Sarepta to buy Myonexus to expand muscular dystrophy therapy portfolio
Category: #health  By Mateen Dalal  Date: 2019-02-28
  • share
  • Twitter
  • Facebook
  • LinkedIn

Sarepta to buy Myonexus to expand muscular dystrophy therapy portfolio

Sarepta Therapeutics has recently confirmed that it is planning to acquire Myonexus Therapeutics in a $165 million deal, by exercising a purchase option that was part of a gene therapy partnership commenced by the firms in the previous year.

The transaction is aimed at broadening the pipeline of Sarepta with the five Myonexus candidates. Supposedly, these five candidates all use the same gene therapy platform that was licensed from Nationwide Children’s Hospital, designed for delivering treatment to skeletal and cardiac muscle robustly and systematically, without crossing the blood-brain barrier promiscuously.

According to Sarepta, all the five gene therapy candidates have been developed for offering the first-ever corrective therapy for five distinctive Limb-girdle muscular dystrophy (LGMD) forms, which are LGMD2B, LGMD2C, LGMD2D, LGMD2E, and LGMD2L.

The firm informed that three of the five programs are presently in clinical stages. MYO-101 from Myonexus, designed for LGMD2E, is the furthest in development and is aimed at restoring the dystrophin-related protein complex by transferring a gene which codes for and restores beta-sarcoglycan protein.

Myonexus, in November 2018, had unveiled the launch of a first-in-human, Phase I/IIa trial for MYO-101. It was a placebo-controlled study designed for evaluating a single infusion of MYO-101 intravenously among children aged between 4 and 15 years and having LGMD2E, with considerable symptoms of the disease.  

Apparently, the study was designed to assess up to two levels of dosage of the gene therapy candidate in two cohorts among nine patients, with their baseline measurements established on the basis of a recruitment study.

Doug Ingram, president and CEO of Sarepta, said acquiring Myonexus will enable the company to rapidly move and find solutions for LGMD patients, while continuing to develop and validate its gene therapy engine. The five gene therapies for LGMD under development are an ideal fit for the mission of Sarepta, to develop therapies having the potential to rescue patient lives from serious and life-limiting rare genetic disorders, Ingram added.

  • share
  • Twitter
  • Facebook
  • LinkedIn


About Author

Mateen Dalal    

Mateen Dalal

Mateen has completed his Bachelor’s degree in electronics and telecommunication engineering, post which he lent his proficiency to the industry, working as a quality and test engineer. Drawn intricately toward the field of content creation however, Mateen soon switc...

Read More

More News By Mateen Dalal

Air France-KLM & JAL make bids to purchase stake in Malaysia Airlines
Air France-KLM & JAL make bids to purchase stake in Malaysia Airlines
By Mateen Dalal

Air France–KLM S.A., one of the leading global air transport players, has reportedly made a proposal to invest in Malaysia Airlines and gain around 49 percent stake. Japan Airlines (JAL) is also looking to acquire a 25 percent stake in Mal...

Accenture opens innovation center in South Beach Road office
Accenture opens innovation center in South Beach Road office
By Mateen Dalal

Accenture, an Irish multinational professional services company, has recently opened a global innovation center within the Accenture’s office at South Beach Road, which is supported by EDB (Economic Development Board) in Singapore. It will pro...

Verrica unveils positive data from VP-102 study in molluscum treatment
Verrica unveils positive data from VP-102 study in molluscum treatment
By Mateen Dalal

Findings from the study are being produced at the 17th Annual Winter Clinical Dermatology Conference in Kohala Coast The data indicated a considerable increase in the percentage of patients with lesion clearance in VP-102 group Verrica Pharmaceu...