Gene Therapy

Solid Bio is back in business after the FDA lifts clinical hold on Duchenne MD gene therapy

Three months after the FDA dropped a clinical hold on Solid Biosciences $SLDB over safety concerns related to their gene therapy for Duchenne muscular dystrophy, the biotech is back in business. Execs said Monday morning that the hold is gone and the FDA has signed off on their response.

Solid Bio’s stock shot up 18% in early trading Monday.

Solid Bio CEO Ilan Ganot

The very first patient to be treated with SGT-001 experienced a serious adverse event, triggering alarm bells after investigators saw a “decrease in platelet count followed by a reduction in red blood cell count, transient renal impairment and evidence of complement activation.” 

In response, investigators changed the study, adding IV glucocorticoids after treatment, amping up monitoring activity and providing eculizumab on an as-needed basis to fight any complement activation that may occur in future.

The full hold at Solid came just weeks after CEO Ilan Ganot put out an 11th-hour alert right before its IPO saying that the FDA had put a partial hold on the program.

Solid has some major league rivals in this field. Sarepta is prepping data from its program and Pfizer is also engaged. Those programs have not been tarnished by safety issues up to this point.

The FDA has been on high alert on Solid since James Wilson — a pioneer in gene therapy who developed the AAV delivery tech that Solid is using — resigned from the advisory board after an animal study raised a red flag about potential safety issues when used at a high dose. Wilson was alarmed after a monkey was killed by liver toxicity after being exposed to a high dose AAV treatment, with evidence of motor neuron damage in piglets. A number of companies use AAV tech in-licensed for their work. But none of the others have been hit with a hold.

Solid CEO Ilan Ganot — a former JP Morgan investment banker — has made much of the fact that he’s a Duchenne MD dad out to find a gene therapy that could cure the lethal, rare disease. By introducing a synthetic dystrophin transgene construct, called microdystrophin, via a viral vector, the company hopes to prove it can do what Sarepta and others have been groping for with one decisive intervention. And he had attracted some heavyweight backers, including RA Capital and their colleagues at Bain.

Assurances from the company that they were getting back on track has helped assuage investors, pushing the stock back up after a damaging meltdown.

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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