Vivek Ramaswamy ramps up biotech startup #14 — this time working on a gene therapy for sickle cell disease and β-thalassemia

Vivek Ramaswamy’s Roivant Sciences has nabbed its latest clinical-stage program for biotech startup #14 — Aruvant — and this time they’re going up against a couple of the leaders in the gene therapy world.

Their new drug is now dubbed RVT-1801, and it has a familiar profile, inserting a modified fetal hemoglobin gene into autologous stem cells through a lentiviral vehicle in order to treat sickle cell disease and β-thalassemia.

According to the wheeling and dealing execs at Roivant, their program is “the only known clinical-stage gene therapy to deliver the gene encoding fetal hemoglobin, which has been modified to optimize oxygen carrying capacity and anti-sickling properties.”

The drug is coming from the lab of Punam Malik, director of the Cincinnati Comprehensive Sickle Cell Center at Cincinnati Children’s, which received an undisclosed upfront payment and a milestone schedule. Malik is scheduled to reveal more about their work at the upcoming ASH conference next Monday. 

And Malik, who trained as a physician in India, was sold on Roivant’s strategy for developing new treatments for patients around the world.

Roivant is also establishing a non-profit group to improve access to drugs for patients in the developing world.

“A critical reason why we chose to work with Roivant on this program was their authentic commitment to patients globally,” said the scientist.

But they won’t be operating alone. At least 3 other biotechs are also spearheading gene therapies for these two ailments.

Just weeks ago CRISPR Therapeutics $CRSP and its partners at Vertex got a green light to start Phase I/II clinical work on CTX001, also designed to spur the production of fetal hemoglobin in patients’ stem cells. Bluebird bio $BLUE has also been working on a gene therapy for these conditions, while Sanofi’s Bioverativ $BIIV and Sangamo $SGMO had their IND approved for a rival gene therapy a few months ago. Global Blood Therapeutics, meanwhile, is looking for an OK on their new sickle cell disease drug voxelotor while Novartis and others have their own new meds in the pipeline.

Ramaswamy has raised more than $3 billion to back his growing stable of companies, which now employ more than 750 scientists and execs and support staff around the world. 

Image: Vivek Ramaswamy at BIIS18 in Shanghai Endpoints News

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Recursion Pharmaceuticals Salt Lake City, UT
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