List of Gene Editing/CRISPR Companies with Phase 3 Active Clinical Trial - 16

BASF Corporation is the largest affiliate of BASF SE and the second largest producer and marketer of chemicals and related products in North America. BASF creates chemistry for a sustainable future. They combine economic success with environmental protection and social responsibility.BASF Corporation operates more than 100 production and research and development sites throughout North America and operates Verbund sites in Geismar, Louisiana and Freeport, Texas. At Verbund sites, production plants, energy and material flows, logistics, and site infrastructure are all integrated.

BioNTech is a global next generation immunotherapy company aspiring to translate science into survival. Around 6.100 pioneers currently work with us, employing a wide array of computational discovery and therapeutic drug platforms for the rapid development of novel biopharmaceuticals. Our broad portfolio of oncology product candidates includes individualized and off-the-shelf mRNA-based therapies, innovative chimeric antigen receptor (CAR) T cells, several protein-based therapeutics, including bispecific immune checkpoint modulators, targeted cancer antibodies and antibody-drug conjugate (ADC) therapeutics, as well as small molecules.   Based on our deep expertise in mRNA vaccine development and in-house manufacturing capabilities, we are developing multiple mRNA vaccine candidates for a range of infectious diseases alongside its diverse oncology pipeline. To learn more about us, please visit www.BioNTech.com. Our privacy statement can be found here:  https://www.biontech.com/int/en/home/data-privacy-statement.html Our imprint can be found: https://www.biontech.com/int/en/home/imprint.html

Cook MyoSite is on a mission to make regenerative medicine a part of everyday medicine. As an industry leader in muscle cell technology, we’re establishing new paradigms for muscle-related disorders. Established in 2002, Cook MyoSite was created to guide the Cook Group organization into the expanding world of cellular technologies. We’re proud to be a part of the vibrant medical community in Pittsburgh, Pennsylvania.

CRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom.

IDEAYA is an oncology-focused precision medicine company. Our teams are committed to the discovery and development of targeted therapeutics for patient populations selected using molecular diagnostics. We are advancing therapeutics that have the potential to be first-in-class and/or best-in-class, with a primary focus in synthetic lethality – an emerging class of precision medicine targets. IDEAYA is headquartered in South San Francisco, California.

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.

Intellia Therapeutics is a leading genome editing company whose mission is to develop potentially curative gene editing treatments that can positively transform the lives of people living with severe and life-threatening diseases. We are focused on the development of proprietary therapeutics using a recently developed biological tool known as the CRISPR/Cas9 system. The promise of the CRISPR/Cas9 system is the driving force behind the creation of Intellia. Our founders have a shared belief that the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course. This technology offers the potential for us to develop curative therapeutic options for patients with chronic diseases by addressing the underlying cause of the disease. It can bring new hope and cures to people who now have nowhere to turn for help. Our combination of deep scientific expertise and clinical development experience, along with our leading intellectual property portfolio, puts Intellia in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Intellia has the right people, assets and ambitious vision needed to take full advantage of the CRISPR/Cas9 technology’s attributes – high potency, specificity, simplicity of use, broad applicability, and multifunctional programmability – and accelerate the advancement of curative products into the clinic. View our social media community guidelines at https://www.intelliatx.com/terms-and-conditions/ .

Iovance Biotherapeutics (NASDAQ: IOVA) aims to be the global leader in innovating, developing and delivering tumor infiltrating lymphocyte (TIL) therapies for patients with cancer. We are pioneering a transformational approach to cure cancer by harnessing the human immune system’s ability to recognize and destroy diverse cancer cells in each patient. The Iovance TIL platform has demonstrated promising clinical data across multiple solid tumors. We are committed to continuous innovation in cell therapy, including gene-edited cell therapy, that may extend and improve life for patients with cancer. We are adding to our talented team as we execute our commercial launch, advance our research and development efforts, and build our internal manufacturing capabilities. Iovance is headquartered in the San Francisco Bay Area. Our Iovance Cell Therapy Center (iCTC), a state-of-the-art manufacturing facility, is located in Philadelphia. We also have a research site in Tampa, Florida. Any information expressed by Iovance is subject to the risk factors and information on forward-looking statements contained in its filings with the Securities and Exchange Commission and available here: https://ir.iovance.com/sec-filings

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.

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.

Replimune Group, Inc., a biotechnology company, develops oncolytic immune-gene therapies to treat cancer. It uses its proprietary Immulytic platform to design and develop product candidates that are intended to activate the immune system against cancer. The company's lead product candidate is RP1, a selectively replicating version of herpes simplex virus 1, which is in Phase I/II clinical trials for a range of solid tumors; and that is in Phase II clinical trials for patients with cutaneous squamous cell carcinoma. It is also developing RP2, which is in Phase I clinical trials for an anti-CTLA-4 antibody-like protein in order to block the inhibition of the immune response otherwise caused by CTLA-4; and RP3 to express immune-activating proteins that stimulate T cells. Replimune Group, Inc. was founded in 2015 and is headquartered in Woburn, Massachusetts.

Sarepta Therapeutics, headquartered in Cambridge, Massachusetts, is a global biotechnology company on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development. Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing. For information on our Community Guidelines, please visit sarepta.com/community-guidelines. We want to share a reminder with all job seekers and candidates regarding the persistence of recruiting fraud. Please read a message about recruiting fraud and steps you can take to protect yourself here: https://www.sarepta.com/recruiting-fraud

TargetRx is a clinical stage, high-tech pharmaceutical R&D company focused on developing next-generation targeted therapies for cancer patients, particularly those who are resistant or refractory to current treatments. TargetRx has developed a series of cutting-edge and efficient small molecule drug discovery platforms, which combine innovative research and development capabilities for drug design, screening, evaluation and optimization. Since its establishment, TargetRx has been granted over 170 patents from authorities in China, the US, Europe, Japan and other regions. Several potential best-in-class compounds are at different stages of multi-national clinical trials. BD Contact: shujun.mai@tjrbiosciences.com.

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.

Thermo Fisher Scientific Inc. is the world leader in serving science, with annual revenue of approximately $40 billion. Our Mission is to enable our customers to make the world healthier, cleaner and safer. Whether our customers are accelerating life sciences research, solving complex analytical challenges, increasing productivity in their laboratories, improving patient health through diagnostics or the development and manufacture of life-changing therapies, we are here to support them. Our global team delivers an unrivaled combination of innovative technologies, purchasing convenience and pharmaceutical services through our industry-leading brands, including Thermo Scientific, Applied Biosystems, Invitrogen, Fisher Scientific, Unity Lab Services, Patheon and PPD.

Zai Lab is an innovative, research-based, commercial stage biopharmaceutical company based in China and the U.S. focused on bringing transformative medicines for cancer, autoimmune, and infectious diseases to patients in China and around the world. The company was founded by Dr. Samantha Du, Chairperson and CEO, in 2014 to help address significant unmet medical needs in China and to be at the forefront of medical innovation.