Novartis, already up on CRISPR, CAR-T, looks to new gene editing R&D

Novartis has penned a new deal with Homology Medicines as it looks to boost its already impressive R&D work in cell and gene therapy.

5AM Ventures-seeded biotech Homology will allow Novartis to use its gene editing tech to develop new treatments for certain ophthalmic and hemoglobinopathy diseases, although neither company would tell me what exactly they were seeking to target.

And though financial details are not being revealed, we do know that Homology gets an upfront payment as well as equity investment from Novartis, and some cash "to advance the programs and to explore new opportunities for Homology’s technology platform." Homology could also get undisclosed biobucks in the form of sales success from the pact.

Under the deal, Homology holds on to U.S. commercial rights and is set to share U.S. profits with Novartis for in vivo applications related to the hemoglobinopathy program, already an area of focus for Homology. The Swiss Big Pharma has connections with the biotech, given that it was one of its many backers in a $83.5 million series B round back in August.

This tech builds on the work Novartis is doing out of its phoenix-from-the-flames cell and gene therapy unit, which includes CAR-T and collaborative work on CRISPR-Cas9. In late August, Novartis became the first-ever company to gain approval for a chimeric antigen receptor T (CAR-T) med in the form of Kymriah (tisagenlecleucel), a cell therapy now given the green light for a rare form of acute lymphoblastic leukemia. And since 2015, it’s been working with Intellia Therapeutics and Caribou Biosciences on using CRISPR ex vivo for engineering CAR-Ts and hematopoietic stem cells.

It now has a third way, as Lloyd Klickstein, M.D., Ph.D., head of translational medicines, new indications discovery unit at the Novartis Institutes for Biomedical Research, told FierceBiotech: "With this collaboration, Novartis is adding another emerging technology to our cell and gene therapy toolbox. We believe Homology’s technology has great potential for development of new therapies.

"Their technology, known as AAV-mediated editing by directed homologous recombination (AMEnDR), uses adeno-associated virus (AAV) vectors isolated from human CD34-positive cells for genome editing. Uniquely, Homology’s AAVs are able to trigger homologous recombination, a naturally occurring process, to change DNA letters inside cells and correct specific gene mutations with what appears to be efficiency greater than that observed with other gene editing technologies being explored today."

Klickstein added that studies are required to test whether directed homologous recombination may be a preferred method of genome editing, compared to other approaches in certain diseases.

He said that this pact is part of the Novartis strategy to "work with innovative leaders" in the emerging genome editing field. He would not be drawn out on how and when the pact was made, and whether this was part of its investment strategy back in August when it backed its funding round.

"These are early days in the exploration of genome editing technologies for therapeutic use," Klickstein said. "It’s too early in our collaboration with Homology to discuss timelines, but we are acutely aware of the urgent needs of our patients and our hope is to develop these technologies rapidly to bring definitive new therapies to the clinic."

Homology’s own leading candidate is currently in preclinical IND-enabling studies and is slated for future trials in an inborn error of metabolism disease.