Annexon Biosciences is a clinical-stage biopharmaceutical company focused on developing innovative therapies for autoimmune and neurodegenerative diseases that are influenced by the classical complement pathway. The company’s research revolves around C1q, a key molecule that, when aberrantly activated, contributes to various disease processes. Its lead product candidates include ANX005, a monoclonal antibody designed for intravenous administration, which has completed Phase 1b trials for treating Guillain-Barré syndrome, and ANX007, formulated for intravitreal use, which has also completed Phase 1b trials aimed at glaucoma treatment. Additionally, Annexon is advancing preclinical candidates targeting conditions such as warm autoimmune hemolytic anemia, Huntington's disease, amyotrophic lateral sclerosis, and geographic atrophy. Founded in 2011 and headquartered in South San Francisco, California, the company employs a biomarker-driven strategy to ensure effective engagement of its therapies in the intended patient populations.
Scout Bio, Inc. researches and develops one-time therapies for major chronic pet health conditions using veterinary biotechnology and gene therapy. It offers therapies for anemia associated with chronic kidney disease (CKD) in cats. The company offers pet therapeutics, including small molecular drugs, protein therapies, and gene therapies. Scout Bio, Inc. was founded in 2016 and is headquartered in Philadelphia, Pennsylvania.
Pyramid Biosciences, Inc. is a biotechnology company based in Waltham, Massachusetts, established in 2015. The company specializes in discovering and developing targeted therapeutics that address disorders regulated by the tropomyosin receptor kinase (TRK) family. Its pipeline includes TRK modulators aimed at treating dermatology and oncology disorders linked to the dysregulation of the TRK signaling pathway. Notable products in development include PBI-100, which offers anti-itch, anti-inflammatory, and antiproliferative effects for dermatological conditions, and PBI-200, an orally active TRK inhibitor designed to overcome resistance mutations associated with existing TRK inhibitor therapies in oncology. By selectively modulating TRK receptors, Pyramid Biosciences aims to control key signaling pathways, thus enabling healthcare professionals to manage symptoms and influence disease progression effectively.
Beckley Psytech Limited manufactures psychedelic drug for treating psychiatric and neurological diseases. Beckley Psytech Limited was formerly known as Beckley Psychedelics Limited and changed its name to Beckley Psytech Limited in May 2020. The company was founded in 2014 and is based in Oxford, United Kingdom.
ImaginAb, Inc. is a clinical-stage biotechnology company specializing in immune oncology imaging, with a focus on enhancing precision medicine for cancer treatment. Founded in 2007 and based in Inglewood, California, the company develops engineered antibody fragments known as minibodies, which are designed for diagnostic imaging using Positron Emission Tomography (PET). ImaginAb's portfolio includes precision imaging products targeting CD8 and CD4 for immune-oncology applications, as well as PSMA PET for prostate cancer diagnostics. By creating highly targeted diagnostic imaging agents that retain the specificity of parental antibodies while being optimized for clinical use, ImaginAb addresses critical needs in patient selection and treatment monitoring for cancer immunotherapy.
Larimar Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases. The company’s lead compound, CTI-1601, is currently being evaluated in a Phase 1 clinical program as a potential treatment for Friedreich’s ataxia, a rare and progressive genetic disease. Larimar also plans to use its intracellular delivery platform to design other fusion proteins to target additional rare diseases characterized by deficiencies in intracellular bioactive compounds.
At Arch Oncology, they are aiming high to discover and develop new antibody therapeutics to treat patients living with cancer. They are committed to advancing antibodies with best-in-class potential.Their next-generation anti-CD47 antibodies are highly differentiated, with the potential to improve upon the safety and efficacy profile relative to other agents in this class.
ViaCyte, Inc. is a regenerative medicine company based in San Diego, California, focused on developing innovative cell replacement therapies for diabetes. The company is working on several product candidates, including VC-01, designed for type 1 diabetes, and VC-02, which facilitates blood vessel interaction with implanted pancreatic precursor cells. Their VCTX210 product utilizes a proprietary human stem cell line that helps prevent immune system rejection, eliminating the need for immunosuppressants. ViaCyte's therapies differentiate stem cells into pancreatic beta-cell precursors that are implanted in an encapsulation device, enabling the production of insulin in response to blood glucose levels. The company's aim is to provide long-term solutions for both type 1 and type 2 diabetes patients, reducing their dependence on insulin and minimizing associated health complications. Founded in 1999 and formerly known as Novocell, Inc., ViaCyte also has operations in Athens, Georgia.
Silence Therapeutics is a biotechnology company based in the United Kingdom that specializes in the discovery and development of innovative RNA therapeutics for various medical conditions, including hematology, cardiovascular diseases, and rare metabolic disorders. The company utilizes short interfering RNA (siRNA) technology to target and degrade specific messenger RNAs, thereby modulating gene expression. Its notable product candidates include SLN124, aimed at treating iron overload disorders by silencing the TMPRSS6 gene, and SLN360, which targets the LPA gene to lower lipoprotein levels and reduce cardiovascular risks. Silence Therapeutics also engages in strategic collaborations, including a partnership with AstraZeneca to advance small interfering RNA therapeutics for multiple disease areas. Established as SR Pharma plc, the company rebranded to Silence Therapeutics in 2007 and is headquartered in London.
