Joseph McIntosh, M.D., has jumped ship from PTC Therapeutics and landed at gene therapy biotech Aruvant Sciences as its new chief medical officer, effective immediately.

The biotech came into being back in 2018 when Vivek Ramaswamy’s Roivant and Cincinnati Children’s Hospital Medical Center launched the company, which focuses on developing gene therapies for blood diseases, starting with sickle cell disease and beta thalassemia.

A rare disease specialist, McIntosh will as CMO oversee clinical development at Aruvant, with his initial focus zeroing in on the clinical program for ARU-1801, a one-time, potentially curative gene therapy for patients with sickle cell disease and beta thalassemia.

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ARU-1801, also known as RVT-1801, is designed to increase the number of functioning red blood cells in a patient by inserting a modified fetal hemoglobin gene into the individual's own stem cells.

Treating sickle cell and beta thalassemia with fetal hemoglobin is not a new idea. Both disorders are caused by mutations in the beta-globin gene, which result in missing or defective hemoglobin. Fetal hemoglobin, found in newborn babies and later replaced by adult hemoglobin, can be protective in adults who have blood disorders. This is because fetal hemoglobin has a different component that is not affected by the mutations that cause sickle cell and beta thalassemia.

Other biotechs, including Bluebird Bio, have a lentiviral vector-based gene therapy for patients with beta thalassemia, with CRISPR Therapeutics and Vertex Pharmaceuticals trialing a CRISPR-edited approach.

McIntosh, who’s also had stints at Eisai and Pfizer, comes to Aruvant from PTC, where he was vice president and head of clinical development responsible for a portfolio of assets, including two gene therapies, across hematology, oncology and genetic rare diseases.

He also worked on the biotech’s approval of Translarna, its Duchenne muscular dystrophy asset.

"I am thrilled for this exciting opportunity to work on ARU-1801 and to join such a talented team at Aruvant," said McIntosh. "We have the important goal of providing patients with sickle cell disease a potential cure with a lower conditioning chemotherapy burden.


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