List of Gene Editing/CRISPR Companies with Phase 1 Active Clinical Trial - 29

AvenCell - Based on our deep science and focused on patients in need, we develop next generation immunotherapies for hard-to-treat cancers.

On August 20, 2024, Base Therapeutics announced that its AccuBase® base editor has officially received authorization for two patents from the United States Patent and Trademark Office (USPTO). This marks that both of Base Therapeutics' AccuBase® technologies have secured patent protection in China and overseas markets. Base Therapeutics' strategic positioning in the gene editing field is at the forefront internationally, making it one of the biopharmaceutical companies with the most authorizations domestically and abroad.

At CAMP4 Therapeutics, we are pioneering a novel approach to programmable therapeutics. We combine a deep understanding of regulatory RNA and gene expression with a complementary and customizable oligonucleotide modality. Our platform’s proprietary insights enable us to harness the power of RNA to upregulate the expression of genes and unlock the potential to create treatments for hundreds of diseases affecting millions of patients. Join us at the forefront.

Capstan Therapeutics is a biotechnology company dedicated to advancing in vivo cell reprogramming. The core platform technology comprises proprietary targeted lipid nanoparticles conjugated with a recombinant protein binder such as monoclonal antibodies (tLNPs) designed to deliver payloads, including mRNA or gene editing tools, that can reprogram specific cell types in vivo. The platform technology has the potential to generate transformative therapies with applications across a broad range of disease areas, including oncology, autoimmune disorders, fibrosis, and monogenic blood disorders.

Caribou Biosciences, Inc., a clinical-stage biopharmaceutical company, engages in the development of genome-edited allogeneic cell therapies for the treatment of hematologic malignancies and solid tumors in the United States and internationally. Its lead product candidates are CB-010, an allogeneic anti-CD19 CAR-T cell therapy that is in phase 1 clinical trial to treat relapsed or refractory B cell non-Hodgkin lymphoma; and CB-011, an allogeneic anti-BCMA CAR-T cell therapy for the treatment of relapsed or refractory multiple myeloma. The company also develops CB-012, an allogeneic anti-CD371 CAR-T cell therapy for the treatment of relapsed or refractory acute myeloid leukemia; and CB-020, an allogeneic CAR-NK cell therapy for the treatment of solid tumors. Caribou Biosciences has collaboration with AbbVie Manufacturing Management Unlimited Company to develop CAR-T cell therapies. The company was incorporated in 2011 and is headquartered in Berkeley, California.

Celyad Oncology is a cutting-edge biotechnology company dedicated to pioneering the discovery and advancement of revolutionary technologies for chimeric antigen receptor (CAR) T-cells. Its primary objective is to unlock the potential of its proprietary technology platforms and intellectual property, enabling to be at the forefront of developing next-generation CAR T-cell therapies. By fully leveraging its innovative technology platforms, Celyad Oncology aims to maximize the transformative impact of its candidate CAR T-cell therapies and redefine the future of CAR T-cell treatments. Celyad Oncology is based in Mont-Saint-Guibert, Belgium. For more information, please visit www.celyad.com.

Century Therapeutics, Inc., a biotechnology company, develops transformative allogeneic cell therapies for the treatment of solid tumor and hematological malignancies. The company’s lead product candidate is CNTY-101, an allogeneic, induced pluripotent stem cells (iPSCs)-derived chimeric antigen receptors (CAR)-iNK cell therapy targeting CD19 for relapsed, refractory B-cell lymphoma. It is also developing CNTY-103, a CAR-iNK candidate targeting CD133 + EGFR for recurrent glioblastoma; CNTY-102, a CAR-iT or CAR-iNK candidate targeting CD19 + CD79b for relapsed, refractory B-cell lymphoma and other B-cell malignancies; and CNTY-104, a CAR-iT or CAR-iNK multi-specific candidate for acute myeloid leukemia. Century Therapeutics, Inc. has a research collaboration with Outpace Bio, Inc. for CAR engineering. The company was founded in 2018 and is headquartered in Philadelphia, Pennsylvania.

