Beam Therapeutics Reports Data Highlighting Advanced Manufacturing Process for BEAM-101, Investigational Baseline Drug for Sickle Cell Disease

MADRID, June 14, 2024 (GLOBE NEWSWIRE) — Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base sequencing, today reported data highlighting its streamlined, closed and automated manufacturing process for its base sequencing. Gene therapy for CD34+ hematopoietic stem and progenitor cells (HSPC) in a poster presentation at the European Hematology Association (EHA) Hybrid Congress. The optimized process was used for the production of BEAM-101 in the BEACON Phase 1/2 clinical trial in patients with severe sickle cell disease (SCD), and the data includes both preclinical and GMP clinical manufacturing experience to date.

The implementation of autologous cell and gene therapy, especially for sickle cell disease, is complex and has important implications for product quality and patient experience, said Giuseppe Ciaramella, Ph.D., president of Beam. Data presented today at EHA show that our improved manufacturing process for BEAM-101, which incorporates key technology and automation improvements, has achieved a reproducible and robust product and performance that meets high quality standards. The use of sorting technology is an important advantage, since the avoidance of double line breaks, and the high frequency of sorting generated in each cell, have contributed to significantly higher sorting rates. Ultimately, these results, combined with our impressive data package, support our belief that BEAM-101 has the potential to be a very differentiating product by reducing the number of stem cell collections required to deliver a patient dose and making a deep decision. sickle cell disease.

Beam has designed its automated CD34+ HSPC manufacturing process for BEAM-101 to be flexible for multiple patient-initiated factors and variable cell numbers, while maintaining robust cell yields and high drug product quality suitable for use in the BEACON Phase 1/2 clinical trial . Today’s data highlights the following:

• The integration of key technologies has resulted in stronger performance and improved performance.
• Automation has improved manufacturing performance, including increased yield and process consistency, and provided a three-fold increase in process capacity while reducing process length, contamination risk and operator variability.
• The drug product reproducibly met the upper limit of performance in all 14 development runs using sickle cell donor cells and nine GMP clinical runs using SCD patient cells.
• Application of basic programming to an optimized, closed and automated process produced a clinical drug product with a consistent CD34+ purity ranging from 84% to 95% and a programming rate ranging from 88% to 94%.

About BEAM-101
BEAM-101 is a cell-based gene therapy study for sickle cell disease (SCD). The one-time treatment consists of autonomous CD34+ hematopoietic stem and progenitor cells (HSPCs) that are genetically programmed into the promoter regions of the HBG1/2 genes and administered through a hematopoietic stem cell transplant. The BEAM-101 formulation is designed to prevent the transcriptional repressor BCL11A from binding to the promoter without interfering with BCL11A expression, leading to increased production of healthy and diseased fetal hemoglobin (HbF) and thereby mimicking the effects of the observed naturally occurring variation. in inheritance of fetal hemoglobin. HbF is the predominant form of hemoglobin during growth and early life. The safety and efficacy of BEAM-101 is being evaluated in the ongoing BEACON Phase 1/2 study, an open-label, single-arm, multicenter trial in elderly patients with SCD.

About Beam Therapeutics
Beam Therapeutics (Nasdaq: BEAM) is a biotechnology company committed to developing a leading, fully integrated platform for precision genetic medicine. To achieve this vision, Beam has assembled a platform that includes a range of gene editing and delivery technologies and is in the process of building in-house manufacturing capabilities. The Beam series of gas sequencing technologies is supported by base sequencing, a proprietary technology designed to enable precise, predictable and efficient single base changes, in the target gene sequence, without creating double-strand breaks in the DNA. This has the potential to enable a wide range of potential planning strategies that Beam uses to develop a diverse portfolio of basic planning programs. Beam is a principle-driven organization committed to its people, cutting-edge science, and a vision to provide life-saving treatments to patients with serious illnesses.

Cautionary Note 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 these forward-looking statements, including, but not limited to, statements related to: medical claims. and the strengths of our technologies, including sickle cell disease; our plans, and expected timing, of developing our BEAM-101 program; and our ability to develop lifelong, curative, precision medicine for patients through fundamental programming. 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 related to: and our ability to successfully realize the benefits of our portfolio. prioritization and strategic realignment; our ability to develop, obtain regulatory approval, and commercialize our product candidates, which may take longer or cost more than planned; our ability to raise additional funds, which may not be available; the uncertainty that our product candidates will receive the regulatory approval necessary to initiate human clinical studies; that preliminary testing of our products and preliminary or interim data from preclinical studies and clinical trials may not be predictive of the results or success of ongoing or later clinical trials; that initiation and enrollment, and the expected time to progress, of our clinical trials may take longer than expected; that our product candidates may experience manufacturing or supply disruptions or failures; risks associated with competing products; and other risks and uncertainties noted under the heading Risk Factors Summary and Risk Factors in our Annual Report on Form 10-K for the year ended December 31, 2023, our Quarterly Report on Form 10-Q for the quarter ended March 31, 2024. , and and any subsequent filings 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 differ may occur from time to time, and it is impossible 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:
Holly Manning
Beam Therapeutics
hmanning@beamtx.com

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