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Biotechnology

Bicycle Therapeutics plc, founded in 2009 and based in Cambridge, UK, is a clinical-stage biopharmaceutical company. Its core mission is to create innovative therapeutic classes for diseases currently lacking effective treatments. The company's leading investigational product, BT1718, is a Bicycle Toxin Conjugate (BTC) that is undergoing Phase I/IIa clinical trials. This candidate is engineered to target tumors specifically expressing Membrane Type 1 matrix metalloprotease. Other oncology assets in their pipeline include BT5528, also a BTC in Phase I/II studies, which targets EphA2, and BT8009, currently in Phase I/II clinical development, designed to target Nectin-4. Beyond cancer, Bicycle Therapeutics is advancing THR-149, a plasma kallikrein inhibitor that has completed Phase II clinical trials for the treatment of diabetic macular edema. Their portfolio also includes Bicycle tumor-targeted immune cell agonists (TICAs) such as BT7480, which targets Nectin-4, and BT7455, an EphA2/CD137 TICA presently in preclinical development. The company actively fosters collaborations with various biopharmaceutical entities and organizations, broadening its development efforts into therapeutic areas like anti-infectives, cardiovascular conditions, ophthalmology, and respiratory illnesses. Notable partnerships include a clinical trial and license agreement with Cancer Research Technology Limited and Cancer Research UK. They also maintain research collaborations with AstraZeneca, Sanofi, Oxurion, and the Dementia Discovery Fund. Furthermore, a significant discovery collaboration and license agreement with Genentech is in place for the identification and development of Bicycle peptides for multiple immuno-oncology targets.

Biotechnology

Ocular Therapeutix, Inc. is a biopharmaceutical company specializing in the creation, advancement, and commercialization of ophthalmic treatments. Their innovative approach leverages a proprietary bioresorbable hydrogel technology to address a range of eye diseases and conditions. The company currently offers two key products: ReSure Sealant, an ophthalmic device designed to prevent fluid leakage from corneal incisions after cataract surgery, and DEXTENZA, a dexamethasone-based ophthalmic insert used to manage post-surgical inflammation and pain in the eye, as well as to treat allergic conjunctivitis. In addition to their commercial offerings, Ocular Therapeutix is actively developing several product candidates in various clinical stages, including: OTX-TKI, an axitinib intravitreal implant in Phase 1 clinical trials for wet age-related macular degeneration (AMD) and other retinal diseases. OTX-TIC, a travoprost intracameral implant, currently in Phase 2 studies for open-angle glaucoma and ocular hypertension. OTX-CSI, a cyclosporine intracanalicular insert that has successfully completed Phase 2 clinical trials for dry eye disease. OTX-DED, a dexamethasone intracanalicular insert undergoing Phase 2 evaluation for the temporary relief of dry eye symptoms. Strategic partnerships are also a significant part of Ocular Therapeutix's operations. This includes a collaboration with Regeneron Pharmaceuticals, Inc. focused on developing and commercializing products that combine Ocular Therapeutix's sustained-release hydrogel technology with Regeneron's large molecule VEGF-targeting compounds for retinal disease treatment. Another partnership with AffaMed Therapeutics Limited covers the development and commercialization of DEXTENZA and OTX-TIC. Additionally, a discovery-focused collaboration with Mosaic Biosciences aims to pinpoint novel targets and therapeutic agents for dry age-related macular degeneration (dAMD). Established in 2006, Ocular Therapeutix, Inc. maintains its headquarters in Bedford, Massachusetts.

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Operating primarily out of Australia, Opthea Limited is a clinical-stage biopharmaceutical firm dedicated to developing and marketing treatments for ophthalmic conditions. Its therapeutic pipeline leverages a robust intellectual property portfolio centered on Vascular Endothelial Growth Factors (VEGF) such as VEGF-C and VEGF-D, along with VEGF Receptor-3. This scientific foundation aims to address disorders characterized by abnormal blood and lymphatic vessel formation and excessive vascular leakage. The company's flagship product, OPT 302, is currently undergoing clinical trials. This soluble form of VEGFR-3 is being explored as a groundbreaking treatment for both wet neovascular age-related macular degeneration (wet AMD) and diabetic macular edema (DME). Notably, OPT 302 functions as a novel VEGF-C/D inhibitor, intended to be administered alongside existing VEGF-A inhibitors to combat wet AMD and various other retinal disorders. Initially incorporated in 1984 as Circadian Technologies Limited, the company officially adopted the name Opthea Limited in December 2015. Its corporate headquarters are located in South Yarra, Australia.