CorrectSequence Therapeutics (Correctseq) is a clinical-stage biotech company employing its proprietary transformer base editing (tBE) technology to pioneer next-generation gene editing therapies. Our leading pipeline candidate, CS-101, utilizing innovative base editor targeting HBG, curing β-hemoglobinopathies, has obtained IND approval from the China NMPA. Clinical data demonstrates its superior performance, indicating significant potential for CS-101 to become a best-in-class gene editing therapy for curing patients with β-hemoglobinopathies. Proof-of-concept (POC) data in mice for in vivo pipelines using tBE-editing therapies via lipid nanoparticle (LNP) delivery are available, including targets for metabolic dysfunction and associated diseases. Ex vivo multiplex editing of T cells on multiple targets simultaneously preserved T cell growth and function in vivo compared to non-edited cells, establishing tBE as the ideal gene editing tool for the next-generation cell therapy development. tBE offers significant advantages in controlling off-target effects and enhancing in vivo editing efficiency, making it ideal for both multiplex editing and precise single-target editing. It is compatible with various delivery system, including ex vivo editing in hematopoietic stem cells (HSCs) and T cells, as well as in vivo editing via LNPs or adeno-associated viruses (AAVs). We are developing multiple pipeline programs targeting genetic diseases, metabolic disorders, and cardiovascular diseases. Our focus on biotechnology innovation, research and development underscores our commitment to providing efficient, reliable and safe solution for unmet medical needs.

CureVac (Nasdaq: CVAC) is a pioneering multinational biotech company founded in 2000 to advance the field of messenger RNA (mRNA) technology for application in human medicine. In more than two decades of developing, optimizing, and manufacturing this versatile biological molecule for medical purposes, CureVac has introduced and refined key underlying technologies that were essential to the production of mRNA vaccines against COVID-19, and is currently laying the groundwork for application of mRNA in new therapeutic areas of major unmet need. CureVac is leveraging mRNA technology, combined with advanced omics and computational tools, to design and develop off-the-shelf and personalized cancer vaccine product candidates. It also develops programs in prophylactic vaccines and in treatments that enable the human body to produce its own therapeutic proteins. Headquartered in Tübingen, Germany, CureVac also operates sites in the Netherlands, Belgium, Switzerland, and the U.S. Further information can be found at www.curevac.com.

Epigenic Therapeutics is a frontier biotechnology company dedicated to developing next generation gene editing therapy utilizing regulation of epigenetic genome for a variety of diseases. Founded in 2021 by leading scientists focused on discovering gene editing technologies and developing gene editing therapies, the company has multiple product candidates in the pipeline, including treatment for metabolic, cardiovascular, viral hepatitis, ocular and rare diseases.

Excision BioTherapeutics is a private company focused on developing advanced gene editing therapeutics to address the medical need for the treatment of life-threatening disease caused by viral infections. Our mission is to advance gene editing therapeutics for our indications into safe and efficacious medicines that will greatly improve the lives of infected individuals around the world.

Fate Therapeutics (Nasdaq: FATE) is a clinical-stage biopharmaceutical company dedicated to the development of next-generation cellular immunotherapies for cancer and immune disorders.

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GentiBio, Inc., is an early stage biotherapeutics company co-founded by pioneers in Treg biology and synthetic immunology to develop engineered regulatory T cells (EngTregs) programmed to treat autoimmune, alloimmune, autoinflammatory and allergic diseases. GentiBio's proprietary autologous and allogeneic EngTregs platform integrates key complimentary technologies needed to successfully restore immune tolerance and overcome major limitations in existing regulatory T-cell therapeutics. GentiBio is at the forefront of leveraging a unique therapeutic modality that can be used to address the fundamental cause of many diseases that result from overshooting and/or malfunctioning of the immune system.

GenVivo will make first- and best-in-class immune therapies that are immediately and easily deployable, highly effective, and which improve survival and quality of life beyond current therapies.

