Courier | Over US$1 billion in cooperation with Roche, what are the characteristics of this cutting-edge gene therapy company?

▎WuXi AppTec Content Team Report

Roche continues to build on gene therapy in ophthalmology following the acquisition of gene therapy company Spark Therapeutics and its innovative therapy Luxturna. On July 19, Avista Therapeutics, a biotechnology company aiming to develop innovative gene therapies for rare eye diseases, announced that it has entered into a collaboration with Roche to develop a novel adeno-associated virus (AAV) gene therapy vector for eye diseases strong>. To this end, Avista will receive an upfront payment of $7.5 million and will be eligible to receive additional payments, clinical and sales milestone payments and resulting product royalties during the collaborative research phase, total potential transaction The amount could exceed $1 billion.

Avista is focused on developing innovative gene therapies for retinal diseases, including rare eye diseases that have a profound impact on quality of life. The company’s founder and management team composed of a group of outstanding experts in viral vector development and clinical ophthalmology, including AAV vector generation expert Dr. Leah Byrne, and José-Alain, chair of the Department of Ophthalmology at the University of Pittsburgh School of Medicine Dr. Paul Sieving, an internationally renowned medical scientist, etc. The CEO of the company is Dr. Robert Lin.

According to a press release from Avista, its computationally-guided in vivo scAAVengr (single-cell AAV engineering) platform utilizes a high-throughput approach with built-in quantitative assays to design novel cell-specific AAVs , which can rapidly transform novel gene therapy into clinical products for vision impairment diseases. The company will develop a proprietary pipeline based on an AAV variant kit that can deliver genes to individual retinal cell types. Previously, Avista has received $10 million in seed funding and multi-year foundational support from UPMC Enterprises, which invests in translational science with the potential to fundamentally transform healthcare. Recently, Avista was launched as a spin-off of UPMC.

This collaboration aims to apply Avista’s scAAVengr platform technology to develop intravitreal AAV capsids that match Roche-defined capsid characteristics. Under the terms of the collaboration, Roche has the right to evaluate and license Avista’s novel capsids and will be responsible for preclinical, clinical and commercialization activities for gene therapy programs using these novel capsids.

“We are delighted to be partnering with Roche. This will complement our internal pipeline and will accelerate the delivery of transformative therapies to patients. Traditional treatments for retinal dystrophies target only symptoms and complications , ignoring the underlying biology of the disease, current vector technologies are severely limited in their ability to target key cell types in the retina.” Dr. Robert Lin, CEO of Avista and Vice President of UPMC Enterprises, added: “Avista’s Founded to address this problem, the company’s innovative scAAVengr platform enables us to deliver gene therapy payloads via intravitreal injection to reduce immunogenicity to treat a variety of retinal diseases.”

▲Avista CEO Dr. Robert Lin (Photo source: Avista’s official website)

“UPMC is pleased to support the launch of Avista, a team with unparalleled expertise in AAV technology and clinical ophthalmology, who are addressing various issues that have a profound impact on patients’ quality of life. blinding disease,” said Ms. Jeanne Cunicelli, President of UPMC Enterprises. “We believe Avista is uniquely positioned to address a high unmet medical need in inherited retinal diseases.”

References:

[1]Avista Therapeutics Partners with Roche to Develop Next-Generation AAV Gene Therapy Vectors for Ocular Diseases. Retrieved July 19, 2022, From https:https://www.avistatx.com/ pressrelease/avista-partners-with-roche-to-develop-aav-therapy

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