Gene editing stocks

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.

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.

Beam Therapeutics Inc., founded in 2017 and based in Cambridge, Massachusetts, operates as a pioneering biopharmaceutical firm. Its core mission involves engineering precise genetic remedies to tackle a spectrum of severe human ailments, primarily within the United States. The company's developmental portfolio features several key candidates: BEAM-101 is being advanced to treat both sickle cell disease and beta thalassemia. BEAM-102 is specifically designed for addressing sickle cell disease. BEAM-201, an allogeneic chimeric antigen receptor T-cell therapy, is under investigation for individuals suffering from relapsed or refractory T-cell acute lymphoblastic leukemia. BEAM-301 is a liver-targeted candidate aimed at patients afflicted with Glycogen Storage Disease Type Ia. Beyond these flagship programs, Beam Therapeutics is also engaged in devising treatments for alpha-1 antitrypsin deficiency, various ocular conditions, and other disorders impacting the liver, muscular system, and central nervous system. To further its research and development objectives, Beam Therapeutics has forged numerous strategic alliances and partnerships. These include collaborations with Boston Children's Hospital; a research and clinical trial agreement with Magenta Therapeutics, Inc.; a licensing deal with Sana Biotechnology, Inc.; and a research partnership with the Institute of Molecular and Clinical Ophthalmology Basel. Additionally, the company maintains research collaborations with both Pfizer Inc. and Apellis Pharmaceuticals, Inc., alongside a comprehensive collaboration and license agreement with Verve Therapeutics, Inc.

Beam's platform is focused on permanent genetic editing.

Prime Medicine, Inc. operates as a biotechnology firm, dedicated to delivering genetic therapies for various diseases by strategically employing advanced gene editing technology. Central to their platform is the "Prime Editor" system, which combines a unique Prime Editor protein—a hybrid composed of a Cas protein and a reverse transcriptase enzyme—with a specialized pegRNA molecule. This pegRNA plays a crucial role by both guiding the Prime Editor to a precise location within the genome and providing the essential template for introducing the desired modification to the target DNA sequence. The company, founded in 2019, maintains its headquarters in Cambridge, Massachusetts.

FDA clearance of PM647’s IND is directly positive for Prime Medicine because it permits the company to move its one-time in vivo prime-editing treatment for AATD into U.S. human studies, creating a new clinical-development milestone and increasing the probability of future value from the program.

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.

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.

Scribe Therapeutics is a clinical-stage biotechnology company focused on developing in vivo CRISPR-based genetic medicines. The company engineers proprietary technologies to target the genetic drivers of disease, with an initial focus on atherosclerotic cardiovascular disease (ASCVD), aiming to shift treatment from chronic care to preventative intervention.

Korro Bio, Inc., a biopharmaceutical firm based in Cambridge, Massachusetts, is dedicated to exploring, advancing, and bringing to market genetic therapeutics. These treatments are formulated through RNA editing technology, aiming to address a wide array of medical conditions, encompassing both uncommon and widespread diseases.

Korro is the direct developer of KRRO-121, a GalNAc-conjugated RNA-editing oligonucleotide intended to treat hyperammonemia in UCDs and HE.

Wave Life Sciences Ltd., a clinical-stage biotechnology company, designs, develops, and commercializes ribonucleic acid (RNA) medicines through PRISM, a discovery and drug development platform. The company’s medicines platform, PRISM combines multiple modalities, chemistry innovation, and deep insights into human genetics to deliver scientific breakthroughs that treat both rare and prevalent disorders. It is developing WVE-006, a GalNAc-conjugated RNA editing oligonucleotide for the treatment of alpha-1 antitrypsin deficiency; WVE-007, a GalNAc-conjugated small interfering RNA designed to silence INHBE mRNA targeting obesity; WVE-008, a GalNAc-conjugated RNA editing oligonucleotide for the treatment of liver disease; WVE-N531, an exon splicing oligonucleotide for the treatment of Duchenne muscular dystrophy; and WVE-003, an allele-selective oligonucleotide for the treatment of Huntington’s disease (HD). The company has collaboration agreements with GlaxoSmithKline for the research, development, and commercialization of oligonucleotide therapeutics; Takeda Pharmaceutical Company Limited for the research, development, and commercialization of oligonucleotide therapeutics for disorders of the Central Nervous System; and Asuragen, Inc. for the development and potential commercialization of companion diagnostics for investigational allele-selective therapeutic programs targeting HD. Wave Life Sciences Ltd. was founded in 2012 and is based in Singapore.

ProQR uses the Axiomer platform for targeted RNA editing.

ToolGen Incorporated is a biotechnology enterprise based in South Korea, primarily focused on pioneering advancements in genome editing technology. The company's main endeavor involves engineering CRISPR-Cas9 genetic modification tools specifically designed for editing genes in eukaryotic cells. Furthermore, its operations extend to commercializing its intellectual property, exploring therapeutic applications, and participating in the seed sector. Established in 1999, ToolGen's corporate headquarters are situated in Seoul, South Korea.

Cibus, Inc. is an agricultural biotechnology company that develops and licenses gene-edited plant traits. The company's products enable farmers to achieve higher yields and reduce the use of chemicals, such as fungicides, insecticides and fertilizers, and offer sustainable ingredients. it has patented core technology platform, RTDS, a scalable, standardized, end-to-end, semi-automated and high-throughput gene-editing system marketed under the Trait Machine brand name. The company was formerly known as Calyxt, Inc. and changed its name to Cibus, Inc. in June 2023. Cibus, Inc. was incorporated in 2010 and is headquartered in San Diego, California.

Cibus has a direct agricultural genome-editing and external-client technology linkage that could connect to seed-company licensing demand.

Evogene has a direct agricultural genome-editing platform linkage and serves external agricultural clients.

