On November 20th, Eli Lilly and Precision BioSciences announced a research collaboration to develop therapies for genetic disorders using Precision’s genome editing technology, ARCUS. The agreement includes an exclusive license agreement for ARCUS to develop therapies starting with Duchenne muscular dystrophy. The deal includes an initial cash payment of $100 million and a $35 million investment by Lilly in Precision’s common stock.
A wide range of genetic diseases is caused by mutations in a single gene, multiple genes, or chromosome damage. These diseases range from sickle cell disease to retinitis pigmentosa to various neurodegenerative diseases, including Huntington’s disease and Duchenne muscular dystrophy. Genome editing is the idea of blocking, eliminating or fixing the mutated genes, which would cure the disease in theory. One approach that has received a lot of attention for genome editing is CRISPR. However, other proteins, such as homing endonucleases, can specifically target mutated genes.
Developed by Precision BioSciences, ARCUS technology is derived from the enzyme I-Crel, a homing endonuclease. These are small DNA-cleaving enzymes with the ability to target specific sequences. Using the algal I-Crel as a scaffold to build synthetic protein, Precision BioSciences created a novel, proprietary, and versatile approach for genomic editing. ARCUS is said to produce highly specific, small proteins with the ability to make efficient insertions, deletions, and repairs to mutated genes. One of the differentiating factors between CRISPR and ARCUS is that ARCUS is produced by a single gene, while CRISPR is made of two separate genes, the CAS protein and the guide RNA.
Partnering with Eli Lilly
Eli Lilly and Precision BioSciences have entered an exclusive license agreement for ARCUS to develop therapies starting with Duchenne muscular dystrophy and two other undisclosed genes. Lilly has the right to select up to three additional genes for development under the terms of this collaboration. Precision BioSciences will conduct the pre-clinical studies, and Lilly will conduct the clinical development and commercialization. Precision BioSciences will receive Lilly’s initial payment of $135 million, $35 million investment in Precision’s common stock, and $100 million cash payment. Additionally, Precision BioSciences is eligible to receive up to $420 million in development and commercialization per product, as well as royalties. Precision BioSciences has the opportunity to co-fund clinical studies of one product to receive a higher royalty for that product.
“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.”
The Growing Field of Genome Editing
Genome editing using homing endonuclease treatment is still experimental, and it has never reached the clinic. However, the number of genome editing therapies in clinical trials is increasing by the year. Most treatments follow an ex vivo approach, where cells from the patient are collected and dosed outside the body. These cells are then reintroduced to the patient’s body after they have been modified. Very few in vivo treatments have been performed to date. This year, the first in vivo CRISPR therapy was administered to treat type 10 retinal degenerative disease, creating a breakthrough in the field. It also highlights that there is a lot of room in the market for new genome editing technologies.
By Daniel Ojeda, Ph.D.
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