Averna Therapeutics

Company Overview

Averna Therapeutics (formerly Exsilio Therapeutics) is a clinical-stage genomic medicine company headquartered in Watertown, Massachusetts, focused on developing RNA-based therapeutics that durably insert therapeutic genetic instructions into safe harbour sites in the human genome. The company was founded in 2022 and rebranded as Averna Therapeutics in early 2025. Averna raised $82 million in a Series A financing round in 2024, co-led by Novartis Venture Fund and Delos Capital, with participation from OrbiMed, Insight Partners, J.P. Morgan Life Sciences Private Capital, CRISPR Therapeutics, Innovation Endeavors, Invus, Arc Ventures, and Deep Insight. Averna was recognised as one of Endpoints News's 11 Most Promising Biotech Companies in 2025.

Headquarters and Global Presence

Averna Therapeutics is headquartered at 134 Coolidge Avenue, Watertown, Massachusetts, United States, within the greater Boston biotechnology corridor. The company's operations are currently focused in the United States, where it is advancing its safe harbour genomic medicine platform through preclinical and early clinical development. The involvement of co-founder Tal Zaks and Israeli biotech connections reflects broader international scientific networks supporting the company's technology development.

Founding and History

Averna Therapeutics was founded in 2022, initially under the name Exsilio Therapeutics, to develop a novel approach to gene therapy based on site-specific retrotransposons. One of the company's key co-founders is Tal Zaks, MD, PhD, who served as Chief Medical Officer at Moderna during the development and launch of the COVID-19 mRNA vaccine, bringing deep expertise in RNA medicine and LNP delivery to the venture. The company raised an $82 million Series A financing in 2024, secured a high-profile investor syndicate including CRISPR Therapeutics and the Novartis Venture Fund, and rebranded as Averna Therapeutics in early 2025. Thomas M. Barnes, PhD, was appointed as CEO on 27 January 2025.

Therapy Areas and Focus

Averna's technology platform is designed to be broadly applicable across multiple disease areas where durable genomic correction or augmentation could provide therapeutic benefit. Genetic diseases represent the primary near-term focus, where the ability to insert a corrective gene safely and durably into the genome could offer curative potential. Oncology and autoimmune disease are additional target areas where delivery of immunological or cytotoxic instructions into specific cell types could enable new treatment modalities. The company's LNP-based delivery system, analogous to validated mRNA vaccine platforms, enables non-viral administration with the potential for redosing and scalable manufacturing.

Technology Platforms and Modalities

Averna's scientific foundation is a proprietary approach inspired by naturally occurring site-specific retrotransposons — mobile genetic elements that have evolved to insert at defined safe harbour locations in the genome without disrupting normal cellular function. Averna's platform encodes the retrotransposon machinery in RNA, which is delivered to cells via lipid nanoparticles (LNPs). Once inside the cell, the RNA instructions are translated and catalyse the durable integration of a therapeutic gene payload into a safe harbour genomic site. Because the instructions are RNA-based, the system avoids the risks associated with integrating viral vectors such as AAV and enables redosing. The approach can theoretically accommodate large genetic payloads — a key limitation of current gene therapy technologies — and is applicable to multiple cell and tissue types.

Key Pipeline and Programs

Averna Therapeutics is a platform-stage company, with its pipeline in preclinical development across genetic diseases, oncology, and autoimmune conditions. The company's strategy is to validate its safe harbour retrotransposon insertion technology across multiple disease targets, establishing proof of concept before advancing selected programmes into clinical trials. The $82 million Series A financing provides the capital base to progress multiple disease programmes towards clinical candidate selection and Investigational New Drug (IND) applications. Patent activity around the safe harbour gene insertion technology reflects the company's efforts to protect and differentiate its platform from competing genomic medicine approaches.

Key Personnel

Thomas M. Barnes, PhD, serves as Chief Executive Officer of Averna Therapeutics, appointed on 27 January 2025. Tal Zaks, MD, PhD, who served as the company's Interim CEO and is a co-founder, continues as Executive Chairman of the Board, contributing his expertise from Moderna's mRNA vaccine programme. Robert Mabry, PhD, was appointed Chief Scientific Officer in March 2026, overseeing scientific strategy. Richard Morris was appointed Chief Financial Officer in March 2026, managing the company's financial operations as it progresses its pipeline.

Strategic Partnerships

Averna's $82 million Series A financing, closed in 2024, was co-led by Novartis Venture Fund and Delos Capital, with a high-profile syndicate including OrbiMed, Insight Partners, J.P. Morgan Life Sciences Private Capital, CRISPR Therapeutics, Innovation Endeavors, Invus, Arc Ventures, and Deep Insight. The inclusion of CRISPR Therapeutics as an investor is particularly notable, reflecting interest in Averna's novel gene insertion approach from a leading gene editing company. The involvement of Novartis Venture Fund links Averna to one of the world's largest pharmaceutical companies and its gene therapy and genomic medicine interests. Averna continues to build its platform and explore further strategic partnerships to accelerate development across its target disease areas.

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