Beam Therapeutics reports progress on Ex Vivo and In

First subject scheduled to be enrolled in the BEAM-101 phase 1/2 clinical trial for the treatment of sickle cell anemia in the second half of 2022

BEAM-301 named fourth development candidate for treatment of glycogen storage disease type Ia

Appointment of two additional development candidates scheduled for 2022

Company to provide updates on its 40-year pipeline and operationse JP Morgan Healthcare Annual Conference January 10, 2022 at 2:15 p.m. ET

CAMBRIDGE, Mass., Jan. 9, 2022 (GLOBE NEWSWIRE) – Beam Therapeutics Inc. (Nasdaq: BEAM), a biotech company that develops precision genetic drugs through core publishing, today outlined planned milestones for 2022 in its ex vivo programs targeting the editing of hematopoietic stem cells (HSC) and T cells and in vivo programs targeting liver cell editing using lipid nanoparticles (LNP) for delivery. Updates include that the company has selected its fourth development candidate and its first in vivo Basic edition candidate, BEAM-301, which aims to correct the R83C mutation for the potential treatment of patients with glycogen storage disorder Ia (GSDIa).

“We made significant progress across our core publishing portfolio in 2021, which resulted in the approval by the United States Food and Drug Administration of the first application of a new investigational drug. of a basic editing process, BEAM-101. We have also extended our platform, in particular with delivery of LNP from basic editors to the liver and our proprietary technology to accelerate delivery of LNP to other tissues, including HSCs, ”said John Evans, CEO of Beam. “We believe 2022 will be our most important year yet, with preparations underway to launch the BEACON-101 clinical trial with BEAM-101 for the treatment of sickle cell anemia and to complete our transition to becoming a stage company. clinical. We believe we are well positioned today, with four development candidates, a rich pipeline of early stage programs, and a platform of cutting-edge publishing and delivery technologies enabling us to deliver a new class of genetic drugs from precision. None of this would be possible without the commitment of our remarkable team of intrepid innovators. We look forward to the year ahead and continue our work to bring life-changing drugs to as many patients as possible. “

Ex vivo HSC programs

  • BEAM-101 is an experimental autologous, patient-specific HSC therapy that incorporates core modifications designed to mimic the single nucleotide polymorphisms seen in people with inherited fetal hemoglobin persistence. BEAM-101 aims to potentially attenuate the effects of mutations causing sickle cell disease (SCD) or beta thalassemia by causing an increase in fetal hemoglobin, which inhibits the polymerization of hemoglobin S (HbS). The BEACON-101 trial is a phase 1/2 clinical trial designed to evaluate the safety and efficacy of BEAM-101 for the treatment of sickle cell anemia. The trial should include an initial “sentinel” cohort of three patients, treated one at a time to confirm successful engraftment, followed by administration to a total of 45 patients. Beam has started the site selection and institutional review board approval processes for the BEACON-101 trial and plans to enroll the first subject in the second half of 2022.
  • BEAM-102 is designed to treat SCD by directly modifying the causal HbS point mutation to recreate a naturally occurring variant of normal human hemoglobin, HbG-Makassar. The Makassar variant has been reported to have the same function as the more common HbA variant and does not cause SCD. Beam plans to submit an Investigational New Drug (IND) application for BEAM-102 in the second half of 2022.

Ex vivo T cell programs

  • BEAM-201 is an investigational multiplex-based anti-CD7 CAR-T therapy designed to treat relapsed / refractory acute lymphoblastic T-cell leukemia, a serious disease affecting children and adults. Beam plans to submit an IND application for BEAM-201 in the second half of 2022.
  • Beam plans to appoint a second CAR-T development candidate in 2022.

In Vivo LNP liver targeting programs

  • BEAM-301, the company’s newest development candidate, is a liver-targeting LNP formulation of basic editing reagents designed to correct the R83C mutation. R83C is the most common pathogenic mutation in GSDIa, a life-changing genetic disease for which no approved disease-modifying therapy is available today. Beam plans to launch studies enabling IND for BEAM-301 in 2022.
  • Beam plans to appoint a second developmental candidate targeting the liver in 2022.

JP Morgan Healthcare Conference
Mr. Evans will present updates to Beam’s pipeline and activities in a presentation at 40e JP Morgan Healthcare Annual Conference on Monday, January 10, 2022 at 2:15 p.m. ET. A live webcast will be available in the investors section of the Company’s website at www.beamtx.com and will be archived for 60 days following the presentation.

About Beam Therapeutics
Beam Therapeutics (Nasdaq: BEAM) is a biotechnology company committed to establishing the leading fully integrated platform for precision genetic drugs. To realize this vision, Beam has assembled a platform that includes a suite of gene editing and delivery technologies and is in the process of developing in-house manufacturing capabilities. Beam’s suite of gene-editing technologies is anchored in base editing, a proprietary technology that enables precise, predictable and efficient simple base changes at the level of targeted genomic sequences without making double-stranded breaks in DNA. This enables a wide range of potential therapeutic editing strategies that Beam uses to advance a diverse portfolio of core editing programs. Beam is a values-driven organization committed to its people, cutting-edge science and a vision to provide lifelong treatment to patients with serious illnesses.

Caution regarding forward-looking statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Investors are cautioned not to place undue reliance on such forward-looking statements, including, but not limited to, statements relating to: our plans, and expected timeline, to nominate additional development candidates, initiate studies enabling IND and submit IND applications; the therapeutic applications and the potential of our technology, in particular with regard to sickle cell anemia, beta-thalassemia, T-ALL, GSDIa and LNP; the planned launch and design of our BEACON-101 clinical trial, including the timeline for enrollment of the first subject in the trial; our presentations planned at a future conference; and our ability to develop precision, curative, and permanent genetic drugs for patients through core editing. Each forward-looking statement is subject to significant risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement, including, without limitation, risks and uncertainties relating to: our ability to develop, obtain regulatory approval for, and commercialize our product candidates, which may take longer or cost more than anticipated; our ability to raise additional funds, which may not be available; our ability to obtain, maintain and enforce patents and other intellectual property protections for our product candidates; the potential impact of the COVID-19 pandemic; that preclinical testing of our product candidates and preliminary or interim data from preclinical studies and clinical trials may not be predictive of the results or success of ongoing or future clinical trials; that recruiting for our clinical trials may take longer than expected; that our product candidates may experience interruptions or failures in manufacturing or supply; risks associated with competing products; and other risks and uncertainties identified under the headings “Summary of Risk Factors” and “Risk Factors” in our annual report on Form 10-K for the year ended December 31, 2020, our quarterly report on Form 10-Q for the quarter ended March 31, 2021, our Quarterly Report on Form 10-Q for the quarter ended June 30, 2021 and our Quarterly Report on Form 10-Q for the quarter ended September 30, 2021, and in any subsequent filing with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of this press release. Factors or events that could cause our actual results to vary may occur from time to time, and we cannot predict all of them. We assume no obligation to update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as required by applicable law.

Contacts:

Investors:
Chelcie Lister
THRUST Strategic Communication
[email protected]

Media:
Dan Budwick
1AB
[email protected]

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