Beam Therapeutics Inc. is pioneering the field of precision genetic medicine through its innovative base editing technology. This approach allows for precise modifications at the DNA level, enabling the correction of genetic mutations that cause serious diseases. Beam's base editing applications are designed to address a variety of genetic disorders, including sickle cell disease, T-cell cancers, glycogen storage disease type 1a, and alpha-1 antitrypsin deficiency. The company employs both ex vivo and in vivo strategies for delivering its therapies, utilizing advanced methods such as electroporation and lipid nanoparticles to ensure effective and targeted treatment.

In addition to its core base editing technology, Beam Therapeutics has licensed complementary technologies to enhance its research and therapeutic capabilities. This includes RNA base editing systems for transient editing of the transcriptome and the Cas12b nuclease technology, which expands the range of genomic sites that can be targeted for editing. Furthermore, Beam has secured exclusive rights to prime editing technology, allowing for precise DNA sequence alterations without double-stranded breaks. Collectively, these technologies position Beam Therapeutics at the forefront of genetic medicine, with a robust pipeline aimed at addressing significant unmet medical needs through innovative therapeutic solutions.

United States

Beam Therapeutics Inc., a biotechnology company, engages in developing precision genetic medicines for patients suffering from serious diseases in the United States. The company is developing therapies for the development of sickle cell disease and beta-thalassemia; CAR-T cell therapies for pediatric T-cell acute lymphoblastic leukemia and pediatric acute myeloid leukemia; therapies for alpha-1 antitrypsin deficiency and glycogen storage disorder 1A; and therapies for ocular and central nervous system disorders. It has a research and clinical collaboration agreement with Magenta Therapeutics, Inc. Beam Therapeutics Inc. was founded in 2017 and is based in Cambridge, Massachusetts.

Cancer

Genetic Diseases

Hematologic Diseases

Rare diseases

Beam Therapeutics Inc

Beta thalessemia

Sickle cell disease

Beam has exclusively licensed the use of prime editing, an emerging gene editing technology that utilizes a reverse transcriptase to rewrite short sequences of DNA at a CRISPR-directed location, without causing a double-stranded break, from Prime Medicine in certain fields and for certain applications similar to those Beam is already pursuing with base editing. The license gives Beam the exclusive right to make any transition mutation (such as an A-to-G or C-to-T change) as well as to exploit any approach for the treatment of sickle cell disease using prime editing.
 
BEAM-101 reproduces single base changes seen in individuals with hereditary persistence of fetal hemoglobin, or HPFH. Individuals with sickle cell disease (SCD) or beta-thalassemia, who also have HPFH, typically experience a milder form of the disease or may be asymptomatic.
 
Using adenine base editors (ABEs), BEAM-101 recreates HPFH by installing base edits in the gamma globin gene promoters, HBG1 and HBG2, that disrupt repressor binding and lead to increased expression of gamma globin. Two theoretical types of off-target events that are possible as a consequence of these edits are guide-dependent and guide-independent deamination.
 
<a href="https://investors.beamtx.com/news-releases/news-release-details/beam-therapeutics-announces-first-development-candidates-sickle">source</a>
Gene editing

BEAM-101

Blood cancer

T-cell lymphoma

BEAM-102 is an investigational therapy designed to edit the causative hemoglobin S point mutation directly to recreate a naturally occurring normal human hemoglobin variant, HbG-Makassar. The Makassar variant is believed to be a normal- functioning variant of hemoglobin.
Gene editing

BEAM-201

Alpha-1 Antitrypsin Deficiency

BEAM-302

Gene & Cell therapies

Glycogen Storage Disease Type Ia

BEAM-301 is an investigational in vivo base editing medicine designed to directly correct the R83C mutation, one of the primary disease-causing mutations of glycogen storage disease type Ia (GSDIa).

Beam Therapeutics Inc

Company Details