As Beam Therapeutics sets its sights on the clinic, it has signed on a new chief medical officer. Amy Simon, M.D., arrives from Alnylam, where she held various roles over a decade, culminating in vice president of clinical development.
During her time at Alnylam, Simon led clinical programs, from natural history studies and phase 1 through phase 4 studies and approvals in the U.S. and EU. Her work at the RNAi specialist includes leading the team that developed Givlaari, a treatment for acute hepatic porphyria, which scored an FDA nod in 2019.
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Simon joins as Beam prepares an IND application for its lead program for the second half of this year, said Beam CEO John Evans in a statement Monday.
“Amy’s translational and clinical development expertise will be invaluable as we prepare for our next phase as a clinical-stage company,” Evans added.
Beam’s lead program, BEAM-101, is a base-editing treatment for sickle cell disease and beta thalassemia. Both blood disorders are caused by mutations in the beta-globin gene, which result in missing or defective hemoglobin.
Beam’s approach changes a single base, or letter, in the genome to mimic a base change seen in people who have inherited higher levels of fetal hemoglobin, which can be protective in adults with blood disorders. Other companies such as Aruvant and CRISPR Therapeutics are developing genetic medicines to boost patients’ levels of fetal hemoglobin, a form of the oxygen-carrying protein found in newborn babies but is later replaced by adult hemoglobin.
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Beam is working on a second adenine base editor, BEAM-102, which changes a single base to correct the mutation that leads to sickle cell disease. Both BEAM-101 and BEAM-102 are delivered via electroporation. The company is working on two other delivery methods: adeno-associated viruses and lipid nanoparticles (LNPs).
The company boosted its LNP work in February with its acquisition of GuideTx for $120 million upfront. The hope is GuideTx’s technology will boost the reach of Beam’s medicines into new tissues and disease areas.