Summit Therapeutics PLC to receive US$22mln milestone payment as final patient enrols for DMD trial

Summit Therapeutics PLC to receive US$22mln milestone payment as final patient enrols for DMD trial: "Summit Therapeutics PLC (NASDAQ:SMMT LON:SUMM) will receive a US$22mln milestone payment from Sarepta Therapeutics (NASDAQ:SRPT) after the final patient was enrolled in a dosing trial of utrophin modulator, ezutromid.

Summit hopes the trial will identify utrophin modulation as a potential disease-modifying treatment for all patients suffering Duchenne Muscular Dystrophy (DMD),  a progressive and fatal muscle wasting disease that affects 50,000 boys and young men globally."



'via Blog this'

Summit Therapeutics PLC to receive US$22mln milestone payment as final patient enrols for DMD trial

Summit Therapeutics PLC to receive US$22mln milestone payment as final patient enrols for DMD trial: "Summit Therapeutics PLC (NASDAQ:SMMT LON:SUMM) will receive a US$22mln milestone payment from Sarepta Therapeutics (NASDAQ:SRPT) after the final patient was enrolled in a dosing trial of utrophin modulator, ezutromid.

Summit hopes the trial will identify utrophin modulation as a potential disease-modifying treatment for all patients suffering Duchenne Muscular Dystrophy (DMD),  a progressive and fatal muscle wasting disease that affects 50,000 boys and young men globally."



'via Blog this'

Preclinical data reinforces potential efficacy, durability of investigational gene therapy for DMD

Preclinical data reinforces potential efficacy, durability of investigational gene therapy for DMD: "Solid Biosciences announced today that new data from two preclinical studies reinforce the potential of its investigational microdystrophin gene therapy, SGT-001, to be an effective treatment approach for Duchenne muscular dystrophy (DMD). The preclinical data, which were presented at the American Society of Gene and Cell Therapy (ASGCT) 20th Annual Meeting, demonstrated that a single administration of SGT-001 resulted in sustained and significant microdystrophin expression and improvements in muscle function, with no observed immune response. The Company plans to initiate clinical trials for SGT-001 in the second half of 2017."



'via Blog this'