Pharmaceutical Business review

Avista Therapeutics, Roche partner to develop AAV gene therapy vectors for eyes

Image: The companies will develop new AAV gene therapy vectors for the eyes. Credit: Rudy and Peter Skitterians from Pixabay.

Applying Avista’s single-cell adeno-associated virus (AAV) engineering (scAAVengr) platform technology, the partnership intends to develop intravitreal AAV capsids matching a capsid profile defined by Roche.

As per the terms of the partnership, Roche can evaluate and license novel capsids from Avista.

It will be responsible for carrying out a preclinical, clinical and commercialisation activities for gene therapy programmes using these novel capsids, which will be different from Avista’s internal pipeline.

Avista Therapeutics CEO Robert Lin said: “We are excited to enter into this collaboration with Roche, a global leader in health care.

“This collaboration will complement our in-house pipeline and will accelerate the delivery of transformative therapies to patients.”

Avista will secure an upfront payment of $7.5m and, if successful, it can get further payments during the research phase of the partnership, besides clinical and sales milestone payments and royalties for resulting products with a total potential deal value that may be more than $1bn.

Avista’s computationally guided, in vivo scAAVengr platform uses a high-throughput approach with built-in quantitative validation of novel cell-specific AAVs. This process allows quick translation of transformative gene therapies to the clinic for diseases affecting people’s vision.

It will develop a pipeline that is built on a toolkit of variants of AAV to target gene delivery to individual retinal cell types.

Avista secured $10m in seed funding and foundational support from UPMC Enterprises, which invests in translational science.