BEAM

John Evans introduced Beam's vision of providing lifelong cures using base editing. Beam is expanding its pipeline with a new development program for PKU (BEAM-304) leveraging base editing and LNP delivery. Amy Simon detailed PKU's clinical manifestations and unmet needs. Gopi Shanker discussed the base editing approach for BEAM-304 and early preclinical data. Kiran Musunuru shared insights on PKU variants and regulatory interactions. Sravan Emany announced a strategic financing agreement to support risto-cel's commercialization and strengthen the balance sheet.

Anticipate filing IND for BEAM-304 in 2026, report initial BEAM-301 data in GSDIa, complete BEAM-103 healthy volunteer study, and continue advancing in vivo HSC editing. Look forward to submitting risto-cel BLA as early as year-end and accelerating the path to approval for lead programs.

Q: Talked about regulatory path forward and addressing multiple mutations in PKU, and strategic financing impact on reallocating capital.

A: Gopi mentioned research underway for other mutations with fast timelines, Amy spoke about FDA collaboration and adaptive trial design, Sravan said financing provides flexibility for commercial launch and pipeline growth.

Q: Questions on IP for R408W and LNP delivery in 304.

A: John said Beam has access to necessary IP, and 304 uses similar LNP approaches as 302, 301 with internal ionizable lipids.

Q: Questions on FDA draft guidance for individualized therapy and PKU mutations, 304 guide RNAs and bystander editing.

A: Kiran discussed FDA interactions and plausible mechanism framework, Gopi talked about unique guide RNAs for mutations and confident bystander profile.

Q: Question on sickle cell market share assumptions for risto-cel.

A: Giuseppe Ciaramella talked about market demand, risto-cel's manufacturing process optimization.

Q: Questions on AATD upcoming readout and pivotal.

A: John talked about data update on 302, dosing schedule, efficacy, safety, and durability.

Q: Questions on PKU patient responses to current options and Phase I/II age range.

A: Amy said R408W patients don't respond well to current options, and FDA collaborative on age range recruitment.

Q: Questions on A1AT, GSK returning rights, and U.S. enrollment for pivotal.

A: John didn't comment on GSK but said BEAM-302 has best-in-class data, and U.S. enrollment part is premature.

Q: Question on PKU patient mutation copies and Phase I/II design.

A: Gopi said one copy correction sufficient, and Amy talked about age range recruitment.

Q: Questions on PKU editing rate vs other diseases and OpEx for PKU program.

A: Gopi explained low threshold for PKU editing, Sravan said PKU is in runway guidance and cost balanced across portfolio.

Q: Questions on plausible mechanism pathway for AATD and LNP safety for PKU.

A: John said plausible mechanism not necessary for AATD traditional path, and Beam has expertise in LNP for PKU.

Q: Question on next-gen sickle cell program prioritization.

A: Giuseppe Ciaramella talked about in vivo program's ease of delivery and progress.

Q: Question on 103 healthy volunteers and in vivo editing efficiencies.

A: Giuseppe Ciaramella talked about 103's antibody component and comparable editing efficiencies