Biotech stocks

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.

Gene therapy specialist with extreme concentration on a few pivotal readouts.

As a vertically integrated genetic medicine company with in-house AAV manufacturing, MeiraGTx benefits from the regulatory validation of the AAV-vector delivery mechanism in hearing loss. Success in the OTOF space paves the regulatory and CMC (Chemistry, Manufacturing, and Controls) pathway for other AAV-based genetic therapies.

Sana Biotechnology, Inc. is a biotechnology firm focused on creating treatments using engineered cells. The company develops advanced ex vivo and in vivo cell engineering platforms to address a range of conditions with significant unmet medical needs, including various cancers, diabetes, central nervous system disorders, cardiovascular diseases, and genetic illnesses. Its portfolio of drug candidates includes SG295 and SG242, which are designed to target CD19+ cancer cells involved in non-Hodgkin Lymphoma, chronic lymphocytic leukemia, and acute lymphoblastic leukemia. For multiple myeloma, Sana is progressing SG221, SG239, and SC255. Other pipeline assets include SG328 for ornithine transcarbamylase deficiency, and SG418 for sickle cell disease and beta-thalassemia. Additionally, the company is advancing SC291, a CD19 allogeneic T cell therapy, SC451 for type I diabetes mellitus, and SC379 for secondary progressive multiple sclerosis, Pelizaeus-Merzbacher disease, and Huntington's disease. Founded in 2018 as FD Therapeutics, Inc., the company adopted its current name, Sana Biotechnology, Inc., in September 2018 and is based in Seattle, Washington.

Taysha Gene Therapies, Inc. is a biotech firm specializing in the creation and market introduction of gene therapies that utilize adeno-associated virus (AAV) vectors. Its core mission is to tackle inherited diseases affecting the central nervous system (CNS). The company's development pipeline features several key programs: TSHA-120 is aimed at giant axonal neuropathy; TSHA-102 is in development for Rett syndrome; TSHA-121 and TSHA-118 are both being advanced for CLN1 disease; TSHA-105 addresses SLC13A5 Deficiency; and TSHA-101 targets GM2 gangliosidosis. Furthermore, Taysha has forged a strategic alliance with The University of Texas Southwestern Medical Center to jointly advance and bring to market innovative gene therapy solutions. Founded in 2019, the company operates from its headquarters in Dallas, Texas.

Abeona Therapeutics Inc. is a biopharmaceutical company in the clinical development stage, focused on creating innovative gene and cell therapies to address severe and uncommon genetic diseases. Its leading program, EB-101, an autologous gene-corrected cell therapy, is presently in a Phase III clinical trial for treating recessive dystrophic epidermolysis bullosa. The company's pipeline also features several other promising therapies: ABO-102, an adeno-associated virus (AAV)-based gene therapy for Sanfilippo syndrome type A. ABO-201, intended for CLN3 disease. ABO-401, developed to treat cystic fibrosis. ABO-50X, aimed at various genetic eye disorders. Additionally, Abeona is advancing AAV-based gene therapy solutions through its proprietary AIM vector platform. The company, which was founded in 1974 and is headquartered in New York, New York, changed its name to Abeona Therapeutics Inc. in June 2015, having previously been known as PlasmaTech Biopharmaceuticals, Inc.

Developing a Phase 3 gene editing candidate for this specific market.

Intellia is developing a CRISPR-based, non-AAV gene-editing modality. The FDA's hold on a CNS-directed AAV program may modestly increase relative interest in alternative genetic platforms, while Intellia's own recent experience with a lifted clinical hold provides limited evidence that non-AAV approaches can remain differentiated. This is an indirect and speculative read-through.

FDMT is directly developing 4D-150, an engineered AAV R100 gene therapy, and is advancing it into Phase 3.

Sarepta is a leader in gene therapy platforms.