Evogene operates an agricultural technology platform serving external clients. A standalone Vylor focused on gene-edited traits, genomics, and licensing may expand the addressable market for external technology partnerships, but the retrieved evidence does not establish a direct commercial relationship.

Operating as an investment holding company, Genscript Biotech Corporation, established in 2002 and headquartered in Nanjing, People's Republic of China, specializes in the global provision of innovative life science research solutions, encompassing both products and services. Its market presence spans North America, Europe, China, Japan, other Asia Pacific regions, and numerous international markets. The enterprise structures its operations into four key divisions: The Life Science Services and Products segment delivers a wide array of research offerings, such as gene and oligonucleotide synthesis, molecular cloning, protein and peptide engineering, antibody production, molecular diagnostic instruments, and genomic editing materials. These resources are instrumental for fundamental biological investigations, pharmaceutical and drug discovery, disease diagnostics, vaccine development, and applications in agriculture, environmental science, and food production. The Biologics Development Services division supports pharmaceutical, biotechnology, governmental, and academic institutions. It provides comprehensive services for the full lifecycle of therapeutic antibodies, from initial discovery through pre-clinical and clinical development, alongside pre-clinical and clinical development for plasmids and viruses, and broader gene and cell therapy development. Dedicated to industrial applications, the Industrial Synthetic Biology Products segment engineers non-pathogenic microbial strains and manufactures specialized enzymes. These enzymes find utility across the food processing, animal feed, pharmaceutical, and chemical sectors. Finally, the Cell Therapy unit focuses on the pioneering discovery and advanced development of chimeric antigen receptor T-cell (CAR-T) therapies, specifically designed to combat both liquid and solid malignant tumors.

Nanjing Vazyme Biotech Co., Ltd., a prominent biotechnology firm, specializes in the comprehensive investigation, development, and manufacturing of essential biological components. These core offerings include enzymes, antigens, antibodies, and unique polymer organic compounds. The company's extensive product portfolio encompasses a broad spectrum of scientific research reagents. This range features solutions for advanced techniques such as PCR cloning and point mutation, nucleic acid extraction, reverse transcription, quantitative PCR (qPCR), cell and protein analysis, gene editing applications, and user-friendly instant granule formulations. Furthermore, Vazyme provides sophisticated reagents crucial for high-throughput sequencing library construction, including those for DNA and RNA library preparation, single-cell epigenetics, magnetic bead-based methodologies, quantitative assays, and the effective storage and extraction of cell-free DNA. Complementing these, the company also delivers a variety of biomedical reagents and related services. Vazyme's diverse clientele spans scientific research institutions, specialized high-throughput sequencing service providers, manufacturers of molecular diagnostic reagents, pharmaceutical enterprises, contract research organizations (CROs), hospitals, and other healthcare facilities. Established in 2012, the company maintains its headquarters in Nanjing, China.

TFBS Bioscience Inc. functions as a contract research organization (CRO), specializing in offering biological analysis and testing services throughout Taiwan. The company plays a crucial role in supporting advanced medical innovations by fabricating viral vectors, which are essential for cell and gene therapy products. Furthermore, TFBS Bioscience conducts comprehensive biosafety and final product release evaluations for a diverse range of biopharmaceutical items, including cell and gene therapies, vaccines, and other biologics. Established in 2016, the firm's main operational base is located in New Taipei City, Taiwan.

Oxford Biomedica plc is a biopharmaceutical firm focused on the discovery, advancement, and sophisticated bioprocessing of innovative cell and gene therapies. Operating globally, with a strong presence across Europe, the company organizes its activities into two segments: Platform and Product. Its proprietary LentiVector platform technology facilitates the integration of genes into non-dividing cells, such as neurological cells in the brain and retinal cells in the eye, and can incorporate diverse therapeutic genes. The company's developmental pipeline includes promising candidates like Axo-Lenti-PD, a gene-based treatment for Parkinson's disease. Other pre-clinical stage programs feature OXB-302, targeting haematological tumors, and OXB-401, OXB-40X, and OXB-40Y, all addressing liver indications. Oxford Biomedica maintains numerous strategic alliances with prominent pharmaceutical entities such as Novartis, Bristol Myers Squibb, Sio Gene Therapies, Orchard Therapeutics, Boehringer Ingelheim, the UK Cystic Fibrosis Gene Therapy Consortium, Immatics, Arcellx, and the Vaccines Manufacturing and Innovation Centre. Additionally, it engages in research collaborations, including a partnership with Circularis Biotechnologies to identify novel tissue-specific promoters for in vivo lentiviral gene therapy, Virica Biotech for scaling viral medicines, Isolere Bio for enhancing downstream manufacturing efficiencies of biologics, and BiologIC Technologies for utilizing a novel biocomputer system in viral vector development. Founded in 1995, Oxford Biomedica plc is headquartered in Oxford, United Kingdom.

Oxford Biomedica is directly identified as the CDMO that manufactured materials supporting GeoVax’s Phase 3 program, giving it the strongest evidenced service-capture pathway among the listed suppliers.

Charles River supplies AAV and plasmid products for clinical and commercial gene-therapy programs.

Charles River provides lentiviral-vector and gene-modified cell-therapy manufacturing services. If Mankind funds ACTx through clinical development, demand for outsourced process development, GMP vector production, testing, and clinical supply could increase, although no ACTx-specific Charles River contract is disclosed.

Lonza operates cGMP manufacturing capabilities for autologous and allogeneic cell therapies and viral vectors, creating direct outsourced-manufacturing optionality.

Clinical advancement of an in vivo prime-editing therapy can increase future demand for qualified LNP process development, cGMP manufacturing, and fill-finish capacity. Lonza has relevant capabilities, but no PM647-specific supply mandate is evidenced.