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Voyager Expands Stable of AAV Vectors with Regenx Licensing Deal

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NEW YORK (GenomeWeb) – Voyager Therapeutics this week announced that it has licensed the non-exclusive rights to use Regenx Biosciences' proprietary adeno-associated viral vectors in three of its drug-development programs, including one focused on an expressed RNAi treatment for monogenic amyotrophic lateral sclerosis (ALS).

The arrangement also gives Voyager rights to use Regenx's vectors to develop therapies for Friedreich's ataxia and Huntington's disease (HD).

According to Jeff Goater, Voyager vice president of business development, the deal provides access to a suite of novel delivery vectors, including ones that have shown promise in crossing the blood-brain barrier, that complement his company's own AAVs.

"Internally, we have our own efforts around vector design and engineering," he told Gene Silencing News. While it is possible Voyager may choose to use an in-house vector with a particular drug candidate, "if one of the Regenx serotypes is optimal for what we're looking to do, this agreement gives us the flexibility … to move forward with that from a development perspective."

In exchange for the non-exclusive rights to its AAV vectors, including sublicensing rights, Regenx will receive an undisclosed upfront payment, ongoing fees, milestones, and royalties on the sale of products incorporating its technology. Regenx will also receive a portion of any revenues Voyager receives from sublicenses.

Voyager was launched earlier this year with $45 million in startup funding from investment firm Third Rock Ventures. The company already has a drug in the clinic — a Phase Ib Parkinson's disease therapy — but this drug, like the firm's preclinical Friedreich's ataxia treatment, is based on gene replacement.

However, the company is also exploring the other end of the therapeutic spectrum in its monogenic ALS program, aiming to use RNAi to knock down the gene SOD1, mutant forms of which are associated with inherited ALS.

Meanwhile, Voyager is also considering expanding its RNAi efforts to include HD. This disorder is caused by the expansion of a CAG repeat in exon 1 of the gene huntingtin, and numerous research groups have in recent years been testing expressed RNAi-based treatments for the condition including one at Genzyme and the lab of Voyager co-founder and University of Massachusetts Medical School (UMMS) investigator Phillip Zamore.

Goater cautioned, however, that while Voyager is interested in HD — as evidenced by the disease's inclusion in the Regenx deal — the company does not have a formal pipeline program for the indication at this point.

In support of these efforts, Voyager is developing its own suite of AAV delivery vectors, including ones stemming from the work of two other co-founders: Stanford University's Mark Kay and UMMS' Guangping Gao.

Notably, Gao earlier this year published details on several recombinant AAV (rAAV) serotypes that could reach the spinal cord and the forebrain following intrathecal injection. One of these vectors delivered an artificial microRNA targeting SOD1 in a mouse model of ALS, which slowed disease progression.

But in line with its goal of having "the best possible vector" for each of the disease areas it is pursuing, Goater noted, Voyager opted to also pick up access to Regenx's technology, including a series of novel AAV serotypes that have been shown to be quite promising when it comes to CNS delivery including AAV9 and AAVrh10.

These vectors, in fact, were the same that Gao described in his recent ALS paper. Meanwhile, in a paper published last year by a team out of the University of Florida, AAV9 was shown to effectively target neurons in a mouse model of Pompe disease. A few years prior, a separate University of Florida group published a review of data suggesting AAV9's ability to cross the blood-brain barrier.

Goater declined to comment on the specific routes of administration Voyager is considering for its ALS and other programs. Timelines for the company's preclinical programs also remain undisclosed.

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