Hopstem Bioengineering Co., Ltd. was founded on January 2017 in Hangzhou, by neuroscientists and stem cell biologists from Johns Hopkins University. Hopstem has established world-leading iPSCs platform of neural differentiation and cell engineering. Hopstem has developed several critical patented technologies，as well as CMC platform for iPSC-derived cell therapy products. Hopstem has iPSC reprogramming patent, GMP iPSC line with global licensing rights, iPSC-derived cell product manufacturing and quality system, and a variety of cell therapy products and pipelines, targeting CNS diseases, optical diseases and tumor. Hopstem’s first clinical product - neural progenitor cell hNPC01 targets neural injury diseases such as stroke and traumatic brain injury, has received China's IND approval and entered clinical trials. Hopstem is committed to translating the most cutting-edge technologies to provide global patients with safe, efficient and affordable cell therapy products.

iCell Gene Therapeutics is developing Chimeric Antigen Receptor (CAR) engineered cells with improved functional properties and expanded applicability. We are a clinical stage company focused to demonstrate clinical proof of concept for unique, patent protected, targets and strategies. Our pipeline includes multiple additional first in man CAR strategies to target hematological cancers, non-hematological cancers and autoimmune disorders.

Inari is the SEEDesign™ company, using new breeding technology to push the boundaries of what is possible by designing nature-positive seeds for a more sustainable global food system. A combination of AI-powered predictive design and a pioneered multiplex gene editing toolbox enables us to unlock the full potential of seed and advance critical solutions with broad applications for growing more food with fewer resources.

OUR MISSION: Deliver Innovation. Empower Discovery. Improve Life. For over 25 years, Mirus Bio has pioneered the development of transfection reagents and proprietary technologies for nucleic acid delivery applications. Our work is both technical and cutting-edge, and we promote a culture of creativity, responsibility, and respect. Our team members are engaged and motivated by the desire to support our customers and partners to achieve strong results. Mirus, together with Gamma Biosciences, is establishing the future of science and medicine by developing products that enable product developers within the cell and gene therapy space. As we approach a new era of addressing and conquering life-altering diseases, we will continue to expand our expertise and leadership in transfection to be integral in the future of advanced therapies.

MOMA Therapeutics is a clinical-stage, next-generation precision medicine company dedicated to targeting highly dynamic proteins that underlie human disease via a small molecule approach, utilizing its proprietary KNOMATIC platform. The platform was designed to exploit key vulnerabilities inherent to all dynamic proteins, namely their dependence on well-coordinated, stepwise changes in protein conformation. By focusing on genetically validated targets with high translation potential MOMA is rapidly advancing its pipeline toward responses in the clinic. In January 2024, MOMA announced a five-year discovery collaboration with Roche focused on critical cancer dependencies. We are unapologetically scientific, and we know this will help us achieve our mission to get new medicines to people in need. We’ve removed the bureaucracy and believe having a teammate’s back is critical to our success. You see, MOMA isn’t your typical biotech company. We are drug discovery professionals, industry specialists and scientific experts. More importantly, we’re collectively unstoppable.

Our mission at Poseida Therapeutics is to create cell and gene therapies with the capacity to cure. We are developing novel products using our broad gene engineering platform technologies, with the goal of bringing new and better treatments to patients with serious diseases, first in the blood-based cancer Multiple Myeloma and next in solid tumors like prostate cancer.

Precision BioSciences, Inc. (Nasdaq: DTIL), is a clinical stage biotechnology company dedicated to improving life (DTIL) with its novel and proprietary ARCUS® genome editing platform. ARCUS is a highly specific and versatile genome editing platform designed with therapeutic safety, delivery, and control in mind. Using ARCUS, Precision is developing allogeneic CAR T and in vivo gene correction therapies for cancer and infectious diseases.

At Promega Corporation, creativity and connection drive discovery and innovation. We celebrate scientific discovery and the creative application of science to solve problems. Interactions with our customers, partners and vendors are central to everything we do, and we value those relationships. We seek out and build connections across the globe and in our local communities because creative problem solving requires a network of diverse ideas and viewpoints. We develop and evolve technologies and instrumentation in collaboration with scientists and researchers around the globe. From simple cloning vectors used to address fundamental questions in biology to bioluminescent assays that help us monitor food safety and water quality, we work side-by-side with our partners in academic, industrial and government institutions to bring the best minds to bear on the toughest questions. Since 1978, we have done this all with a steadfast commitment to sustainable business models that protect our environment, inspire and develop our employees as individuals, support the wellbeing and growth of our communities, and strengthen our relationships with our customers. With 16 branches around the world, Promega products are used by a global community of life scientists who are asking fundamental questions about biological processes as well as by scientists who are applying scientific knowledge to diagnose and treat diseases, discover new therapeutics, and use genetics and DNA testing for human identification.

