Update on Myonexus’ LGMD gene therapies

Published Date
25/06/2018
Author
Jenny Sharpe
Category
Research
AAV virus

Myonexus Therapeutics gave an update on its LGMD gene therapy programmes at Sarepta’s recent Research and Development (R&D) day. The two companies formed a partnership in May.

Myonexus are currently developing gene therapies for five types of LGMD (see below). All of these share a similar design, where the desired gene is packaged into a type of adeno-associated virus (AAV). This shared design means that learnings from one study will help to inform another.

Myonexus plan to initiate a phase 1/2a trial at Nationwide Children’s Hospital, USA, testing MYO-101 in Q3 2018. This will be a randomised, placebo-controlled, double blind trial recruiting nine participants. The trial will also have a ‘crossover’ design, where all participants assigned to the placebo will get to have the gene therapy at some point.

Following positive results from a small safety trial completed in 2017, Myonexus are planning to test MYO-102 in a phase 1/2a trial at Nationwide Children’s Hospital, USA. This will be a randomised, placebo-controlled, double blind trial recruiting nine participants. The trial will also have a ‘crossover’ design, where all participants assigned to the placebo will get to have the gene therapy at some point.

Myonexus are currently testing MYO-103 in preclinical safety studies using mice. The company is expecting to meet with the US Food and Drug Administration (FDA) very soon to discuss its plans for MYO-103.

MYO-201 is currently being evaluated in a phase 1 trial at Nationwide Children’s Hospital, USA, where it was injected into the feet of six participants (intramuscular delivery). Myonexus reported that, so far, MYO-201 appears to be safe and can increase dysferlin protein levels. The company is planning a phase 1/2a trial testing intravenous delivery of MYO-201(an injection into the bloodstream, rather than the muscle).

Myonexus is currently testing MYO-301 in a mouse model of LGMD 2L. This will help to determine how well it works and whether it is safe to be tested in humans in future.

For more information, view Myonexus’ slides (no. 194 – 235)

If you have any further questions about the latest research into LGMD, please contact our Clinical Research Officer, Dr Sofia Nnorom, at research@musculardystrophyuk.org or call 020 7803 4813.

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