ImmunityBio, Inc. is a clinical-stage biotechnology firm, founded in 2014 and based in San Diego, California. The company is focused on developing groundbreaking therapies and vaccines designed to treat a wide array of cancers and infectious diseases. Its innovative approach relies on a comprehensive suite of immunotherapy and cell therapy platforms. These advanced technologies include novel antibody-cytokine fusion proteins, synthetically engineered immunomodulators, cutting-edge vaccine technologies, natural killer (NK) cell therapies, and strategies that harness the adaptive (T-cell) immune system. ImmunityBio currently has several therapeutic candidates in advanced clinical development, with agents undergoing Phase II or III trials. These investigational treatments are aimed at various liquid and solid tumors, such as bladder, pancreatic, and lung cancers, as well as significant infectious pathogens including SARS-CoV-2 and HIV. The company actively engages in strategic partnerships, holding collaboration agreements with esteemed organizations like the National Cancer Institute, the National Institute of Deafness and Communication Disorders, and Amyris, Inc. Additionally, ImmunityBio has secured licensing agreements with numerous entities, including CytRx Corporation, EnGeneIC Pty Limited, GlobeImmune, Inc., Infectious Disease Research Institute, Sanford Health, Shenzhen Beike Biotechnology Co. Ltd., Sorrento Therapeutics, Inc., and Viracta Therapeutics, Inc.

A clinical-stage biopharmaceutical company harnessing novel protein-regulation technology to develop engineered tumor-infiltrating lymphocyte (TIL) cell therapies for the treatment of patients with solid tumors. Its proprietary cytoDRiVE® technology is designed to precisely regulate the timing and level of protein function by using FDA-approved small-molecule drugs.

Cellectis S.A., a clinical-stage biotechnology company founded in 1999 and headquartered in Paris, France, concentrates on immuno-oncology. The firm engineers therapeutic solutions utilizing gene-edited T-cells, which are modified to display chimeric antigen receptors (CARs) for the specific identification and destruction of cancer cells. Its operations are divided into two principal segments: Therapeutics and Plants. The company's pipeline features several allogeneic CAR T-cell product candidates. These include UCART19, aimed at CD19-expressing hematologic malignancies such as acute lymphoblastic leukemia; ALLO-501 and ALLO-501A, developed to treat relapsed or refractory diffuse large B-cell lymphoma and follicular lymphoma; ALLO-316, targeting renal cell carcinoma; UCART123, for acute myeloid leukemia; and UCART22, addressing B-cell acute lymphoblastic leukemia. Additionally, Cellectis is advancing UCARTCS1 and ALLO-715 for the treatment of multiple myeloma. Cellectis has forged significant strategic partnerships and alliances with companies including Allogene Therapeutics, Inc., Les Laboratoires Servier, The University of Texas M.D. Anderson Cancer Center, and Iovance Biotherapeutics. It also maintains a strategic research and development collaboration with Cytovia Therapeutics, Inc.

Century Therapeutics, Inc. is a biotechnology company dedicated to pioneering allogeneic cell therapies for a range of cancers, specifically solid tumors and hematological malignancies. The firm's foremost clinical program is CNTY-101, an allogeneic, induced pluripotent stem cell (iPSC)-derived CAR-iNK cell therapy. This therapy is specifically designed to target CD19 in patients with B-cell lymphoma that has relapsed or proven refractory to previous treatments. In addition to its lead candidate, the company's robust pipeline features several other therapeutic assets: CNTY-103: A CAR-iNK candidate aimed at CD133 + EGFR for recurrent glioblastoma. CNTY-102: A CAR-iT therapy targeting CD19 + CD79b, intended for relapsed or refractory B-cell lymphoma and other B-cell cancers. CNTY-104: A multi-specific CAR-iT or CAR-iNK candidate in development for acute myeloid leukemia. CNTY-106: Another multi-specific CAR-iNK or CAR-iT candidate for multiple myeloma. Established in 2018, Century Therapeutics, Inc. is based in Philadelphia, Pennsylvania.

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.

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.

Oxford BioMedica has directly relevant adenovirus development, GMP manufacturing, analytics, fill-finish, and regulatory-support capabilities.

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.

