We are excited to announce that Protective Antigen has been selected as Clovertex’s Target of the...
For July 2023, we focus our attention on the Adeno-Associated Virus serotype 9 (AAV9) as the 'Clovertex Drug Target of the Month'. AAV9 is a potent vector used for delivering gene therapies to treat a myriad of diseases, demonstrating a remarkable ability to cross the blood-brain barrier and to transduce cells in the central nervous system.
AAVs have taken center stage this month, with a surge of deals in the pharma industry revolving around AAV-based therapies. Key amongst these deals is Sangamo Therapeutics' agreement with Prevail Therapeutics, a subsidiary of Eli Lilly and Company. Through this agreement, Prevail has been granted rights to evaluate certain proprietary AAV capsids developed by Sangamo. Generated through Sangamo’s AAV capsid engineering platform, SIFTER, these capsids have shown potential for high-efficiency delivery of investigatory gene therapy constructs to the central nervous system in pre-clinical animal models, opening up new treatment options for a range of previously inaccessible areas.
A second significant development is Alexion's (a subsidiary of AstraZeneca) agreement with Pfizer to acquire a portfolio of preclinical rare disease gene therapies. This agreement includes novel AAV capsids, offering synergistic opportunities to advance next-generation genomic medicines across AstraZeneca and further bolstering Alexion's ambition to transform patient outcomes with genomic medicine.
Lastly, Neurogene, a New York gene therapy startup, is anticipated to raise approximately $200 million from a Nasdaq swap with Neoleukin Therapeutics. Neurogene is known for its AAV9-based one-time gene therapies, with NGN-101 currently being tested in children with CLN5 Batten disease in a Phase I/II clinical trial. This deal will provide Neurogene with the necessary resources to further its AAV-based gene therapy work.
These examples underline the significance of AAVs as a promising therapeutic delivery system and its potential to transform patient outcomes for CNS diseases, thus making it the worthy 'Clovertex Drug Target of the Month'. The protein model rendering features the cryo-EM structure of AAV9 (PDB 7MT0).
About Clovertex
Clovertex is an industry leader specializing in meeting the scientific IT needs of pharmaceutical companies, with a robust focus on cloud-based or on-premises cryo-EM processing. As an Advanced Tier Services Partner with Amazon Web Services (AWS), Clovertex leverages its comprehensive expertise to architect, build, automate, and manage software applications designed to meet specific research needs. Our all-encompassing solutions are meticulously tailored to streamline the complex processes associated with pharmaceutical research, underscoring our commitment to innovation, efficiency, and customer satisfaction. By integrating state-of-the-art technology with deep scientific understanding, Clovertex paves the way in optimizing the intersection of information technology and pharmaceutical development.
Useful links
3. Neurogene and Neoleukin Announce Definitive Merger Agreement
4. Clovertex website
