Beam Therapeutics Inc. competitors & alternatives

Biotech

CRISPR Therapeutics AG (CRSP) is a biotechnology firm dedicated to pioneering gene-based medicines for severe diseases. The company achieves this through its exclusive Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) technology, which enables precise and targeted alterations to an organism's genetic code. Its extensive pipeline includes therapeutic candidates spanning multiple medical areas, such as blood disorders (hemoglobinopathies), various cancers (oncology), regenerative medicine, and rare conditions. The company's flagship investigational therapy is CTX001, an ex vivo CRISPR gene-edited treatment. This therapy aims to benefit patients with transfusion-dependent beta-thalassemia or severe sickle cell disease by modifying their own hematopoietic stem cells to markedly boost the production of fetal hemoglobin within red blood cells. CRISPR Therapeutics is also advancing several other genetically engineered allogeneic (donor-sourced) CAR-T investigational therapies: CTX110, designed to combat cluster of differentiation 19-positive malignancies; CTX120, which targets B-cell maturation antigen for multiple myeloma that has relapsed or proven resistant to previous treatments; and CTX130, focused on Cluster of Differentiation 70 for a spectrum of solid tumors and blood cancers. Furthermore, the company is developing VCTX210, an immune-evasive, gene-edited stem cell-derived product candidate for treating type 1 diabetes. It is also pursuing various in vivo gene-editing initiatives aimed at addressing disorders affecting the liver, lungs, muscles, and central nervous system. The company has forged strategic alliances with significant partners, including Bayer Healthcare LLC, Vertex Pharmaceuticals Incorporated, ViaCyte, Inc., Nkarta, Inc., and Capsida Biotherapeutics. CRISPR Therapeutics AG was established in 2013 and maintains its headquarters in Zug, Switzerland.

Biotech

Intellia Therapeutics, Inc. is a biotechnology firm dedicated to advancing therapeutic treatments through its expertise in genome editing. The company's pipeline includes several in vivo (administered within the body) programs. NTLA-2001 is currently undergoing a Phase 1 clinical trial for transthyretin amyloidosis, while NTLA-2002 targets hereditary angioedema. Additionally, Intellia is developing various other liver-focused therapies for conditions such as hemophilia A and B, hyperoxaluria Type 1, and alpha-1 antitrypsin deficiency. Its ex vivo (processed outside the body) pipeline features NTLA-5001, a candidate for acute myeloid leukemia. The company is also progressing proprietary programs focused on creating engineered cell therapies to address diverse oncological and autoimmune disorders. At the core of Intellia's technology is the Clustered, Regularly Interspaced Short Palindromic Repeats/CRISPR associated 9 (CRISPR/Cas9) system. Intellia Therapeutics, Inc. has established multiple licensing and collaboration agreements. These include a partnership with Novartis Institutes for BioMedical Research, Inc. to engineer hematopoietic stem cells for treating sickle cell disease, and a co-development agreement with Regeneron Pharmaceuticals, Inc. for potential products against hemophilia A and B. The company also collaborates with Ospedale San Raffaele and holds a strategic alliance with SparingVision SAS to develop novel genomic medicines for ocular diseases, leveraging CRISPR/Cas9 technology. The company, initially known as AZRN, Inc., was founded in 2014 and operates from its headquarters in Cambridge, Massachusetts.

Beacon TherapeuticsBiotechnology

75% match

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Beacon Therapeutics is a clinical-stage biotechnology company dedicated to saving and restoring sight for people living with rare and prevalent ocular diseases. The company works to deliver advanced retinal gene therapies to patients experiencing blindness.

Similar biotechnology company

Base Therapeutics is a biotechnology company that develops next-generation gene editing technologies, including its proprietary AccuBase base editing system and enhanced prime editing technology. The company focuses on creating therapeutic solutions for cancer and genetic diseases, alongside engineered immune cell therapies such as BASE-NK and CAR-T products. It serves patients and partners with pharmaceutical and agricultural enterprises by licensing its precision gene editing tools.

Verve Therapeutics, Inc.BiotechnologyUnited States

72% matchPrivate

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Verve Therapeutics, Inc. is a biotechnology company headquartered in Boston, Massachusetts, dedicated to pioneering single-course gene editing medicines for cardiovascular disease.

Biotech

Editas Medicine, Inc., a clinical stage genome editing company, focuses on developing transformative genomic medicines to treat a range of serious diseases. The company develops a proprietary gene editing platform based on CRISPR technology. Its lead program is EDIT-401, a one-time therapy designed to reduce LDL cholesterol through the upregulation of the LDL receptor to treat hyperlipidemia. The company also develops therapies to treat Sickle cell disease and transfusion-dependent beta thalassemia; and in vivo gene editing medicines indicated for other cells and tissues. It has a research collaboration with Juno Therapeutics, Inc. to develop alpha-beta T-cell experimental medicines for the treatment of solid and liquid tumors, and autoimmune disease. The company was formerly known as Gengine, Inc. and changed its name to Editas Medicine, Inc. in November 2013. Editas Medicine, Inc. was incorporated in 2013 and is based in Cambridge, Massachusetts.

Similar biotechnology company

Prime Medicine, Inc. is an American biotechnology company developing gene therapies based on prime editing. The company is headquartered in Cambridge, Massachusetts.

