Sarepta isn’t allowing an ongoing issue with its lead gene therapy candidate to dampen its enthusiasm for the field. It just announced a tie-up with Lacerta will add another three programs to the eight already on its books and extends its focus beyond muscular dystrophy.

The $30 million equity investment gives Sarepta exclusive rights to central nervous system-targeted gene therapy for Pompe disease, plus two unnamed programs in other rare CNS disease. University of Florida spinout Lacerta will carry out preclinical work on the therapies before passing the baton to Sarepta once clinical testing starts.

Sarepta said it has also committed to pay Lacerta development and sales-based milestones as well as single-digit royalties on net sales under the terms of the deal, which also gives it access to "world-class talent" as well as an additional dosing and delivery platform, capsid screening library, and proprietary manufacturing technology.

"We’re building a significant gene therapy division and we need tools," said CEO Doug Ingram on the company’s second-quarter results. The latest deal "moves us forward on our mission is to deliver life-enhancing therapies to those living with underserved diseases and in so doing to become one of the most meaningful global genetic medicine companies in the coming few years," he added.

The tie-up with Lacerta follows an alliance with Myonexus for five gene therapy programs focusing on limb girdle muscular dystrophy (LGMD), plus earlier collaborations with Genethon and Nationwide Children’s Hospital signed last year. At a stroke makes gene therapy Sarepta’s biggest R&D category in terms of the number of projects in play, overtaking its RNA-targeted portfolio.

Last month, Sarepta’s lead Duchenne muscular dystrophy (DMD) gene therapy was placed under a clinical hold as a result of a quality issue that the company thinks won’t hold up the clinical development program for long and—ironically—might even allow it to accelerate its pivotal trial plans.

Ingram said on the call that the biotech intends to respond to the FDA’s concerns by the end of the month and thinks it should be lifted "in advance of our meeting with the agency to align on our clinical pathway."

Last month, shares in the company rocketed on the strength of data from three patients in a phase 1/2 study which showed an increase in microdystrophin to a level that might stop, or even reverse, DMD progression.

Dosing is due to start this month in patients with beta-sarcoglycanopathy, the lead LGMD indication, in the coming weeks. Sarepta and Myonexus have four other LGMD diseases in their sights and said they will reveal more about those plans later this year.

"Between our gene therapy programs for both DMD and limb-girdle as well as the in-licensed assets from Lacerta, our team is preparing for the potential launch of multiple gene therapy products over the coming years," commented Bo Cumbo, Sarepta’s chief commercial officer.

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