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Ovid partners with gene therapy startup, Gensaic to restock drug pipeline

Ovid Therapeutics has struck a deal with young biotechnology company Gensaic, hoping the startup’s method of delivering genetic medicines can yield new treatments for rare brain diseases.

Key highlights:

Expanding the pipeline

Under the deal, the partners will develop up to three gene-based treatments for neurological conditions Ovid is targeting. So long as there is an agreement of terms, Gensaic will claim rights to license any gene therapies that emerge from the deal. Ovid also invested $5 million in the startup and committed to participate in future financing rounds.

The deal is the latest step in a rebuilding plan for Ovid, ex-Teva Pharmaceutical and Bristol Myers Squibb executive Jeremy Levin formed seven years ago.

Levin’s plan in starting Ovid was to grab medicines overlooked elsewhere, license them and develop them for rare brain diseases. That strategy led Ovid developing two medicines, one for Angelman’s syndrome and another for rare forms of epilepsy. Both of which helped the biotech to go public in 2017.

Ovid hasn’t been successful, however. The Angelman’s drug failed a Phase 3 trial in 2020, erasing more than half of the company’s value. One year later, Ovid, aiming to bolster its dwindling cash reserves, sold rights to the epilepsy drug back to Takeda. Though Ovid can still receive milestone payments and royalties from the drug, which is now in late-stage testing, its only remaining in-house programs are in preclinical testing. At just over $2 apiece, shares trade near all-time lows.

Recently, Ovid has taken steps to restock its pipeline. One experimental medicine for treatment-resistant epileptic seizures could start human trials later this year, while a licensing deal with AstraZeneca and a related partnership with Tufts University could yield other drug candidates that might follow in 2024.

The alliance with Gensaic provides Ovid with three more prospects, whilst pushing Ovid into the gene therapy field.

New field: Gene therapy

Gensaic was seeded in 2021 as M13 Therapeutics and is currently housed in Cambridge, Massachusetts biotech startup incubator LabCentral. Over the past two years, the company has won awards in multiple startup competitions for its research into a method of gene therapy delivery designed to overcome the limitations of standard approaches.

Many gene therapies rely on modified viruses to send genetic instructions into the body’s cells. Those delivery vehicles are used in multiple products approved for rare inherited diseases, but they also come with weaknesses, too. For example, the adeno-associated virus (AAV) can only carry a relatively small amount of genetic cargo and is sometimes shut down by the body. Another, the lentivirus, also has limited carrying capacity and has been linked in rare cases to the development of cancers.

Gensaic instead aims to use tiny particles derived from “phages,” the viruses that infect bacteria, to deliver genetic material. Gensaic claims these particles can be engineered to target multiple tissue types — among them the lung and brain — and can carry much larger genes. Gensaic believes they may have the potential to be administered more than once, too, though that hasn’t yet been proven.

In a statement, Levin said the approach “appears to be optimal” for carrying “substantial genetic cargo” across the blood-brain barrier, a filtering mechanism the body uses to keep foreign substances out of the brain.

“We believe it may hold the potential to treat a broad continuum of diseases in the brain,” Levin said.

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