IND Accepted for Sickle Cell Disease Cell Therapy


Beam Therapeutics will assess the safety and efficacy of BEAM-101 for SCD in the phase 1/2 BEACON-101 study.

Beam Therapeutics has received investigational new drug (IND) clearance for their cell therapy candidate BEAM-101 for the potential treatment of sickle cell disease (SCD).

“We are thrilled to share that the FDA has cleared our first IND. BEAM-101 has the potential to offer a one-time treatment for patients with sickle cell disease, and this clearance enables the important transition from a preclinical to a clinical-stage company, bringing us closer to our ultimate goal of helping patients,” John Evans, chief executive officer, Beam Therapeutics, said in a statement. “As leaders in the field of base editing, this milestone underscores the expertise of our team and the significant potential of our technology. We are grateful to the team members who have dedicated countless hours to this effort, and we look forward to this program’s continued evaluation in the clinic. As we look toward 2022, we believe we are well-positioned both financially and organizationally to execute on our vision.”

BEAM-101 is an ex vivo base editing autologous cell therapy product designed to alleviate the effects of mutations in SCD or beta-thalassemia by mimicking single nucleotide polymorphisms seen in these diseases. The approved IND marks a first for the next-generation CRISPR base editing technology which can make single base changes without double strand DNA breakage. The safety and efficacy of BEAM-101 will be evaluated in the phase 1/2 BEACON-101 clinical trial.

Beam is also developing another base editing candidate for SCD called BEAM-102, which they have initiated IND-enabling studies for. This candidate is designed to directly correct the SCD causative mutation by recreating HbG Makassar.

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“In addition to the clearance of our IND for BEAM-101, we are pleased to announce that we have initiated IND-enabling studies for BEAM-102, our Makassar base editing approach for the treatment of sickle cell disease,” Giuseppe Ciaramella, PhD, president and chief scientific officer, Beam, added to the statement. “We have also made continued progress on BEAM-201, our multiplexed base editing approach for the treatment of relapsed/refractory T-cell acute lymphoblastic leukemia and T-cell lymphoblastic leukemia as well as continued advancement across our robust portfolio, as showcased by a number of recent and upcoming data presentations demonstrating our leadership in base editing as well as optimized delivery technologies for our programs. Importantly, we also expect to nominate our first development candidate for in vivo base editing in the liver using LNP delivery for the treatment of patients with glycogen storage disease type Ia (R83C mutation) by the end of the year. We’ve made extraordinary progress as a company this year and remain committed to advancing novel science so that we may reach as many patients as possible.”

Beam Therapeutics will present preclinical data on BEAM-102 at the 63rd American Society of Hematology Annual Meeting & Exposition, December 11-13, as well as their in vivo high-throughput lipid nanoparticle (LNP) screening approach to identify novel LNPs to deliver base editors to hematopoietic stem cells and other tissues beyond the liver. The company also presented positive preclinical data on their CD5+ chimeric antigen receptor (CAR) T multiplex base editing approach at the 36th Annual Meeting of the Society for Immunotherapy of Cancer (SITC), November 10-14, 2021.

Beam Therapeutics provides business and pipeline updates and reports third quarter 2021 financial results. News release. November 8, 2021.
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