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Oculis Holding AG is a biopharmaceutical company in the clinical development stage, specializing in the creation of innovative eye drop medications for various ocular conditions, addressing both the anterior and posterior segments of the eye. A key asset in its therapeutic portfolio is OCS-01, a topical formulation of dexamethasone, currently progressing through Phase 3 clinical trials for the management of diabetic macular edema. Additionally, OCS-02, a topically administered biological candidate, is engaged in Phase 2b clinical studies to treat keratoconjunctivitis sicca, commonly known as dry eye disease. The company is also advancing OCS-05, an innovative neuroprotective agent, which targets acute optic neuritis and an array of other neuro-ophthalmic disorders, including glaucoma, diabetic retinopathy, geographic atrophy, and neurotrophic keratitis. This enterprise maintains its headquarters in Zug, Switzerland.

Surrozen, Inc.SRZN · BiotechSouth San Francisco, United States

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Surrozen, Inc., a biotechnology company, discovers and develops drug candidates to selectively modulate the Wnt pathway for tissue repair. Its lead product candidates are antibody-based therapeutics that target various disease areas, including diseases of the intestine, liver, retina, cornea, lung, kidney, cochlea, skin, pancreas, and central nervous system. The company develops SZN-814, which combines Frizzled 4 (Fzd4 agonism) and vascular endothelial growth factor (VEGF antagonism) to treat diabetic macular edema (DME), neovascular age-related macular degeneration (wet AMD), and retinopathy indications; SZN-8143, which combines Fzd4 agonism, VEGF antagonism, and interleukin-6 for the treatment of DME/wet AMD/uveitic macular edema; SZN-113 to treat Fuchs’ endothelial corneal dystrophy and geographic atrophy; and SZN-413, a Fzd4 targeted bi-specific antibody for the treatment of retinal vascular associated diseases. Surrozen, Inc. has a collaboration and license agreement with Boehringer Ingelheim International GmbH to research, develop, and commercialize Fzd4 bi-specific antibodies; and a research collaboration agreement with TCGFB, Inc. to discover antibody therapeutics targeting transforming growth factor beta, or TGF-ß, for the treatment of pulmonary fibrosis. Surrozen, Inc. was founded in 2015 and is headquartered in South San Francisco, California.

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Outlook Therapeutics, Inc. is a biopharmaceutical company in the advanced stages of clinical development, focused on inventing and bringing to market monoclonal antibody treatments for various eye conditions. Its flagship drug candidate, ONS-5010, is an ophthalmic formulation of bevacizumab currently undergoing crucial Phase III clinical trials. This investigational product is being evaluated for its potential to treat wet age-related macular degeneration and other serious retinal diseases. The company has forged strategic collaboration and licensing agreements with several partners, including IPCA Laboratories Limited, Laboratorios Liomont, S.A. de C.V., BioLexis Pte. Ltd., and Zhejiang Huahai Pharmaceutical Co., Ltd. Incorporated in 2010, the firm was formerly known as Oncobiologics, Inc., changing its name to Outlook Therapeutics, Inc. in November 2018. Its corporate base is located in Iselin, New Jersey.

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TransThera Sciences (Nanjing), Inc., clinical stage biopharmaceutical company, discovers, develops, and commercializes small molecule therapies for oncology, inflammatory, and cardiometabolic diseases. The company provides Tinengotinib (TT-00420), a MTK inhibitor that is in clinical stage for the treatment of recurrent or refractory drug-resistant solid tumors. It also develops TT-00973, a kinase inhibitor for treating various cancers; TT-01488, a BTK inhibitor for the treatment of B-cell lymphoma; TT-01688, an oral S1P1 modulator for treating various inflammatory diseases; TT-00920, an oral PDE9 inhibitor used for treating central nervous system diseases; TT-01025, an oral irreversible VAP-1 inhibitor designed for the treatment of NASH; and TT-02332, a NLRP3 inhibitor for metabolic and inflammatory diseases. The company has a collaboration with Neurocrine Biosciences, Inc. to develop NLRP3 inhibitors for multiple disease. TransThera Sciences (Nanjing), Inc. was formerly known as Nanjing TransThera Biosciences Co. Ltd. and changed its name to TransThera Sciences (Nanjing), Inc. in July 2021. The company was incorporated in 2014 and is headquartered in Nanjing, China.

