Avista Therapeutics Expands AI Gene Therapy Platform to Advance Genetic Medicine Delivery

The expansion includes an AI-driven enhancement of its adeno-associated virus (AAV) capsid design programme and the launch of an Innovation Platform focused on large gene delivery and manufacturing constraints.

Avista Therapeutics, a preclinical gene therapy company, has announced a significant expansion of its gene therapy platform, aimed at addressing persistent challenges in the delivery of genetic medicines. 

The expansion includes an AI-driven enhancement of its adeno-associated virus (AAV) capsid design programme and the launch of an Innovation Platform focused on large gene delivery and manufacturing constraints.

The company said the expanded platform uses machine learning and structural dynamics to overcome limitations in AAV capsid engineering, a key bottleneck in gene therapy development. The announcement was made on Monday from Pittsburgh.

Central to the expansion is the launch of ARTEMIS, an AI-powered platform designed to accelerate capsid engineering. ARTEMIS is built on large non-human primate datasets generated internally using Avista’s scAAVengr-HUnT workflow. These datasets capture single-cell resolution data on millions of AAV variants across different serotypes, tissues, routes of administration, and animal models.

According to Avista, ARTEMIS integrates these datasets with proprietary protein language models and molecular dynamics simulations of capsids. By combining sequence-based models with structural and mechanistic information, the platform aims to identify capsid features linked to biological fitness—an area that sequence-only approaches have struggled to address.

In parallel, Avista has launched an Innovation Platform focused on tackling technical barriers in AAV targeting, delivery of large genetic payloads, and scalable manufacturing. The platform brings together structural biology, protein engineering, and clinical expertise to support the development of new approaches in gene therapy design and production.

“The powerful combination of sequence and structural mechanism AI models uniquely positions Avista as a leader in capsid design, with the ability to rapidly develop safe and highly efficient delivery of genetic medicines in tissues across the body,” said Robert Lin, PhD, chief executive officer of Avista Therapeutics.

Lin added that the company is extending these capabilities beyond retinal indications, with a pipeline that includes preclinical programmes for X-linked retinoschisis (XLRS), PRPF31-related retinal disease, and central nervous system (CNS) indications.

Avista Therapeutics was spun out of the University of Pittsburgh and operates at the preclinical stage. The company focuses on applying artificial intelligence and computational biology to improve the delivery efficiency and scalability of gene therapies targeting retinal and CNS disorders.

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