Protalix BioTherapeutics, Inc., a biopharmaceutical company, engages in the development, production, and commercialization of recombinant therapeutic proteins based on the ProCellEx plant cell-based protein expression system. The company provides Elelyso for the treatment of Gaucher disease; and Elfabrio for the treatment of adult patients with a confirmed diagnosis of Fabry disease. It is also developing PRX-115, a plant cell expressed recombinant PEGylated Uricase, which is in Phase 2 trial for the treatment of gout; and PRX-119, a plant cell expressed PEGylated recombinant human DNase I product candidate for the treatment of neutrophil extracellular traps diseases. The company has agreements and partnerships with Pfizer; Fundação Oswaldo Cruz; and Chiesi Farmaceutici S.p.A. Protalix BioTherapeutics, Inc. has strategic partnership with Secarna Pharmaceuticals GmbH & Co KG. The company is headquartered in Hackensack. New Jersey.

ADMA Biologics, Inc. functions as a biopharmaceutical company specializing in the development, manufacturing, and commercialization of advanced biologic therapies derived from blood plasma. These specialized products are engineered to address immune system disorders and infectious diseases, catering to markets across the United States and internationally. Their current product portfolio includes BIVIGAM and ASCENIV, both intravenous immune globulin (IVIG) treatments prescribed for primary humoral immunodeficiency (PI). Additionally, the company offers Nabi-HB, utilized for immediate treatment following acute exposure to the Hepatitis B virus and other specified exposures. Beyond its existing offerings, ADMA is actively developing a pipeline of new plasma-derived therapeutics. This includes immunoglobulin products specifically targeting the prevention and treatment of S. pneumonia infections. The company also oversees its own facilities for source plasma collection. Product distribution is managed through a comprehensive network involving independent distributors, sales agents, specialty pharmacies, and other alternative healthcare providers. ADMA Biologics, Inc. was founded in 2004 and maintains its corporate headquarters in Ramsey, New Jersey.

Ultragenyx Pharmaceutical Inc. is a biopharmaceutical firm dedicated to discovering, developing, and commercializing innovative treatments for rare and ultra-rare genetic conditions. Its operations span North America, Europe, and other international markets. The company's portfolio of marketed biologic products addresses several serious diseases. This includes Crysvita (burosumab), an antibody that targets fibroblast growth factor 23, used to treat X-linked hypophosphatemia and tumor-induced osteomalacia. Mepsevii offers enzyme replacement therapy for both pediatric and adult patients suffering from Mucopolysaccharidosis VII. Dojolvi is available for individuals with long-chain fatty acid oxidation disorders, while Evkeeza (evinacumab) provides a treatment option for homozygous familial hypercholesterolemia. Ultragenyx also boasts a robust pipeline of investigational therapies. Notable candidates include DTX401, an adeno-associated virus 8 (AAV8) gene therapy for glycogen storage disease type Ia, and DTX301, another AAV8 gene therapy aimed at ornithine transcarbamylase deficiency. Other promising developments feature UX143, a human monoclonal antibody for osteogenesis imperfecta; GTX-102, an antisense oligonucleotide designed for Angelman syndrome; UX701, targeting Wilson disease; and UX053, in development for glycogen storage disease type III. To advance its research and development efforts, Ultragenyx Pharmaceutical Inc. maintains strategic collaboration and licensing agreements with numerous partners, such as Kyowa Kirin Co., Ltd., Saint Louis University, REGENXBIO Inc., Bayer Healthcare LLC, GeneTx, Mereo, University of Pennsylvania, Arcturus Therapeutics Holdings Inc., Solid Biosciences Inc., and Daiichi Sankyo Co., Ltd. The company was founded in 2010 and operates from its headquarters in Novato, California.

Celltrion, Inc., a South Korean biopharmaceutical company, specializes in the development and manufacturing of protein-based therapies, including treatments for oncology. Its commercial portfolio features established products such as Remsima, Truxima, and Herzuma, alongside a range of generic pharmaceuticals and diagnostic kits for COVID-19. The company maintains an extensive pipeline of antibody biosimilars currently undergoing various stages of clinical evaluation. Late-stage (Phase 3) candidates include CT-P17, aimed at rheumatoid arthritis, psoriatic arthritis, and inflammatory bowel disease (IBD); CT-P16, targeting metastatic colorectal and breast cancers; CT-P43, for inflammatory conditions like psoriasis, Crohn's disease, and ulcerative colitis; CT-P42, addressing diabetic macular edema; and CT-P59, developed for COVID-19. In Phase 2, CT-P27 is being tested for pandemic and seasonal influenza. Earlier in development, Phase 1 assets comprise CT-P39 for asthma and urticaria, CT-P41 for osteoporosis and bone loss, and CT-G20 for cardiomyopathy. Additionally, Celltrion maintains a collaboration with Inhalon Biopharma, Inc. to advance IN-006, an inhaled formulation of regdanvimab intended for the treatment of COVID-19 patients. Established in 2002, the company operates primarily from its headquarters in Incheon, South Korea.