Biotech

Voyager Therapeutics, Inc. operates as a gene therapy company, concentrating its efforts on developing innovative treatments and pioneering advanced platform technologies. Its leading clinical asset, VY-AADC, is presently undergoing an open-label Phase 1 clinical trial for the management of Parkinson's disease. The company's preclinical portfolio is extensive, featuring VY-SOD102 for amyotrophic lateral sclerosis (ALS), VY-HTT01 for Huntington's disease, and VY-FXN01 for Friedreich's ataxia. Additionally, Voyager is pursuing a Tau program aimed at various tauopathies, including Alzheimer's disease, progressive supranuclear palsy, and frontotemporal dementia, alongside initiatives for spinal muscular atrophy. To advance its gene therapy product pipeline, the company has forged strategic collaboration and licensing agreements with key industry players such as Neurocrine Biosciences, Inc., Pfizer Inc., and Novartis Pharma, A.G. These partnerships cover the research, development, and commercialization of adeno-associated virus-based gene therapy products. Voyager Therapeutics, Inc. was founded in 2013 and maintains its corporate headquarters in Cambridge, Massachusetts.

Genetix BiotherapeuticsBiotech

70% match

Biotech

Genetix Biotherapeutics develops and delivers one-time, curative genetic therapies for patients suffering from rare, severe genetic diseases such as sickle cell disease, beta-thalassemia, and cerebral adrenoleukodystrophy. The company utilizes patients' own blood stem cells, modifying them outside the body to correct underlying genetic defects and restore normal function. It serves patients, families, and healthcare providers by providing these advanced treatments alongside dedicated clinical support and research programs.

Biotech

Metagenomi, Inc. operates as a biotechnology company focused on gene editing, committed to developing therapeutic solutions for patients. The company utilizes a distinctive genome editing toolkit, sourced from metagenomics, for its development efforts within the United States. This advanced toolkit comprises programmable nucleases, base editors, and sophisticated RNA and DNA-mediated integration systems, notably prime editing and CRISPR-associated transposases. Metagenomi has established several key collaborations: a strategic alliance with ModernaTX, Inc. to explore new genome editing systems for in vivo human therapeutic applications; a development and licensing agreement with Affini-T Therapeutics, Inc. to create and commercialize gene-edited T-cell receptor therapies for human cancer; and a partnership with Ionis Pharmaceuticals, Inc. to research, develop, and market investigational medicines leveraging genome editing technologies. Founded in 2016, the company is based in Emeryville, California.

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Precision BioSciences, Inc. is a clinical-stage biotechnology firm based in the United States, specializing in the creation of both in vivo gene editing solutions and ex vivo allogeneic CAR T-cell therapies. At its core is ARCUS, a proprietary genome editing platform designed to address and potentially cure genetic diseases. The company's portfolio also encompasses Ex vivo Allogeneic CAR T Immunotherapy, an innovative approach where specific immune cells, known as T-cells, are genetically modified outside the body to precisely identify and eliminate cancer cells. Among its prominent therapeutic candidates are: PBCAR0191, currently undergoing Phase 1/2a clinical trials for adult patients battling relapsed/refractory (R/R) non-Hodgkin lymphoma or R/R B-cell precursor acute lymphoblastic leukemia (B-ALL). PBCAR19B, an anti-CD19 CAR T candidate leveraging a "stealth cell" platform through a single-step gene edit, engineered to reduce the likelihood of chromosomal abnormalities. PBCAR269A, an investigational allogeneic CAR T immunotherapy formulated to target BCMA, intended for the treatment of R/R multiple myeloma. Precision BioSciences has strategically formed several collaborative alliances. These include an agreement with Les Laboratoires Servier for the development and commercialization of allogeneic chimeric antigen receptor T-cell therapies, aimed at various antigen targets, including hematological cancers beyond CD19, and solid tumors. The company is also partnering with Tiziana Life Sciences to assess foralumab, a fully human anti-CD3 monoclonal antibody, for its utility as a lymphodepleting agent in potential cancer treatments. Furthermore, a collaboration with iECURE, Inc. focuses on advancing ARCUS-based gene editing therapies. Established in 2006, Precision BioSciences, Inc. maintains its corporate headquarters in Durham, North Carolina.

Biotech

Allogene Therapeutics, Inc. operates as a clinical-stage immuno-oncology firm dedicated to the creation and commercialization of genetically engineered allogeneic T-cell therapies for the treatment of various cancers. A pivotal product in their development pipeline is UCART19, an allogeneic chimeric antigen receptor (CAR) T-cell therapy. This candidate is being developed, manufactured, and prepared for market release to address relapsed/refractory (R/R) CD19-positive B-cell acute lymphoblastic leukemia (ALL) in both children and adults. The company's portfolio also encompasses ALLO-501, an anti-CD19 allogeneic CAR T-cell candidate currently in Phase I clinical trials for R/R non-Hodgkin lymphoma. A related therapeutic, ALLO-501A, is progressing through Phase I/II studies, targeting R/R large B-cell lymphoma or transformed follicular lymphoma. Furthermore, Allogene is advancing several other promising candidates: ALLO-715, an allogeneic CAR T-cell therapy in Phase I for R/R multiple myeloma; ALLO-605, another allogeneic CAR T-cell designed for multiple myeloma; and ALLO-647, an anti-CD52 monoclonal antibody. Their investigational therapies also include those targeting CD70 for renal cell cancer, ALLO-819 (an allogeneic CAR T-cell treatment) for acute myeloid leukemia, and DLL3 for small cell lung cancer and other aggressive neuroendocrine tumors. Allogene maintains a robust network of strategic alliances, which includes licensing and collaboration agreements with organizations such as Pfizer Inc., Servier, Cellectis S.A., and Notch Therapeutics Inc. They also hold a clinical trial collaboration with SpringWorks Therapeutics, Inc. Additionally, a strategic partnership with The University of Texas MD Anderson Cancer Center supports the preclinical and clinical evaluation of their allogeneic CAR T-cell product pipeline. Founded in 2017, the company's corporate headquarters are located in South San Francisco, California.