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Beyond the Pitch

Nationwide Children’s Spinouts Focus on Promising Gene Therapy

Gene Therapy

The first successful gene therapy—modifying a person’s DNA to treat disease—occurred in 1990. In the nearly three decades since those pioneering treatments, there are thousands of gene therapy studies and clinical trials to treat disease from arthritis to cancer, blindness to sickle cell, from muscular dystrophy to neurodegenerative diseases.

The Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital here in Columbus is an international leader in the field. Recently, two clinical-stage gene startups that are spinouts of Nationwide Children’s were acquired, and a third received funding with an option for acquisition.

AveXis (with gene therapy in development for the treatment of spinal muscular atrophy – SMA) as acquired by Novartis for $8.7 billion, and Celenex (with potential first-to-market curative therapies for Batten disease) by Amicus Therapeutics for $452 million in cash and milestones. Additionally, Myonexus Therapeutics has entered into a relationship with Sarepta Therapeutics that produced $60 million in funding with an acquisition option.

Milo Biotechnology Targets Multiple Diseases with the Same Gene Therapy.

“The pendulum has swung toward gene therapy, but it’s thanks to Nationwide Children’s that we are where we are,” said Al Hawkins, founder and CEO of Milo Biotechnology. The clinical-stage company is developing therapies to strengthen muscle and prevent muscle atrophy.

“The early stage development engine at Nationwide Children’s, backed by their resources and experience, from clinical to manufacturing to regulatory have allowed our small, virtual company to further than we otherwise could,” Hawkins said.

Milo is currently engaged in the three clinical studies for rare muscular dystrophies—Duchenne, sporadic inclusion body myositis, and Becker muscular dystrophy. In this regard, Milo is a different kind of gene therapy company.

“Most gene therapies are targeting disease from a single genetic defect,” said Hawkins. “Instead, we are upregulating [in scientific parlance, that means increasing] a protein called follistatin, which makes muscles stronger and function better with applicability to a range of diseases instead of a single disease. Think of it as gene therapy 2.0.”

Hawkins sees the therapeutic opportunity for Milo across the approximately 10 different muscular dystrophies. “Or think about age-related muscle wasting,” he said, “or when astronauts go into space, hip replacements, or cancer patients have treatment and it weakens muscles. We have potential applicability to a myriad of disease.

The FDA has taken steps to speed up the approval of gene therapies for certain specialty and rare diseases.

“The FDA has been outstanding to work with in this field,” Hawkins said. “They recognize the risk profile for these patients, given the absence of other treatments.”

Milo has completed Phase I and II clinical trials, demonstrating efficacy results in patients without serious drug-related danger. The next steps, Hawkins said, are investments in even larger Stage II/III trials and manufacturing processes.

The Wall Street Journal features: Gormley’s Take: Biotech Pairing of Venture Fund, Children’s Hospital Bears Fruit.

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