PARIS and BOSTON, April 22, 2024 – In a significant development in the biopharmaceutical industry, Ipsen and Skyhawk Therapeutics have entered into an exclusive global agreement to advance the discovery and development of novel small molecules that target RNA. These molecules are aimed at treating rare neurological diseases.

The partnership grants Ipsen exclusive worldwide rights to develop and commercialize promising candidates following their successful validation. This collaboration utilizes Skyhawk’s innovative platform that combines computational biology, artificial intelligence, and machine learning to rapidly generate small molecules that can potentially correct RNA mis-splicing—an issue that leads to certain neurological disorders.

Skyhawk is well-recognized for its drug discovery capabilities, particularly its ability to design oral medications that can penetrate the central nervous system. Their current leading candidate, SKY-0515, targets Huntington’s disease and is already in early-stage clinical testing.

Under the terms of the agreement, Ipsen will take over the development and commercialization responsibilities of the validated candidates. Skyhawk stands to receive up to $1.8 billion in payments, which include development, regulatory, and commercial milestones, as well as an upfront payment and potential tiered royalties.

This deal marks Ipsen’s ongoing commitment to expanding its therapeutic portfolio, which includes recent acquisitions and licensing in oncology and rare diseases. Steve Glyman, Head of Neuroscience R&D at Ipsen, expressed enthusiasm about the new partnership, highlighting its potential to address unmet needs in movement disorders and other neurological conditions.

Skyhawk Therapeutics’ Chief Scientific Officer, Sergey Paushkin, also emphasized the strategic nature of the partnership, aiming to deliver transformative treatments for patients with rare neurological diseases that currently lack approved therapies.

This collaboration not only underscores Ipsen’s strategic expansion into novel therapeutic areas but also enhances its capabilities in neuroscience, building on its existing expertise and recent expansions.

About PackGene

PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.

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