Eli Lilly, Precision to Research Gene Therapies for Genetic Disorders

November 30, 2020 - Eli Lilly & Company and Precision BioSciences recently announced a collaboration to research and develop potential in vivo gene therapies for genetic disorders.

The collaboration and exclusive license agreement will give Eli Lilly & Company access to Precision’s proprietary ARCUS genome editing platform, which enables a range of therapeutic editing. 

The platform was created by Precision researchers using a natural genome-editing enzyme, I-Crel, Eli Lilly & Company said, which can be optimized to control for potency and specificity. 

Specifically, ARCUS leverages DNA-cutting enzymes to insert, remove, or repair the DNA of living cells and organisms.

Eli Lilly & Company will initially focus on Duchenne muscular dystrophy (DMD) therapies, as well as two other undisclosed gene targets.

"This collaboration with Precision BioSciences represents another milestone in the realization of our vision to create medicines with transformational potential, using new therapeutic modalities such as gene editing to tackle targets and indications which were previously undruggable," Andrew Adams, PhD, vice president of new therapeutic modalities at Eli Lilly & Company, said in the announcement.

Under the terms of the agreement, Precision will lead the pre-clinical research and IND-enabling activities, while Eli Lilly & Company will take responsibility for clinical development and commercialization.

Eli Lilly & Company also has the option to select up to three additional gene targets to study.

Precision will receive an upfront cash payment of $100 million, as well as an equity investment by Lilly of $35 million in Precision’s common stock.

But the company is also eligible to receive up to $420 million in development and commercialization milestone payments per product, along with tiered royalties, if Eli Lilly & Company is successful in launching a new therapy.

"We look forward to working with Lilly to leverage our deep understanding of in vivo gene editing and experience with ARCUS to develop new therapies, including a potentially transformative treatment for Duchenne muscular dystrophy," said Derek Jantz, chief scientific officer and co-founder of Precision BioSciences. 

"Collaborating with Lilly, a global healthcare leader with strong clinical and commercial experience in difficult-to-treat diseases, will help us accelerate our work aimed to solve genetic diseases with unique editing challenges,” he continued.

Genome editing technologies enable researchers to precisely edit DNA of a living organism, which creates new opportunities to correct genetic challenges from inside the body. 

In the US, the cell and gene therapy market has substantially increased over the past few years.

Specifically, pharmaceutical companies that specialized in cell and gene therapies raised nearly $2.6 billion in the third quarter of 2019, a Pharmaceutical Research and Manufacturers of America (PhRMA) report found. 

And currently, The US is dominating the cell and gene therapy market with highest absolute growth of more than 500 percent and growing at a CAGR of around 36 percent. 

Researchers found that as of March 2020, there were 362 investigational cell and gene therapies in clinical development, a 20 percent increase from 2018. 

One of the five gene therapies in development for potential treatments included a gene therapy used to recombinant AAV9 capsid to deliver a shortened version of human dystrophin to treat Duchenne muscular dystrophy. 

And at the end of March, Oregon Health & Science University (OHSU) held the first ever gene therapy clinical trial, BRILLIANCE, to address blindness-causing gene mutation, LCA10. 

The trial is one of 14 different clinical trials investigating new genetic treatments for ophthalmic conditions and nearly 50 vision-related clinical trials. 

And FDA estimated will approve up to 20 cell and gene therapy products a year by 2025.