Pfizer has transferred an AAV capsid, which targets a rare neurological disease, to Alexion, AstraZeneca’s rare disease division, as part of a broader deal announced in July. The deal between Pfizer and Alexion, valued at up to $1 billion plus potential royalties, involves the acquisition of an early rare disease gene therapy portfolio. Voyager Therapeutics was originally in a partnership with Pfizer, dating back over two years, where they received $30 million upfront in a licensing deal for two capsids. Voyager could also gain $10 million per option and up to $500 million in biobucks. One of the option payments was paid by Pfizer in October 2022, and the remaining gene therapy asset is now being transferred to Alexion. Voyager CEO Al Sandrock expressed enthusiasm about partnering with Alexion due to their commitment to advancing genomic medicines.

Voyager has been building its pipeline through partnerships, with Novartis opting into two of its gene therapies in March, resulting in a $25 million payment. Later, Sangamo Therapeutics entered into an agreement to utilize Voyager’s licensing technology to develop a prion disease candidate, aiming for a new drug application in 2025. Voyager also has an extensive collaboration with Neurocrine, encompassing two preclinical assets in the late research stage, targeting Parkinson’s disease and Friedrich’s ataxia, respectively.

While Voyager’s owned programs have yet to enter clinical trials, they are making progress. The company anticipates requesting U.S. regulatory approval for a phase 1 study of an anti-tau Alzheimer’s disease treatment in the first half of 2024. Additionally, Voyager plans to select a development candidate for its SOD1 amyotrophic lateral sclerosis (ALS) program by the end of this year, with a new drug submission expected in mid-2025. This ALS program is targeting the same patient subset as Biogen and Ionis Pharmaceuticals’ Qalsody, which received accelerated approval in April. Voyager Therapeutics continues to advance its gene therapy pipeline through strategic partnerships while aiming to bring its promising therapies into clinical trials in the near future.

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PackGene is a CRO & CDMO technology company that specializes in packaging recombinant adeno-associated virus (rAAV) vectors. Since its establishment in 2014, PackGene has been a leader in the AAV vector CRO service field, providing tens of thousands of custom batches of AAV samples to customers in over 20 countries. PackGene offers a one-stop CMC solution for the early development, pre-clinical development, clinical trials, and drug approval of rAAV vector drugs for cell and gene therapy (CGT) companies that is fast, cost-effective, high-quality, and scalable. Additionally, the company provides compliant services for the GMP-scale production of AAVs and plasmids for pharmaceutical companies, utilizing five technology platforms, including the π-Alpha 293 cell AAV high-yield platform and the π-Omega plasmid high-yield platform. PackGene's mission is to make gene therapy affordable and accelerate the launch of innovative gene drugs. The company aims to simplify the challenging aspects of gene therapy development and industrialization processes and provide stable, efficient, and economical rAAV Fast Services to accelerate gene and cell therapy development efforts from discovery phase to commercialization.

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