Pfizer and Voyager Therapeutics Enter $300 Million Collaboration to Advance Next-Generation AAV Gene Therapies

Oct 04, 2022 10:21 CST Updated Oct 06, 08:59

Voyager Therapeutics

Gene Therapy Developer

Pfizer

Pharmaceutical R&D Developer

Over the past two decades, humanity hasGene TherapyA series of milestone breakthroughs have been achieved in the field. Gene therapy has also gradually become one of the hottest treatment fields, especiallyAdeno-Associated Virus（AAV）It has become the preferred delivery vector for gene therapy and is also widely regarded as the most promising delivery vector to date.

December 10, 2017, U.S. Food and Drug AdministrationManagementBureau（FDA）ApprovedSpark TherapeuticsThe company's gene therapy Luxturna was launched to treat congenital blindness caused by mutations in the RPE65 gene. This is also the first AAV gene therapy to receive FDA approval, ushering in a new era of in vivo gene therapy. Subsequently, Spark was acquired.Roche（Roche）For $4.3 billion.

Novartis（Novartis）Also values AAV gene therapy, whose development treatsSpinal Muscular Atrophy（SMA）The AAV gene therapy Zolgensma was approved for marketing by the FDA on May 24, 2019, with a price as high as$2.125 million, once became the most expensive drug in the world.

October 4, 2022Pfizer（Pfizer）WithVoyager TherapeuticsAnnounced a total of $300 million in cooperation, further validationTRACERThe Potential of the Capsid Development Platform. Pfizer will provide Voyager with a $10 million upfront payment, as well as $290 million in milestone payments and royalties.

It is reported that this collaboration will advance Voyager Therapeutics, Inc.'sGBA1 Type Parkinson's Disease、SOD1 Type ALSAnd other R&D pipelines for nervous system diseases, as well as some gene therapy projects from Pfizer, Inc.

Although AAV is considered the safest gene therapy vector and the FDA has approved a few AAV therapies for marketing, AAV therapies still face safety issues, with toxic effects at high doses causing the death of several patients in clinical trials. Therefore, there is an urgent need to develop safer and more specifically targeted AAV capsids.

Voyager TherapeuticsThe company's proprietary RNA-driven TRACER screening platform generates novel AAV capsids with higher specificity, lower dosage requirements, and reduced off-target risks compared to traditional AAV serotypes. For exampleNovel AAV Capsid for Breaking Through the Blood-Brain Barrier and Targeting the Central Nervous SystemExperiments in non-human primates have shown that after intravenous injection, it can be widely expressed in the brain, with an efficiency 1000-10000 times that of AAV9. In addition, the TRACER screening platform can also develop AAV capsids with enhanced targeting to myocardium, skeletal muscle, eyes, liver, and other tissues.

Previously, in March 2022,Novartis（Novartis）AndVoyager TherapeuticsAnnounced the achievement of a total amount as high as$1.75 billionUnder the collaboration, Novartis will provide Voyager with a $54 million upfront payment, along with up to $1.7 billion in milestone payments and royalties. Novartis will gain access to Voyager's proprietary RNA-driven...TRACERCapsid development platform and the development rights of specific AAV capsids. Through this collaboration, Novartis hopes to develop novel AAV vectors capable of crossing the blood-brain barrier and targeting the central nervous system to treat diseases deep within the brain.

October 2021,Pfizer（Pfizer）AndVoyagerReached a total amount of$630 millionThe AAV gene therapy collaboration for the development of the nervous system and heartBlood vesselTwo items of the disease.

VoyagerCurrently, there are 5 self-developed pipelines, all in the preclinical stage, respectively.Alzheimer's Disease、GBA1-Type Parkinson's Disease、SOD1 Type ALS、HER2-positive brain metastaticBreast Cancer、Huntington's DiseaseThe method of delivering therapeutic antibodies is used to treat Alzheimer's disease and metastatic breast cancer, gene replacement therapy is used to treat Parkinson's disease, and gene silencing therapy is used to treat ALS and Huntington's disease.