ProQR Therapeutics N.V.PRQR · BiotechLeiden, Netherlands

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ProQR Therapeutics N.V. operates as a biopharmaceutical enterprise dedicated to discovering and developing RNA-based therapies designed to address genetic disorders. The company's pipeline includes two primary drug candidates in advanced clinical stages: Sepofarsen is currently in a Phase II/III clinical trial, known as the ILLUMINATE trial, targeting Leber Congenital Amaurosis 10 (LCA10). Ultevursen is also undergoing Phase II/III evaluation for the treatment of USH2A-mediated retinitis pigmentosa and Usher syndrome. Beyond its clinical programs, ProQR is actively developing its proprietary Axiomer RNA base-editing platform technology. Strategic collaborations are central to ProQR's strategy, evidenced by its licensing agreements with institutions such as Radboud University Medical Center, Inserm Transfert SA, Ionis Pharmaceuticals, Inc., and Leiden University Medical Center. Furthermore, a significant license and research partnership exists with Eli Lilly and Company, focused on the discovery, advancement, and commercialization of novel medicines for genetic conditions impacting the liver and nervous system. Established in 2012, ProQR Therapeutics N.V. maintains its corporate headquarters in Leiden, the Netherlands.

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Galapagos NV is a biotechnology company that specializes in the discovery and advancement of novel small molecule and antibody-based therapeutics. Its active clinical development programs include drugs like filgotinib and GLP3667, alongside the Toledo program and therapies targeting idiopathic pulmonary fibrosis. The firm was established on June 30, 1999, by co-founders Onno van de Stolpe, Rudi Pauwels, and Helmuth van Es, and maintains its headquarters in Mechelen, Belgium.

Biotechnology

Opus Genetics, Inc. is a clinical-stage biopharmaceutical company focused on ophthalmology. The company is dedicated to discovering and commercializing innovative therapies to address significant unmet medical needs in patients suffering from refractive and retinal eye disorders. Its portfolio includes Phentolamine Ophthalmic Solution, currently used to reverse pharmacologically induced mydriasis (pupil dilation). This solution is also advancing through Phase III clinical trials for the treatment of presbyopia and impaired vision in dim light or at night. A leading candidate in its retinal pipeline is APX3330, a small-molecule inhibitor targeting the reduction oxidation effector factor-1 protein. APX3330 has successfully completed Phase II clinical trials for diabetic retinopathy. Furthermore, Opus Genetics is developing APX2009 and APX2014, two preclinical product candidates also aimed at various retinal indications. Originally founded in 2018 as Ocuphire Pharma, Inc., the company maintains its headquarters in Farmington Hills, Michigan.

Ocugen, Inc.OCGN · BiotechMalvern, United States

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Ocugen, Inc. is a biopharmaceutical company in the clinical development phase, primarily dedicated to pioneering gene therapies aimed at treating various ophthalmic conditions leading to blindness. The company's developmental pipeline features several key candidates: OCU400: An innovative gene therapy designed to reinstate both the function and structural integrity of the retina across a broad spectrum of genetically diverse inherited retinal diseases, such as retinitis pigmentosa and Leber congenital amaurosis. OCU410: Another gene therapy candidate, currently being developed to address dry age-related macular degeneration (AMD). OCU200: A novel fusion protein, presently undergoing preclinical evaluation for its potential in treating diabetic macular edema, diabetic retinopathy, and wet AMD. Ocugen maintains significant strategic collaborations, including a partnership with CanSino Biologics Inc. for the joint development and manufacturing of gene therapies, and an alliance with Bharat Biotech for the commercialization of COVAXIN within the United States market. The company's corporate headquarters are situated in Malvern, Pennsylvania.

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LianBio is a biopharmaceutical firm dedicated to advancing and marketing therapeutic agents across various critical health sectors, including cardiovascular disorders, oncology, ophthalmological conditions, and inflammatory ailments. Its operations are primarily concentrated in China and the broader Asian region. The company's pipeline features several promising drug candidates targeting a range of severe illnesses. These include mavacamten, aimed at both obstructive and non-obstructive hypertrophic cardiomyopathy, as well as heart failure with preserved ejection fraction. TP-03 is being developed for Demodex blepharitis and meibomian gland dysfunction. For cancer, LianBio is advancing NBTXR3 for head and neck squamous cell carcinoma and other solid tumors, alongside Infigratinib for cholangiocarcinoma (both first-line and second-line settings) and gastric cancers, and BBP-398 also for solid tumors. Furthermore, Omilancor and NX-13 are in development for inflammatory bowel diseases like ulcerative colitis and Crohn's disease. Other programs include LYR-210 for chronic rhinosinusitis and Sisunatovir to combat respiratory syncytial virus. LianBio has established key collaborations, partnering with Tarsus Pharmaceuticals, Inc. on the development and market introduction of TP-03 specifically for Demodex blepharitis. Additionally, it maintains a strategic alliance with Nanobiotix S.A. for NBTXR3, a novel radioenhancer intended for direct intratumoral injection before conventional radiation therapy. Established in 2019, the company's corporate headquarters are located in Princeton, New Jersey.