Based in Leiden, the Netherlands, Pharming Group N.V. operates as a biopharmaceutical firm dedicated to advancing and marketing protein replacement therapies and precise medical solutions. Its global efforts, spanning the United States, Europe, and other international markets, focus on addressing rare diseases and critical unmet medical needs. The company's flagship product, Ruconest, is a recombinant human C1 esterase inhibitor prescribed for managing acute attacks of hereditary angioedema. Pharming's development pipeline also encompasses rhC1INH, which is under investigation for potential applications in pre-eclampsia, acute kidney injury, and COVID-19. Furthermore, they are progressing leniolisib, a phosphoinositide 3-kinase delta (PI3K delta) inhibitor, for individuals afflicted with activated PI3K delta syndrome, as well as an alpha-glucosidase therapy aimed at treating Pompe and Fabry diseases. The company maintains significant partnerships, including a development and license agreement with Novartis and a strategic alliance with Orchard Therapeutics plc. This latter collaboration involves the research, development, manufacture, and commercialization of OTL-105, an experimental ex-vivo autologous hematopoietic stem cell gene therapy designed to treat hereditary angioedema.

Absci Corporation operates as a biopharmaceutical company primarily focused on discovering novel drug targets and developing new therapeutic compounds. Leveraging its distinctive integrated drug creation platform, the firm generates potential biologic medicines and essential cell lines for manufacturing, which it provides to its collaborators, predominantly within the United States. This advanced platform is instrumental in facilitating the development of biologics by meticulously integrating the traditionally separate stages of drug discovery and cell line engineering into one streamlined process. Absci Corporation was founded in 2011 and is based in Vancouver, Washington.

Insilico is an AI-native platform company whose disclosed collaboration uses upfront, milestone, and royalty economics.

Insilico is an AI-native drug-discovery peer whose valuation and funding model depend on converting platform capabilities into pharmaceutical partnerships. A large China-to-West AI-biotech license strengthens the sector validation and partnering benchmark, while also increasing competition for global partners and capital.

Revenue is tied to drug milestones and success of candidates identified by AI platforms.

The Envisagenics-Boehringer agreement is a high-value validation of pharmaceutical demand for AI-enabled target discovery, milestone payments, and royalties. Recursion is an AI-native drug-discovery platform peer, so the transaction may improve partner interest and credibility for Recursion’s platform-led partnering model, while also setting a higher benchmark for future deals.

Gain Therapeutics, Inc. is a biotechnology firm specializing in the creation of advanced treatments for conditions originating from improperly folded proteins. The company primarily directs its efforts towards rare genetic disorders and neurological ailments. It employs its unique Site-Directed Enzyme Enhancement Therapy (SEE-Tx) platform to pinpoint specific allosteric sites on malformed proteins. Subsequently, Gain Therapeutics identifies proprietary small molecules designed to bind to these locations, thereby restoring correct protein conformation and alleviating the disease. Their current development pipeline includes several structurally targeted allosteric regulator candidates aimed at combating a range of diseases, such as Morquio B, GM1 gangliosidosis (GM1), neuronopathic Gaucher disease, GBA1 Parkinson's, Krabbe, and Mucopolysaccharidosis type 1. Established in 2017, the company is headquartered in Bethesda, Maryland.

Based in Watertown, Massachusetts, Disc Medicine, Inc. operates as a clinical-stage biotechnology company. Its core mission involves the discovery, development, and commercialization of novel therapeutic solutions for individuals suffering from serious hematological diseases. The company is actively constructing a pipeline of drug candidates, designed to address a variety of these conditions by precisely targeting fundamental biological mechanisms related to red blood cell biology, particularly heme biosynthesis and iron homeostasis.

