Partner Case Study: Accelerating Gene Editing Manufacturing

In March 2022, Beam Therapeutics and ElevateBio embarked on a groundbreaking partnership to manufacture BEAM-101, an investigational base editing therapy for sickle cell disease. The therapy represents a novel approach to treating this devastating condition by mimicking genetic variants found in individuals with hereditary persistence of fetal hemoglobin. To advance this potentially transformative treatment to clinical trials, Beam turned to ElevateBio’s expertise in cell and gene therapy production, combining rigorous quality standards with the ability to move at unprecedented speed.

The Challenge

Manufacturing gene-edited cell therapies presents unique complexities beyond traditional cell therapy production. Base editing, like all gene editing cell-therapy technologies, requires careful process control to maintain editing efficiency and cellular viability. Traditional manufacturing approaches, optimized for other cell therapies, needed significant adaptation for this novel technology. Additionally, Beam faced urgent timeline pressures – they had identified potential clinical trial participants and needed to accelerate their manufacturing readiness without compromising quality.

"Base editing represents a new frontier in genetic medicine, and its manufacturing demands match that innovation," says Brian Riley, chief manufacturing officer of Beam. "We needed a partner who could not only handle the technical complexity but move at the speed required to help us reach waiting patients."

The Approach: Innovation at Speed

Beam partnered with ElevateBio's BaseCamp to define a plan for clinical supply, which ultimately led to an accelerated tech transfer to support BEAM-101’s path to clinical trials.

BaseCamp's comprehensive strategy combined technical expertise with dedicated program management. The seamless integration of program management and the Manufacturing, Science and Technology (MSAT) group provided end-to-end oversight for technology transfer activities, while parallel manufacturing readiness and analytical method transfer activities compressed traditional timelines. The team leveraged a risk-based approach along with input from the process development group for successful translation of the novel base editing process into GMP manufacturing. This was supported by an accelerated training and qualification program that maintained the highest quality standards.

"We recognized that standard technology transfer approaches would not meet the timeline requirements," explains Mike Paglia, Chief Technology Officer, ElevateBio BaseCamp. "Our team developed an integrated strategy leveraging our development, manufacturing and quality teams that maintained the highest level of quality while dramatically accelerating the timeline of the technology transfer of the Beam-101 process to ElevateBio BaseCamp."

Michael Paglia,
Chief Technology Officer, ElevateBio BaseCamp

The results were immediate. Within four weeks, the teams completed their first training run. A second run followed within a month, and engineering runs began shortly after. This unprecedented speed came without sacrificing thoroughness – the teams transferred nine critical assays, completed comprehensive qualification, and maintained rigorous quality standards throughout.

Technology Innovation

The partnership demonstrated the value of close collaboration between therapy developers and manufacturing partners in establishing robust production processes. The team's experience in transferring and scaling complex cell therapy processes helped create effective workflows and quality control approaches that supported the successful manufacture of BEAM-101.

Results: Breaking New Ground

The partnership achieved several breakthrough milestones in manufacturing excellence. Most notably, the team completed technology transfer two months ahead of schedule – while maintaining exceptional quality standards. The manufacturing process demonstrated consistent editing efficiency and reliable production parameters, establishing a repeatable model for future programs.

Key achievements include:

1. Completion of comprehensive tech transfer ahead of schedule.
2. Achieving successful site-to-site comparability to Beam's in-house manufacturing facility
3. Proudly supplying patient doses for Beam’s BEACON Phase 1/2 clinical trial evaluating BEAM-101 in adult patients with sickle cell disease

Beyond these immediate results, the collaboration delivered strategic value that extends well beyond BEAM-101. By working with ElevateBio, Beam successfully demonstrated the manufacturing scale-up of its base editing technology – a critical milestone in proving the commercial viability of any novel therapeutic platform. This demonstration provides a blueprint for future development programs across Beam's pipeline, potentially accelerating the timeline for bringing additional base editing treatments to patients.

"The speed and quality of this collaboration exceeded our expectations," says Giuseppe Ciaramella, Ph.D., president of Beam. "BaseCamp didn't just manufacture our therapy – they helped us establish a blueprint for base editing manufacturing."

Giuseppe Ciaramella, Ph.D.
President, Beam Therapeutics

The successful manufacturing of BEAM-101 represents more than one company's achievement – it demonstrates the feasibility of rapidly scaling novel genetic medicines. As the field of genetic medicine advances, manufacturing remains a critical determinant of success. The manufacturing platform and processes developed through this collaboration create a pathway for future base editing therapies, potentially accelerating the development of treatments for other genetic diseases.