Rare Disease Report

Collaboration to Explore Small Molecule Treatments for Patients with Neurological Diseases

JULY 09, 2018
Kristi Rosa
When it comes to difficult-to-treat rare neurological diseases, such as Huntington’s disease, researchers are turning more and more towards the use of computational technologies as a way to correct harmful mutations occurring in RNA.

One such company, Skyhawk Therapeutics, Inc. has created a technology platform dedicated to exploring the development of small molecule treatment options for these patients. In an effort to bring these treatments to fruition, Skyhawk has announced a strategic partnership with Celgene Corporation affiliate.

Although there are no current treatments available to slow Huntington’s disease, researchers have been working to develop treatments targeting the disease’s root genetic cause—repetitive CAG expansions in the HTT gene, which is responsible for encoding huntingtin protein. Thus, developing therapeutics to target the HTT transcription and the translation of its mRNA has been a popular area of research.

“Targeting RNA with therapeutics is becoming an increasingly important approach in neurological research,” Richard Hargreaves, PhD, corporate vice president of Neuroscience and Imaging Research and Early Development for Celgene Corporation, said in a recent statement.

Skyhawk specifically has been working on targeting diseases that are driven by “exon skipping,” a kind of RNA mis-splicing, where important regions on the RNA are left out during the RNA splicing process, according to a recent news release. The company’s technology, however, “enables the rational design of small molecules that target specific binding pocket regions on RNA,” during the RNA splicing process. With this approach, the researchers work towards reversing the mis-splicing, and thus, treating the disease.

“This collaboration to discover and develop small molecule splicing modifiers extends our commitment to the neurodegenerative disease are consistent with our leadership and focus on protein homeostasis, and strategy to collaborate with innovators who are focused on disruptive approaches to treat disease,” Dr Hargreaves added.

With the closing of a new equity investment round, Skyhawk is “well-positioned” to continue their work on treating diseases via these small molecules designed to correct RNA expression, Bill Haney, co-founder and CEO of Skyhawk added. Investors from Alexandria Venture Investments and the Duke of Bedford are reportedly among the participants included in the round.

Through the five-year collaboration, Celgene is provided with the exclusive option to in-license worldwide intellectual property rights pertaining to candidates for up to 5 programs with potential utility not only in Huntington’s disease but also amyotrophic lateral sclerosis and other neurological disorders.

The partnership includes an upfront payment of $60 million, potential future license fees, as well as milestone payments and royalties.

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