StrideBio, Takeda Pair to Develop New Gene Therapies for Friedreich’s Ataxia

Takeda and StrideBio have established a collaboration to develop new, more effective adeno-associated viruses (AAV) to be used as vehicles for in vivo delivery of gene therapies for Friedreich’s ataxia (FA) and other diseases.

“StrideBio’s expertise and unique gene therapy technology holds great potential for significantly advancing the field of neurological disease research,” Emiliangelo Ratti, head of the Neuroscience Therapeutic Area Unit at Takeda, said in a press release.

“Our collaboration is a natural extension of Takeda’s neuroscience research and development strategy, including modality diversification, identifying targets with a high degree of association with disease, and a focus on developing innovative medicines for neurologic diseases that have a high unmet medical need,” Ratti said.

FA is caused by mutations in the FXN gene, which codes for the frataxin protein. This protein is found in several cells, but has higher levels of expression in the heart, spinal cord, liver, pancreas, and the skeletal muscles that control movement.

In people with FA, the nervous system is damaged, leading to impaired muscle coordination — or ataxia — that worsens over time.

Because FA is caused by mutations of a single gene, scientists are testing the potential of gene therapy for the disease.

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Gene therapy involves introducing new genetic material into specific cells to correct an existing genetic abnormality. For that, scientists can use different vehicles, including AAVs.

AAvs, the most-used vectors in gene therapy, are modified, noninfectious types of viral vectors designed to target and deliver DNA to specific cells.

However, AAVs efficacy to cross the blood-brain barrier — a highly selective membrane that shields the central nervous system (brain and spinal cord) with its cerebrospinal fluid from the general blood circulation — requires high doses of the viral vector. This makes it costly and prone to induce liver toxicity. Moreover, the gene therapy using AAVs may trigger the immune system’s reaction to develop antibodies that neutralize the therapy’s efficacy.

StrideBio has developed a new platform, called STRucture Inspired Design, to create new AAV capsids (the protein shell of a virus) that escape neutralizing antibodies and have an enhanced affinity for specific tissues, including the central nervous system.

“We are very excited to partner with Takeda given their expertise and commitment to developing treatments for patients with neurological diseases,” said Sapan Shah, PhD, chief executive officer at StrideBio. “We look forward to working together to bring transformative and novel AAV-based gene therapies to patients, while continuing to validate and expand StrideBio’s platform, manufacturing capabilities and pipeline.”