Beam Therapeutics

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• Now, it’s adding another arrow to its drug delivery quiver using its $120 million acquisition of GuideTx, a Georgia Tech spinout.
• Our current base editor technology carries a cytosine base editor and an adenine base editor.
• RNA base editing could be useful for transient editing of the transcriptome.

Ahead of its IPO, the business raised nearly $1 billion in capital raising from investors.
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Beam Therapeutics Signals

Our delivery strategy is to set up a suite of clinically validated technologies, including electroporation and nonviral and viral delivery modalities.
BEAM-102 can be an investigational therapy made to edit the causative hemoglobin S point mutation right to recreate a naturally occurring normal human hemoglobin variant, HbG-Makassar.
The Makassar variant is believed to be a normal- functioning variant of hemoglobin.
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Their first integrated platform, named Ursula, is a next-generation sequencing platform, that may isolate, quantify, and normalize DNA from both cells and tissues, and also perform NGS Library Preparation.
Essentially, it can handle all you need to do for base or gene editing experimentation, short of loading samples onto sequencers.
To orchestrate this degree of automation, Beam’s automation team consists of a traditional automation and robotics team, along with lab informatics, cloud and software infrastructure, and NGS Core teams.
Beam Therapeutics has recently unveiled a fresh CRISPR base editing tool to focus on sickle cell mutations, a promising approach that holds substantial clinical value for patients with these blood disorders.

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Base Editing: Precision Chemistry On The Genome And Transcri

The business is joining forces with Apellis Pharmaceuticals to create new gene-editing treatments for complement-driven diseases, a broad bucket of ailments caused by an overactive part of the immune system.
Beam Therapeutics is an American biotechnology company conducting research in neuro-scientific gene therapies and genome editing.
In the development of therapies, the business relies on CRISPR and prime editing, whereby DNA sequences can be modified even more precisely compared to previous methods.
Beam Therapeutics will manufacture precision genetic medicines by using base editing technology at its RTP site.
BEAM-101 is an investigational therapy that produces base edits made to potentially alleviate the consequences of sickle cell disease by mimicking genetic variants observed in individuals who’ve hereditary persistence of fetal hemoglobin.
Not all gene editing treatments are created equal–that’s why Beam Therapeutics started out with multiple technologies from the likes of MIT, Harvard, the Broad Institute and Editas Medicine.

• Creating potential life-long cures for patients experiencing serious diseases.
• Employees rate Beam Therapeutics 4.5 out of 5 stars based on 24 anonymous reviews on Glassdoor.
• The accumulation of glycogen and inability to generate glucose during fasting can lead to severe and life-threatening hypoglycemia, seizures, metabolic dysfunction in lipids, short stature, liver adenoma, and renal disease.
• To address these limitations, we are using multiplex base editing to create donor-derived CAR-T cells by simultaneously silencing multiple target genes, thereby potentially enabling universal compatibility and resistance to host rejection, fratricide, and immunosuppression.
• Prior to its IPO, the business raised nearly $1 billion in capital raising from investors.

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Beam Therapeutics Inc. is an American biotechnology company conducting research in neuro-scientific gene therapies and genome editing.
In the development of therapies, the business depends on CRISPR and prime editing, whereby single nucleotides in a DNA sequence can be modified without cutting the DNA, theoretically reducing the probability of off-target effects in comparison to previous CRISPR-based methods.
Beam includes a growing portfolio of proprietary gene editing technologies, including base editing, prime editing, nuclease editing, and RNA editing.
Our current base editor technology includes a cytosine base editor and an adenine base editor.
Beam can be exploring the prospect of in vivo base editing programs for SCD, where base editors would be delivered to a patient through an infusion of lipid nanoparticles geared to HSCs, eliminating the need for transplantation.
This approach gets the potential to provide a far more accessible option for patients, particularly in regions where ex vivo treatment and transplantation aren’t widely accessible.

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Companies involved in the research, development, and commercialization of chemically- or biologically-derived therapeutic & theranostic drugs.
Feng Zhang joins the Broad and McGovern institutes and continues on to build up genome editing tools for used in eukaryotic cells from natural microbial CRISPR systems.