Cullinan Therapeutics, Inc., a clinical-stage biopharmaceutical company, develops therapies for autoimmune diseases and cancer in the United States. It engages in the development of CLN-978, a CD19xCD3 bispecific T cell engager for autoimmune diseases in Phase 1 clinical trials; Velinotamig, a BCMAxCD3 bispecific T cell in Phase 1 clinical trials in patients with autoimmune diseases; and CLN-049, a FLT3xCD3 bispecific T cell engager, which is in Phase 1 clinical trials in patients with relapsed and refractory acute myeloid leukemia or myelodysplastic syndrome. The company is also developing Zipalertinib, an orally available small-molecule, irreversible epidermal growth factor receptor inhibitor in Phase 2b portion of the REZILIENT1 clinical trial in patients with EGFR ex20ins non-small cell lung cancer. It has a license agreement with Chongqing Genrix Biopharmaceutical Co., Ltd to develop and commercialize velinotamig; a co-development agreement with Taiho Pharmaceutical Co., Ltd to develop zipalertinib; and a collaboration agreement with Adimab, LLC to discover and/or optimize antibodies. The company was formerly known as Cullinan Oncology, Inc. and changed its name to Cullinan Therapeutics, Inc. in April 2024. Cullinan Therapeutics, Inc. was incorporated in 2016 and is headquartered in Cambridge, Massachusetts.

Immix Biopharma, Inc. is a clinical-stage biotechnology firm dedicated to creating a range of tissue-specific therapies. Operating in both the United States and Australia, their primary focus areas are oncology (cancer) and inflammatory diseases. Their product pipeline includes IMX-110, which is currently undergoing Phase 1b/2a clinical trials for the treatment of soft tissue sarcoma and various solid tumors. They are also developing IMX-111, a targeted biologic agent for colorectal cancers, and IMX-120, another tissue-specific biologic designed to address ulcerative colitis and severe Crohn's disease. In a strategic move, the company has forged a clinical collaboration and supply agreement with BeiGene Ltd. This partnership aims to conduct a Phase 1b combination clinical trial, evaluating IMX-110 in conjunction with BeiGene's anti-PD-1 drug, Tislelizumab, for solid tumor indications. Founded in 2012, Immix Biopharma maintains its headquarters in Los Angeles, California.

Electra Therapeutics, Inc. is a clinical stage biotechnology company. It is engaged in therapies against novel targets for diseases in immunology and cancer. The company was founded by Adam Rosenthal on October 11, 2018 and is headquartered in South San Francisco, CA.

Cardiff Oncology, Inc., a clinical-stage biotechnology company, develops novel therapies for the treatment of various cancers in the United States. Its lead drug candidate is Onvansertib, an oral and selective polo-like kinase 1 inhibitor for treating RAS-mutated metastatic colorectal cancer. The company also focuses on the clinical program in indications, such as investigator-initiated trials in metastatic pancreatic ductal adenocarcinoma, small cell lung cancer, metastatic triple negative breast cancer, and chronic myelomonocytic leukemia; CRDF-004, a Phase 2 open-label randomized multi-center clinical trial of onvansertib in combination with FOLFIRI and bevacizumab or FOLFOX and bevacizumab for the treatment of patients confirmed metastatic and unresectable colorectal cancer in patients with a KRAS or NRAS mutation; and TROV-054, a Phase 1b/2 single-arm clinical trial in KRAS-mutated metastatic colorectal cancer. The company was formerly known as Trovagene, Inc. and changed its name to Cardiff Oncology, Inc. in May 2012. Cardiff Oncology, Inc. was founded in 1999 and is headquartered in San Diego, California.

