Lilly announces the Institute for Genetic Medicine and US$700 million investment

USA – Eli Lilly and Company has announced the formation of the Lilly Institute for Genetic Medicine and a US$700 million investment to build a cutting-edge facility in the Boston Seaport.

This investment, which is part of the company’s strategy to advance RNA-based therapeutics, builds on Prevail Therapeutics, a gene therapy pioneer based in New York City, which was acquired and rapidly expanded in 2020.

Prevail Therapeutics of New York has three gene-based therapies in phase 1/2 trials. PR001 is the lead candidate for Parkinson’s disease patients with GBA1 mutations and neuronopathic Gaucher disease.

By injecting an engineered version of the GBA1 gene into the spinal cord, the treatment aims to address patients’ natural mutations in the GBA1 gene.

Working together, researchers in Boston and New York will use promising RNA and DNA-based technologies to develop therapies with the potential to treat or prevent diseases in ways that traditional medicines find difficult or impossible.


Establishing the Lilly Institute for Genetic Medicine will allow us to pair cutting-edge technologies with our deep biological expertise in several areas including neuroscience and diabetes,” said Andrew C. Adams, vice president of genetic medicine at Lilly and co-director of the Institute.

Lilly will focus on medicines acting at the nucleic acid level to advance an entirely new class that target the root cause of diseases, an approach that is fundamentally different than medicines available today.”

According to the company, the new hub, dubbed the Lilly Institute for Genetic Medicine, will create more than 250 new research jobs in Boston, in addition to expanding its New York team to up to 200 employees. The project is set to open in 2024.

Lilly intends to fuel the development of genetic medicines through the Institute’s operations.

Currently, genetic medicine accounts for more than 20% of Lily's portfolio in diabetes, immunology, and central nervous system treatments.

The new facility will provide resources for researchers in New York and Boston to develop new RNA and DNA-based therapies for diseases that have not yet been addressed by conventional medicine.

The Institute will enhance our efforts on neurodegenerative diseases and integrate Lilly’s genetic medicine research and platforms with the goal of advancing promising and potentially life-altering new medicines from the lab to clinical studies and ultimately to patients,” said Franz Hefti, CEO of Prevail Therapeutics at Lilly and co-director of the Institute.

The hope is that the injected gene will stimulate the production of the enzyme GCase, which can then recycle glycolipids that accumulate and are linked to both neurodegenerative diseases.

The new research facility and Prevail acquisition join other RNA-focused moves made by Lilly in 2021, including a collaboration with MiNA Therapeutics for five targets worth up to US$1.2 billion.

In addition, Lilly signed a deal with ProQR that could be worth more than US$1 billion for an additional five targets using the biotech’s RNA editing technology.

Franz Hefti, CEO of Prevail, and Andrew C. Adams, vice president of genetic medicine at Lilly, will co-direct the institute.

The company also intends to use a portion of the institute as a shared office space for biotech startups, which will be able to work and collaborate with Lilly’s scientists.

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