Sana Biotechnology is focused on utilizing engineered cells as medicines for patients. The ability to modify genes and use cells as medicines will be one of the most important advances in healthcare over the next several decades. Sana is building differentiated capabilities across the spectrum of cell and gene therapy. Three aspirations drive Sana as we look to discover treatments for patients with poor outcomes or currently untreatable diseases. The first is the ability to repair and control the genes in any cell in the body. We are advancing novel delivery technologies with the goal of being able to deliver any payload to any cell in a specific, predictable, and repeatable manner, paving the way for next-generation in vivo gene therapy. Next is the ability to differentiate pluripotent stem cells ex vivo into immune-cloaked functional cells with the aspiration of being able to replace any missing or damaged cells in the body. Last is a belief we can enable broader access to our therapies through focusing on scalable manufacturing solutions, the cost of manufacturing, and aligning with key stakeholders. Sana launched in early 2019 and has over 350 employees in Seattle, Cambridge, and South San Francisco. Sana was founded with the long view – bringing together great people and the best technologies to deliver on the challenging promise of using engineered cells to meaningfully change the outcome of many human diseases.

Synbio Technologies aims to become the most trusted provider of DNA solutions that empower scientific discoveries by providing highly-accurate and cost-effective synthetic biology services and products for researchers around the world. We have developed a full range of DNA reading (sequencing), DNA writing (synthesis), and DNA editing (engineering) capabilities for various applications, including diagnostic DNA probes, precision medicine, protein production, antibody discovery, vaccine development, novel enzymes, molecular breeding, biofuel implication, and more. Our scientific capabilities cover all facets of DNA synthesis and engineering, offering a full range of support for molecular biology work, including but not limited to, oligo synthesis, gene synthesis, subcloning & PCR cloning, plasmid preparation, mutagenesis, DNA variant libraries, CRISPR sgRNA, protein expression and purification in multiple systems. Relying on our strong DNA sequencing, synthesis, engineering technology platform, and expertise in bioinformatics analysis, we have developed proprietary algorithms to facilitate and expedite the antibody discovery process. Our antibody discovery services include hybridoma sequencing, immune repertoire sequencing, antibody design and production, AI-guided heavy chain and light chain pairing, antibody humanization, affinity maturation, and sdAb.

The vast majority of human disease is governed by the epigenome, the subtle machinery that controls the volume of gene expression in cells. Tune Therapeutics is pioneering the creation of epi-therapeutic medicines with its powerful and precise epigenomic control platform, TEMPO. Unlike genome editing, TEMPO dials the volume of gene expression up or down towards healthy levels, without breaking or permanently rewriting DNA – and it can do this in multiple genes at once. This genetic tuning approach allows us to change cell fate and function at will, unlocking the ability to reverse pathways of cancer, genetic disease and aging. Our approach effectively expands the potential reach of genetic medicine – from targeting a limited range of rare conditions, to overcoming thousands of complex and chronic diseases that currently have no curative treatment.

Verve Therapeutics, Inc., a genetic medicines company, engages in developing gene editing medicines for patients to treat cardiovascular diseases. Its lead product candidate is VERVE-101, a single-course gene editing treatment that permanently turns off the PCSK9 gene in the liver. The company also engages in the development ANGPTL3 program to permanently turn off the ANGPTL3 gene in the liver. It has a collaboration and license agreement with Beam Therapeutics; a development and option agreement with Acuitas Therapeutics, Inc.; and a Cas9 license agreement with The Broad Institute and the President and Fellows of Harvard College. The company was formerly known as Endcadia, Inc. and changed its name to Verve Therapeutics, Inc. in January 2019. Verve Therapeutics, Inc. was incorporated in 2018 and is based in Cambridge, Massachusetts.

Vittoria Biotherapeutics is committed to unlocking the promise of cell therapies while addressing their inherent challenges by strengthening efficacy, improving safety, and broadening therapeutic applicability for patients with difficult-to-treat diseases. An Important Note About Privacy: Vittoria Biotherapeutics is committed to your privacy and will not ask for sensitive personal information such as social security number, date of birth, or bank account details via email, job recruiting, or social media platforms. We do not hire virtually or solicit/interview candidates on Indeed, Telegram, Wire, Google Hangouts or similar public platforms. Any questions about career opportunities can be directed to info@vittoriabio.com.