1. Scrip
  2. >>Advanced Therapies

Skyhawk Looks To Soar With Huntington’s Hope

 
• By Kevin Grogan

Oral RNA splicing modulator has gone into a Phase II/III trial

(Shutterstock)

Huntington’s disease (HD) has been a notoriously difficult field for drug development but enthusiasm is growing, not least at Skyhawk Therapeutics which has initiated a Phase II/III trial on a potentially transformative treatment for the devastating neurodegenerative condition.

Boston-based Skyhawk says that the first patient has been dosed in the FALCON-HD trial evaluating SKY-0515, an investigational oral RNA...

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 Advanced Therapies

Capricor Gets Complete Response For DMD Cell Therapy

 
• By Joseph Haas

Seven weeks before its action date, Capricor got an FDA complete response for cell therapy deramiocel in DMD-related cardiomyopathy. It has a Phase III study nearing readout, though.

Concentra Picks Up Cargo In Latest Troubled Biotech Buyout

 
• By Alaric DeArment

The Kevin Tang-led firm will pay $4.38 per share for Cargo, which laid off most of its workforce following the failure of a Phase II CAR-T trial.

Stock Watch: US CAR-T And Vaccine Actions: Net Negative For Access

 
• By Andy Smith

Relaxing CAR-T restrictions may not be an access panacea because of patient fragility and lack of payer incentive. Conversely, the ACIP’s recent recommendation on Thiomersal-containing flu vaccines is probably the thin edge of a more restrictive wedge.

Bluebird Builds Out Leadership Team Under Private Equity Ownership

 
• By Jessica Merrill

The gene therapy developer appointed a new president, chief medical officer and chief of staff following the closing of its acquisition by private equity firms Carlysle and SK Capital.

More from Rare Diseases

Skyhawk Looks To Soar With Huntington’s Hope

 
• By Kevin Grogan

Oral RNA splicing modulator has gone into a Phase II/III trial

Sarepta Market Dynamics For Elevidys Imperiled By Second Patient Death

 
• By Joseph Haas

With two deaths in non-ambulatory DMD patients, Sarepta is attempting damage control and will ask the US FDA to advise, setting up a possible confrontation with CBER director Prasad.

Early Blood Cancer Data Impresses As Incyte Plots Post-Jakafi Strategy

 
• By Kevin Grogan

First results for a first-in-class mutCALR-targeted therapy in essential thrombocythemia presented at EHA point to a lucrative future for the early-stage product.