Our ResearchClinical Trials
An important part of the drug development process is recognizing and validating what matters to patients. To understand their needs, we collaborate with clinical experts, patient advocacy organizations, and disease communities to optimize our clinical trial design and the participant experience.
We believe that patients, their families, and advocates provide unique and important perspectives on the development of potential new treatments. At Beam Therapeutics, we are bringing these voices into the drug development process.
- We take a patient-centric approach to the study endpoints in our clinical trials through
assessments in the literature and discussions with advocacy groups about what is meaningful to patients - We listen to and learn from patients about their disease burden and unmet needs. Patients
articulate their experience in a way that no one else can, because they live it day to day
Taking part in a clinical trial
What is a clinical trial?
- A clinical trial is a research study that looks at new ways to prevent, detect, or treat disease
- Research studies are necessary to learn whether a new treatment is safe and effective
What is informed consent?
- Informed consent is a process in which trial participants are given information about a trial so that they can decide whether to participate
- Trial site personnel review the informed consent process with trial participants in detail so that they understand the trial and its possible risks and benefits
Clinical trials at Beam Therapeutics
Once we open enrollment in a clinical trial, information about the trial, including the eligibility criteria, will be posted at www.clinicaltrials.gov so that patients and their doctors can learn whether they are eligible to participate.
Our Trials
BEAM-101
Sickle Cell Disease
BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease (BEACON)
This is an open-label, single-arm, multicenter, Phase 1/2 study evaluating the safety and efficacy of the administration of autologous base-edited CD34+ HSPCs (BEAM-101) in patients with severe SCD.
BEAM-302
Alpha-1
A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)
This is a Phase 1/2, multicenter, open-label, dose-exploration (Phase 1) and dose-expansion (Phase 2) study to evaluate the safety, tolerability, PK/PD, and efficacy of BEAM-302 in adult patients with AATD-associated lung disease and/or liver disease and to determine the optimal biological dose (OBD).
BEAM-301
GSDIa
A Phase 1/​2, Dose-Exploration Study to Evaluate the Safety and Efficacy of BEAM-301 in Patients With Glycogen Storage Disease Type Ia (GSDIa)
This is a Phase 1/2, multicenter, open-label, single-ascending-dose study to evaluate the safety, tolerability, and efficacy of BEAM-301 in adult patients with GSDIa homozygous or compound heterozygous for the G6PC1 c.247C>T (p.R83C) variant and to determine the optimal biological dose.
Contact [email protected] with questions about Beam’s clinical trials.
Learn More
Our Science
Beam is a values-driven organization with a vision of delivering life-long cures to patients suffering from serious diseases. Â
Our Pipeline
We believe leveraging ex vivo and in vivo approaches, different delivery modalities, and multiple editing approaches will help us target a broad range of diseases with limited or no disease-modifying treatment options.
Our Disease Areas
We seek to potentially cure serious diseases and address significant unmet medical need so that people can reach their full potential. We believe leveraging ex vivo and in vivo approaches, different delivery modalities, and multiple editing approaches will help us target a broad range of diseases with limited or no disease-modifying treatment options.Â
Resources*
American Society of Gene and Cell Therapy
The Clinical Trial Process:
patienteducation.asgct.org/gene-therapy-101/clinical-trials-process.
Global Genes
Participating In A Clinical Trial: A Step-by-Step Guide for Rare Disease Patients and Families:
globalgenes.org/toolkit/participating-in-a-clinical-trial-a-step-by-step-guide-for-rare-disease-patients-and-families/
National Institutes of Health
Clinical Research Trials and You:
www.nih.gov/health-information/nih-clinical-research-trials-you
National Institutes of Health Clinical Center
Learn about clinical studies
clinicaltrials.gov/ct2/about-studies/learn
U.S. Food and Drug Administration
Development & approval process (drugs)
fda.gov/Drugs/DevelopmentApprovalProcess
Everyday Life Foundation for Rare Disease
Guide to Patient Involvement in Rare Disease Therapy Development
Guide-to-Patient-Involvement-FINAL-COMPLETE-GUIDE-Rev.pdf
*Â These resources are for information only. Inclusion of these resources does not indicate endorsement by Beam Therapeutics of an organization or its communications. The list is not intended as a comprehensive list of resources, nor are the resources intended to provide medical advice.