GSK Takes Rare Diseases Option from Prosensa

 
• By Melanie Senior

GlaxoSmithKline's tie-up with Dutch biotech Prosensa around four RNA-modulating therapeutics for Duchenne Muscular Dystrophy, a rare genetic disorder, provides recent evidence that even the largest Big Pharma are embracing the most niche of specialist disease indications.

There's no denying that even the largest Big Pharmas are embracing the most niche of specialist disease indications. GlaxoSmithKline PLC's October 13 tie-up with Dutch biotech Prosensa Holding BV around four RNA-modulating therapeutics for Duchenne muscular dystrophy (DMD), a rare genetic disorder that affects one in 3,500 newborn boys, provides recent evidence. [See Deal]

The two-part deal sees GSK paying $25 million (£16 million) up front for a worldwide license to Prosensa's lead Duchenne...

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
Start Free Trial

Already a subscriber?

More from Strategy

Shigella Vaccine Pipeline And Why It Matters

 
• By Vibha Ravi

A GSK-Bharat Biotech deal on a shigella vaccine candidate has cast a spotlight on the disease which is the second leading cause of diarrheal deaths in children below five. Scrip looks at data from Pharmaprojects, WHO and other sources to highlight why a vaccine matters and what pharma is doing

Asia Deal Watch: SunRock, Escugen Collaborate On CCR9-Targeted ADC

 
• By Joseph Haas, Lisa Takagi, Sushmita Panda, and Xu Hu

Axcelead/Lotte Biologics/Kanaph and NextCure/Simcere also unveil ADC-focused pacts, plus deals involving Avata/Oceanus, GSK/Bharat Biotech, Cullinan/Genrix and more.

BIO Notebook: Woodcock Calls For Doing The Right Thing, Dealmaking Remains Constrained

 
• By Mandy Jackson, Joseph Haas, and Sarah Karlin-Smith

Highlights from Day Four of the BIO International Convention include Woodcock offering practical advice on rare disease trials, the sorry state of dealmaking mid-year, Novartis discussing its approach to partnering, and Generate looking for funding to move into Phase III.

ASCO: Pushing The Frontiers Of ADCs

 
• By Mary Jo Laffler

Antibody-drug conjugates are a major part of the oncology pipeline, and data presented at the American Society of Clinical Oncology meeting highlight the emergence of new technologies.

More from Business

China Signals Restarting IPOs For Unprofitable Biotechs

 
• By Dexter Jie Yan

The China Securities Regulatory Commission has proposed a new “growth tier” for the STAR Market of the Shanghai Stock Exchange, to reinstate the market’s listing standards for unprofitable firms.

BIO Notebook: Woodcock Calls For Doing The Right Thing, Dealmaking Remains Constrained

 
• By Mandy Jackson, Joseph Haas, and Sarah Karlin-Smith

Highlights from Day Four of the BIO International Convention include Woodcock offering practical advice on rare disease trials, the sorry state of dealmaking mid-year, Novartis discussing its approach to partnering, and Generate looking for funding to move into Phase III.

Syncona Looks To Go Private As Market Decline Deepens

 
• By Andrew McConaghie

While some are calling for Syncona to be wound up, the UK-based company believes many existing and new investors will back the creation of new private fund.