Beam Therapeutics Announces Oral and Poster Presentations at 24th American Society of Gene and Cell Therapy Annual Meeting

CAMBRIDGE, Mass., April 27, 2021 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the company will present data highlighting its proprietary base editing approach in several presentations during the 24^th American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, which will be hosted virtually May 11-14, 2021. In addition, Beam has been selected as a workshop presenter in the pre-meeting program taking place on May 10, 2021.

“Since our founding in 2017, we have been diligently focused on advancing our proprietary base editing technology to develop treatments for patients suffering from severe diseases, and we are pleased to present updates showcasing the potential of our technology during ASGCT,” said Giuseppe Ciaramella, Ph.D., president and chief scientific officer of Beam. “Taken together, these data highlight the depth of our understanding of base editing, including our work in delivery optimization to ensure efficient editing in the target organ, as well as analytical technology to confirm specificity and efficacy of our base editors, both of which are critical as we advance toward clinical development. We look forward to presenting these and additional data from our LNP program at ASGCT, as we work toward the nomination of our first development candidate from our liver portfolio and the planned IND application for our lead base editor program, BEAM-101, later this year.”

Pre-Meeting Program: Moving Genome Editing to the Clinic: From Technology to Therapeutics
Session: Gene Editing Workshop
Date and Time: Monday, May 10, 2021, 10:00 a.m. – 2:00 p.m. ET

Poster Presentation: Using Base Editing and LNP Delivery to Correct Disease-Causing Mutations Underlying Genetic Liver Diseases
Session: Synthetic/Molecular Conjugates and Physical Methods for Delivery
Date and Time: May 11-14, 2021

Abstract summary: Base editing enables programmable single-base genomic mutations and has the potential to permanently cure serious genetic diseases. Realizing its therapeutic potential requires three key elements, including the development of new base editors with appropriate specificity, editing window, and efficacy; sgRNA sequence and chemistry for optimal potency and target specificity; and safe and effective methods for mRNA and sgRNA delivery to target organs and intracellular compartments. For this study, Beam evaluated editing levels in the liver of non-human primates using a lipid nanoparticle (LNP) delivery system containing a novel adenine base editor (ABE) and reporter sgRNA. These data demonstrate that Beam’s base editors in combination with LNP delivery have the potential to treat genetic liver diseases.

Oral Presentation: LC-MS Confirmation of Single Amino Acid Correction by Base Editing
Session: Pharmacology/Toxicology Studies or Assay Development
Date and Time: Friday, May 14, 2021, 12:45 p.m. – 1:00 p.m. ET

Abstract summary: Base editing is emerging as a powerful next generation editing technology; however, developing adequate analytical assays to confirm precise base editing at the protein level is critical and has proven historically challenging. In this study, Beam utilized liquid chromatography mass spectrometry (LC-MS) for multiple analytical assays to confirm the precise correction at the amino acid level by base editing, which proved to provide a unique solution for confirmation and quantitation of single amino acid corrections after base editing.

About Beam Therapeutics
Beam Therapeutics (Nasdaq: BEAM) is a biotechnology company committed to establishing the leading, fully integrated platform for precision genetic medicines. To achieve this vision, Beam has assembled a platform that includes a suite of gene editing and delivery technologies and is in the process of building internal manufacturing capabilities. Beam’s suite of gene editing technologies is anchored by base editing, a proprietary technology that enables precise, predictable and efficient single base changes, at targeted genomic sequences, without making double-stranded breaks in the DNA. This enables a wide range of potential therapeutic editing strategies that Beam is using to advance a diversified portfolio of base editing programs. Beam is a values-driven organization committed to its people, cutting-edge science, and a vision of providing life-long cures to patients suffering from serious diseases.

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 these forward-looking statements, including, but not limited to, statements related to: our plans for scientific publications; our expected timing for filing an investigational new drug application for BEAM-101; our efforts toward the nomination of our first development candidate from our liver portfolio; and the therapeutic applications and potential of our technology, including our ability to develop life-long, curative, precision genetic medicines for patients through base editing. Each forward-looking statement is subject to 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 related to: our ability to develop, obtain regulatory approval for, and commercialize our product candidates, which may take longer or cost more than planned; our ability to raise additional funding, which may not be available; our ability to obtain, maintain and enforce patent and other intellectual property protection 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 and clinical trials may not be predictive of the results or success of ongoing or later clinical trials; that enrollment of our clinical trials may take longer than expected; that our product candidates may experience manufacturing or supply interruptions or failures; risks related to competitive products; and the other risks and uncertainties identified under the heading “Risk Factors” in our Annual Report on Form 10-K for the year ended December 31, 2020, and in any subsequent filings with the Securities and Exchange Commission. These forward-looking statements (except as otherwise noted) speak only as of the date of this press release. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them. We undertake no obligation to update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by applicable law.

Contacts:

Investors:
Chelcie Lister
THRUST Strategic Communications
chelcie@thrustsc.com

Media:
Dan Budwick
1AB
dan@1abmedia.com