy than current obesity drugs.

China’s Mid-Cap Companies Thriving On Big Pharma Deals

 

Big pharma is increasingly looking east to restock its pipelines, a process that is helping to forge a new generation of mid-sized R&D-based companies in China and is reflected in their stock price performance.

More from Scrip

China’s Mid-Cap Companies Thriving On Big Pharma Deals

 

Big pharma is increasingly looking east to restock its pipelines, a process that is helping to forge a new generation of mid-sized R&D-based companies in China and is reflected in their stock price performance.

In Brief: Nanoscope Ploughs Forward With FDA Application For Retinitis Pigmentosa Gene Therapy

 
• By 

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.

Stock Watch: Pharma Financial Forecasts Cannot Withstand Headwinds Forever

 
• By 

Pharma company financial guidance emerged largely unscathed from a first quarter punctured by a weakening US dollar and threats of import tariffs on drugs. With other pressures either already evident or on the horizon, guidance revisions may be unavoidable.