Novartis plots Precision attack on sickle cell, paying $75M and putting up $1.4B in biobucks to form in vivo gene editing pact

One year after Intellia’s landmark data, Novartis is making a play for the in vivo gene editing market. The move sees the Swiss pharma pay Precision BioSciences $75 million upfront in a combination of cash and equity investment along with up to $1.4 billion in milestones to secure rights to a potential treatment for sickle cell disease and beta thalassemia. 

Intellia, working with its partner Regeneron, has shown over the past year that CRISPR/Cas9 in vivo gene editing can cause high, seemingly durable levels of gene knockdown in humans. While questions about the Intellia data, and the concept more broadly, remain unanswered, there is now early evidence that the approach may be effective and, as importantly, safe. Precision is one of a clutch of companies barreling toward the clinic in the wake of Intellia, and the potential of its Arcus platform to provide greater precision and versatility than CRISPR/Cas9 and zinc finger nuclease has now attracted a suitor.

Novartis has tasked Precision with designing a nuclease capable of inserting a therapeutic transgene into a safe harbor location to treat sickle cell disease and beta thalassemia. While biotechs such as bluebird bio and Vertex are working on ex vivo gene editing treatments for the diseases, which require cells to be removed, edited and reinserted, Novartis wants to develop a simpler, off-the-shelf treatment.

“The in vivo gene editing approach that we are pursuing for sickle cell disease could have a number of significant advantages over other ex vivo gene therapies currently in development,” Derek Jantz, Ph.D., chief scientific officer and co-founder of Precision, said in a statement. “Perhaps most importantly, it could open the door to treating patients in geographies where stem cell transplant is not a realistic option.”

While ex vivo gene therapy involves complex logistics, an in vivo option could be given off the shelf and provide a one-and-done approach to treating the disease. That prospect has attracted the Bill & Melinda Gates Foundation, which joined with Novartis to discover and develop an accessible in vivo sickle cell gene therapy last year.

To add to its in vivo capabilities, Novartis is set to pay $50 million in cash to partner with Precision. The deal also features a $25 million equity investment priced at $2.01 per share, a 20% premium over the recent average for the stock, as well as up to $1.4 billion in milestones, research funding and royalties ranging from the mid-single-digit to low-double-digit percentages. 

The Novartis deal extends Precision’s cash runway out to the second quarter of 2024, versus mid-2023 prior to the agreement. Precision expects three of its wholly owned preclinical in vivo programs to get to the clinical trial submission stage over the next three years, with a filing for a candidate designed to reduce expression of the PCSK9 gene penciled in for as early as the end of this year.