Vertex Pharmaceuticals is hoping to build upon its existing rare blood disease treatment portfolio, teaming up with Orna Therapeutics’ ReNAgade in efforts to develop next-gen gene therapies for sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).
“These are very expensive experiments to run—a small biotech like ReNAgade or Orna can only run a few of these,” Orna Chief Scientific Officer Joe Bolen, Ph.D., told Fierce Biotech. “Vertex is allowing us to really expand the number of experiments that we can run, explore chemistries that we would never have gotten to if it wasn’t for this collaboration.”
Vertex is paying Orna $65 million upfront, in part via convertible note, according to a Jan. 7 release. Orna—which acquired ReNAgade in May 2024 for an undisclosed amount—could also make up to $635 million in preclinical, R&D, regulatory and commercial milestones related to SCD or TDT products.
Vertex can also option rights in other indications, in which Orna could receive up to $365 million biobucks per product for no more than 10 products. The RNA-focused company will also be able to collect royalties on any potential therapies resulting from the three-year deal.
Massachusetts-based Orna touts its own lipid nanoparticle (LNP) delivery tech that Vertex will now use in efforts to improve development for new gene editing therapies.
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“The approach was pioneered at ReNAgade,” Bolen said, adding that the science stems from work conducted when he served as Moderna’s president of R&D and CSO over 10 years ago.
Orna’s approaches are designed to reach parts of the body other than the liver, such as directly to the bone morrow, which opens the door to treatment for genetic diseases such as SCD and TDT.
The immediate, off-the-shelf technology is designed to bypass harsh preconditioning regimens tied to traditional cell therapies, Bolen explained.
The ultimate goal is to develop medicines that can be broadly used by patients who can’t or won’t undergo the typical ex vivo stem cell treatment available now, Bolen said.
Orna acquired competing RNA company ReNAgade last year, swallowing the biotech that had launched with help from some Moderna vets and $300 million in early 2023. One of those former Moderna leaders is Orna’s current leader Amit Munshi.
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“Vertex is a leader in delivering next-generation approaches to treating hemoglobinopathies, and we are excited to collaborate with them to develop in vivo therapies that leverage our proprietary technologies to achieve unprecedented delivery to HSCs,” Orna CEO Munshi said in the release. “Today’s collaboration further validates our industry leading extra-hepatic LNP delivery chemistries and highlights the importance of delivery to enable the next wave of RNA medicines.”
Orna was one of the top biotech money raisers in 2022, securing a $221 million series B. The same year, the company snagged a partnership with Merck & Co. that focuses on circular RNA (oRNA)—Orna’s attempt to improve on the production, protein expression and immunogenicity of traditional linear mRNA therapies. Merck paid $150 million upfront and is offering Orna up to $3.5 billion in milestones to advance programs against infectious diseases and cancers.
Orna’s internal efforts center around its panCAR delivery system, which produces in vivo CAR products. The biotech’s lead programs are in autoimmune and oncology diseases and are slated to enter the clinic in the first half of 2026.
The company currently staffs about 100 employees, which includes its wholly owned subsidiary ReNAgade, Bolen said.
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For Vertex, the partnership follows recent FDA approvals in both SCD and TDT. The biopharma, alongside CRISPR Therapeutics, received FDA approval in SCD for their gene therapy Casgevy (exa-cel) in late 2023. The therapy was the first treatment to make it to market in the indication, closely followed by bluebird bio’s Lyfgenia (lovo-cel)—also a gene therapy.
Less than six weeks later, Casgevy snagged an FDA nod for TDT, becoming the first treatment for the rare blood disorder using CRISPR gene editing technology.
