End-to-end vector manufacturing optimizes batch success and delivers more doses at lower cost.

It’s 100 percent fatal, hits its victims in the prime of their lives and has companies with prospective treatments either shutting down or spinning their wheels with trial holds and failures. Huntington’s disease might just top the list of intractable neurodegenerative diseases.

PTC Therapeutics forged a financing deal valued at up to $1 billion with Blackstone that will allow it to deliver one new therapy every two or three years, the New Jersey-based biotech reported.

A Phase II trial studying PTC Therapeutics’ PTC518 in Huntington’s disease (HD) has been paused in the United States following a request from the FDA for additional data, the company announced Tuesday afternoon.

The nonprofit biotech Cure Rare Disease (CRD) is headed to the clinic with a CRISPR therapeutic aimed at halting the progression of Duchenne muscular dystrophy in single-patient dosing.

Sarepta Therapeutics announced Thursday that the U.S. Food and Drug Administration has put the Phase II trial of its Duchenne muscular dystrophy (DMD) candidate SRP-5051 (vesleteplirsen) on clinical hold following a serious safety signal.

For years, PTC Therapeutics has attempted to win regulatory approval for its Duchenne Muscular Dystrophy drug, Translarna (ataluren), but time and time again, the U.S. Food and Drug Administration has rejected the attempts. Now, the company believes it has data that will support another filing for possible approval.

Even with the COVID-19 emergency measures, payers still had an eye on the potential flood of gene therapies for rare diseases brewing in the pipeline, and were pondering new ways to pay for them.