Corvus Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical firm specializing in the development and commercialization of immuno-oncology therapies. Their leading investigational compound, Mupadolimab (CPI-006), an anti-CD73 monoclonal antibody, is currently progressing through Phase Ib/II clinical trials for the treatment of non-small cell lung cancer and head and neck cancers. Another promising candidate, CPI-818, functions as a covalent ITK inhibitor. This therapy is undergoing Phase I/Ib clinical evaluation for patients battling various malignant T-cell lymphomas, aiming to impede the proliferation of specific malignant T-cells. Additionally, Ciforadenant (CPI-444), an oral, small molecule antagonist of the A2A receptor, is in Phase II clinical trials for individuals diagnosed with either advanced or refractory renal cell carcinoma. Beyond these clinical-stage assets, Corvus maintains a preclinical portfolio including CPI-182, an antibody designed to block inflammation and myeloid suppression, and CPI-935, an adenosine A2B receptor antagonist intended to prevent fibrosis. The company also benefits from a strategic collaboration with Angel Pharmaceuticals, which aids in advancing its diverse pipeline of targeted experimental drugs. Founded in 2014, Corvus Pharmaceuticals is headquartered in Burlingame, California.

MacroGenics, Inc., a clinical-stage biopharmaceutical company, discovers, develops, manufactures, and commercializes antibody-based therapeutics for the treatment of cancer in the United States. The company’s product pipeline includes lorigerlimab, a bispecific DART molecule that targets checkpoint inhibitors PD-1 and CTLA-4 for the treatment of mCRPC and docetaxel that is in phase 2 clinical trials, as well as for the treatment of platinum-resistant ovarian cancer and clear cell gynecologic cancer which has completed phase 1 clinical trial; MGC026, an ADC that targets B7-H3 and delivers a novel topoisomerase I inhibitor (TOP1i)-based linker-payload; MGC028, an antibody-drug conjugates (ADC) that targets ADAM9 and delivers a novel TOP1i-based linker-payload for the treatment of solid tumors, which is in phase 1 clinical trials; MGC030, a ADC molecule that targets an undisclosed antigen expressed across several solid tumors, which is in preclinical trials. It is also developing T-cell engager programs. The company has collaborations with TerSera Therapeutics LLC; Incyte Corporation; and Gilead Sciences, Inc. MacroGenics, Inc. was incorporated in 2000 and is headquartered in Rockville, Maryland.

Evopoint Biosciences Co., Ltd. operates as a biopharmaceutical company that offers therapies for patients with oncology and multidrug-resistant infections. The company's three core technology platforms are a Small Molecule Drug Discovery Platform, a Novel Antibody Discovery Platform, and a Targeted Protein Degradation Platform. Several of its pipeline products have advanced to Phase III clinical trials or the New Drug Application stage.

Voronoi, Inc., founded in 2015 and based in Incheon, South Korea, is a biotechnology company dedicated to the research, development, and identification of small molecule kinase inhibitors. Its extensive product pipeline addresses various medical needs. Key candidates include VRN01, an oral leucine-rich repeat kinase 2 (LRRK2) inhibitor, being developed for glioblastoma multiforme, brain cancer, and pancreatic cancer. Treatments for lung cancer are represented by VRN07, VRN06, and VRN11, while VRN08 and VRN10 are in development for breast cancer. Additionally, VRN02 targets rheumatoid arthritis and atopic dermatitis. The company's pipeline further encompasses VRN04-1 for autoimmune conditions, VRN04-2 for multiple sclerosis and amyotrophic lateral sclerosis (ALS), VRN13 for pulmonary arterial hypertension, and VRN14 for challenging solid tumors.

Lin BioScience, Inc. is an innovative biopharmaceutical company focused on developing novel therapies across oncology, ophthalmology, and metabolic diseases. Its robust pipeline includes LBS-008, an orally administered treatment currently in Phase 3 clinical trials for adolescent Stargardt disease (STGD1) and Phase 2/3 trials for dry age-related macular degeneration (Dry AMD), both aimed at evaluating its safety and effectiveness. Other key candidates are LBS-007, a natural non-ATP CDC7 inhibitor designed to combat various cancers; LBS-002, an anti-tubulin compound targeting primary and metastatic brain tumors; and LBS-009, an oral medication for non-alcoholic fatty liver disease and Type 2 diabetes. The company is headquartered in Taipei, Taiwan.

