Biogen, Capsigen Partner on Novel AAV Capsid Development for Neurologic Disorders


The partnership will allow for the development of disease-specific AAV vectors that will serve Biogen's current gene therapy pipeline.

Biogen and Capsigen are partnering to develop novel adeno-associated viral (AAV) capsids to deliver gene therapies for the treatment of various central nervous system (CNS) and neuromuscular disorders.1

The partnership aims to combine Capsigen’s experience in capsid engineering with Biogen’s therapeutic development capabilities to accelerate delivery of gene therapies via novel capsids designed to meet highly customized, disease-specific transduction profiles.

“Through this collaboration, we aim to solve key technological challenges in the delivery of gene therapies to target tissues. One of our priorities for technology innovation is the discovery of AAV capsids with improved delivery profiles,” said Alfred Sandrock Jr, MD, PhD, head of research and development, Biogen, in a statement. “We are investing for the long-term by building platform capabilities and advanced manufacturing technologies with the goal of accelerating our efforts in gene therapy.”

Capsigen brings to the partnership its proprietary TRADE™ platform and associated vector engineering technologies to produce dose optimized, fit-for-purpose vectors to use with Biogen’s gene therapy pipeline. As part of the collaboration, Capsigen will receive a $15 million upfront payment and is eligible to receive up to $42 million in potential research milestone payments as well as up to an additional $1.25 billion in development and commercial milestone payments. The company will also receive royalties in future sales that use its capsid technology.

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“At Capsigen, we believe the next revolution in gene therapy will be driven by engineered AAV capsids designed to meet disease-specific transduction profiles,” said John Bial, chief executive officer, Capsigen, in a statement. “Biogen is a leader in neuroscience, and we are excited for the opportunity to work with them to potentially bring new treatments to patients. This collaboration is consistent with our strategy to work with world-class companies to develop the next generation of gene therapies.”

In late 2020, Sio Gene Therapies (previously Axovant) entered into a similar partnership with Viralgen to support AAV-based vector manufacturing of material for their phase 1/2 trial (NCT04669535) assessing an investigational agent, AXO-AAV-GM2, in the treatment of GM2 gangliosidosis (GM2-G) also known as Tay-Sachs disease or Sandhoff disease. The first infant patient with GM2-G was dosed in the trial in March 2021. The agent was previously granted an orphan drug and a rare pediatric disease designation by the FDA and is the first GM2-G investigational gene therapy to enter clinical trials.2

At the time, Javiér Garcia, chief executive officer of Viralgen, said that the company was “thrilled to partner with [Sio Gene Therapies]” and that it was excited to offer “support and priority access to our platform as they advance their AAV gene therapies for the rare fatal pediatric diseases GM1 and GM2 gangliosidosis.” He also lauded the company’s production platform as a complement to the development efforts of Sio Gene Therapies on the trials and commercialization fronts.

These efforts likely speak to the rapid expansion of AAV-mediated therapies for neurologic and CNS disorders, with a number of companies in recent weeks announcing positive news for their respective agents. Among them, are Voyager Therapeutics, which in late April announced the lifting of an FDA-driven clinical hold on its investigational new drug (IND) application for VY-HTT01, an AAV1-based gene therapy for patients with Huntington disease. The hold was placed on the application in October 2020, due to chemistry, manufacturing, and controls concerns from the FDA.3 Just a few weeks prior to that, the agency granted a fast-track designation to LEXEO Therapeutics for its AAV-mediated agent LX1001 as a potential treatment for adult patients with APOE4-associated Alzheimer disease.4

Additionally, that news came on the heels of the announcement from REGENXBIO that its AAV9-based single intracisternal injection agent, RGX-121, had been administered to the first patient in a phase 1/2 study for patients up to 5 years old with severe mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome.5

1. Biogen and Capsigen announce collaboration to discover and develop novel AAV capsids for targeted CNS and neuromuscular disorders. News release. May 10, 2021. Accessed May 11, 2021.
2. Axovant and Viralgen Sign Strategic Gene Therapy Development and Manufacturing Partnership. News release. September 15, 2020. Accessed May 11, 2021.
3. Voyager Therapeutics Receives FDA Clearance Of IND Application For Gene Therapy Candidate VY-HTT01 For Treatment Of Huntington’s Disease. News release. April 26, 2021. Accessed May 11, 2021.
4. LEXEO Therapeutics Announces FDA Fast Track Designation Granted to LX1001 for the Treatment of APOE4 Associated Alzheimer’s Disease. News release. April 20, 2021. Accessed May 11, 2021.
5. REGENXBIO Announces Dosing of First Patient in Cohort 3 of Phase I/II Trial of RGX-121 for the Treatment of MPS II (Hunter Syndrome). News release. April 14, 2021. Accessed May 11, 2021.
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