Kexing Biopharm Co., Ltd. is a global pharmaceutical enterprise primarily involved in the exploration, manufacturing, and commercialization of advanced recombinant protein-based drugs and microbial preparations, serving both the Chinese domestic market and international clientele. The company strategically directs its research and development endeavors towards crucial medical areas, specifically focusing on antiviral therapies, oncology, immunology, hematological conditions, digestive system health, and degenerative diseases. Its extensive product lineup features diverse pharmaceutical solutions. This includes innovative recombinant human proteins such as interferon a1b, erythropoietin, and granulocyte stimulating factor. Additionally, their offerings encompass microbial formulations like Clostridium butyricum combined live bacteria powder, traditional Chinese patent medicines (e.g., Kehuang Capsule), and chemical drugs like Entecavir. Kexing Biopharm also provides cutting-edge biologics, including liraglutide (a human glucagon-like peptide-1), along with other specialized therapeutics such as Albumin-Paclitaxel, Infliximab, Bevacizumab, adalimumab, trastuzumab, neratinib maleate tablets, and lenalidomide capsules. Established in 1989, the company's main operations are situated in Jinan, China.

This biopharmaceutical firm is currently in the clinical development stage, concentrating on the creation of innovative treatments for neuropsychiatric disorders such as schizophrenia and bipolar depression. Central to their pipeline is LB-102, their primary investigational drug, which is a methylated variant of amisulpride.

BioCryst Pharmaceuticals, Inc. is a biotechnology company focused on discovering and developing novel, orally administered, small-molecule therapeutics. The company currently markets two key products: Peramivir injection: An intravenous neuraminidase inhibitor, sold under the brand names RAPIVAB, RAPIACTA, and PERAMIFLU, used for the treatment of acute uncomplicated influenza. ORLADEYO: An oral serine protease inhibitor designed to manage hereditary angioedema. BioCryst's development pipeline includes several promising candidates: BCX9930: An oral factor D inhibitor in Phase II clinical trials for complement-mediated diseases. BCX9250: An oral activin receptor-like kinase-2 inhibitor undergoing Phase I clinical evaluation for fibrodysplasia ossificans progressiva. Galidesivir: An RNA dependent-RNA polymerase inhibitor, also in Phase I, aimed at treating a range of RNA viruses, including Marburg, Yellow Fever, Ebola, and Zika. The company engages in strategic collaborations and in-license agreements with numerous partners. These include pharmaceutical entities like Torii Pharmaceutical Co., Ltd., Seqirus UK Limited, Shionogi & Co., Ltd., Green Cross Corporation, and Mundipharma International Holdings Limited. Their alliances also extend to governmental and academic organizations such as the National Institute of Allergy and Infectious Diseases, the Biomedical Advanced Research and Development Authority, the U.S. Department of Health and Human Services, The University of Alabama at Birmingham, Albert Einstein College of Medicine of Yeshiva University, and Industrial Research, Ltd. Established in 1986, BioCryst Pharmaceuticals, Inc. is headquartered in Durham, North Carolina.

ADARx Pharmaceuticals, Inc. is a late-clinical-stage biotechnology company. It focuses on developing next-generation small interfering RNA (siRNA) therapeutics for diseases with significant unmet medical needs. The company was founded by Zhen Li in December 2019 and is headquartered in San Diego, CA.

Kymera Therapeutics, Inc. is a biopharmaceutical company dedicated to discovering and developing pioneering small molecule therapeutics. These treatments operate by harnessing the body's inherent protein degradation system to selectively eliminate disease-causing proteins. The company's pipeline features several programs, including the IRAK4 program, currently in Phase I clinical trials, which targets various immunology-inflammation disorders such as hidradenitis suppurativa, atopic dermatitis, macrophage activation syndrome, generalized pustular psoriasis, and rheumatoid arthritis. Additionally, Kymera is advancing its IRAKIMiD program, designed to address MYD88-mutated diffuse large B cell lymphoma. Other key programs include the STAT3 program, focused on hematological malignancies, solid tumors, autoimmune diseases, and fibrosis, as well as the MDM2 program, which is also aimed at hematological malignancies and solid tumors. The company was founded in 2015 and maintains its headquarters in Watertown, Massachusetts.

Partner on the 'next generation' of JNJ's proprietary immunology cycle (oral drugs).

Protagonist holds a U.S. co-commercialization option with 50/50 profit sharing for the approved rusfertide product, giving the company direct participation in a new polycythemia vera revenue stream.