HemoShear Therapeutics develops new treatments for patients with rare metabolic disorders. It creates human-relevant systems to accurately replicate the biology of organ systems and diseases for use throughout drug discovery and development. They work strategically in partnerships with pharmaceutical, biotechnology, and medical device companies. It generates meaningful human response data to increase confidence in decision-making, reduce the risk of costly failures, and identify efficacious therapies.
Flame Biosciences, Inc. is a clinical-stage biotechnology company based in New York that specializes in the research, development, and commercialization of innovative therapies targeting cancer and inflammatory diseases. Established in 2017, the company is advancing its FL-101 therapy, designed to treat lung cancers associated with inflammation, and FL-201, aimed at alleviating chronic inflammation in patients. By leveraging the emerging field of inflammasome science, Flame Biosciences seeks to provide effective treatments that enhance the quality of life for individuals affected by serious health conditions.
Eledon Pharmaceuticals, Inc. is a specialty pharmaceutical company dedicated to developing products for patients with ear, nose, and throat disorders. The company's lead product, OP0201, is a surfactant-based nasal aerosol designed for individuals at risk of or suffering from otitis media, which is inflammation of the middle ear. In addition to OP0201, Eledon has developed foam-based drug delivery technologies, OP0101 and OP0102, aimed at administering medications to the ear, nose, and sinus cavities. Founded in 2004 and initially known as Novus Therapeutics, the company rebranded in January 2021 and is headquartered in Irvine, California.
Pyramid Biosciences, Inc. is a biotechnology company based in Waltham, Massachusetts, established in 2015. The company specializes in discovering and developing targeted therapeutics that address disorders regulated by the tropomyosin receptor kinase (TRK) family. Its pipeline includes TRK modulators aimed at treating dermatology and oncology disorders linked to the dysregulation of the TRK signaling pathway. Notable products in development include PBI-100, which offers anti-itch, anti-inflammatory, and antiproliferative effects for dermatological conditions, and PBI-200, an orally active TRK inhibitor designed to overcome resistance mutations associated with existing TRK inhibitor therapies in oncology. By selectively modulating TRK receptors, Pyramid Biosciences aims to control key signaling pathways, thus enabling healthcare professionals to manage symptoms and influence disease progression effectively.
Poseida Therapeutics, Inc. is a clinical-stage biopharmaceutical company headquartered in San Diego, California, focused on developing innovative therapies for patients with significant unmet medical needs. The company is advancing a diverse pipeline that includes treatments for hematological malignancies and solid tumors, as well as liver-directed gene therapies aimed at rare diseases. Poseida is known for its proprietary gene engineering platforms, including the non-viral piggyBac DNA Modification System and the Cas-CLOVER site-specific gene editing system, which facilitate the creation of next-generation cell and gene therapeutics. Additionally, the company's portfolio encompasses CAR-T therapies for cancer and gene therapies targeting rare and life-threatening conditions, such as Ornithine transcarbamylase deficiency and methylmalonic acidemia. Founded in 2014, Poseida Therapeutics is committed to addressing critical health challenges through its advanced therapeutic solutions.
Amplyx Pharmaceuticals is a small molecule drug development company focused on creating innovative therapies for life-threatening infections, particularly in patients with compromised immune systems. Founded in 2006 and based in San Diego, California, the company specializes in developing oral and injectable anti-infective drugs, including antifungal agents aimed at treating invasive fungal infections caused by pathogens such as Candida and Aspergillus. Amplyx employs a unique platform approach that enhances the efficacy and reduces the toxicity of existing drugs by combining them with additional small molecules. This strategy addresses critical treatment needs in hospital settings and provides effective therapy options for patients after discharge, particularly those undergoing chemotherapy and other immunosuppressive treatments.
Scout Bio, Inc. researches and develops one-time therapies for major chronic pet health conditions using veterinary biotechnology and gene therapy. It offers therapies for anemia associated with chronic kidney disease (CKD) in cats. The company offers pet therapeutics, including small molecular drugs, protein therapies, and gene therapies. Scout Bio, Inc. was founded in 2016 and is headquartered in Philadelphia, Pennsylvania.
Prevail Therapeutics Inc. is a biotechnology company based in New York that specializes in developing gene therapies for neurodegenerative diseases. Founded in 2017 with support from The Silverstein Foundation for Parkinson’s with GBA, REGENXBIO, and OrbiMed, the company is focused on creating innovative adeno-associated virus (AAV)-based treatments. Its lead candidate, PR001, is currently undergoing Phase I/II clinical trials aimed at treating Parkinson's disease linked to GBA1 mutations and neuronopathic Gaucher disease. Additionally, Prevail is developing PR006 for frontotemporal dementia associated with GRN mutations and PR004 for various synucleinopathies. The company is dedicated to advancing therapies that can modify the course of these debilitating conditions.
Cabaletta Bio is a clinical-stage biotechnology company dedicated to developing engineered T cell therapies for patients with B cell-mediated autoimmune diseases. Utilizing its proprietary technology, the company focuses on chimeric autoantibody receptor (CAAR) T cells, which are engineered to selectively target and eliminate pathogenic B cells responsible for producing harmful autoantibodies while preserving healthy B cells. The company’s lead product candidate, DSG3-CAART, is currently undergoing a Phase I clinical trial for the treatment of mucosal pemphigus vulgaris, a debilitating autoimmune skin disease, as well as Hemophilia A with Factor VIII alloantibodies. Cabaletta's pipeline also includes MuSK-CAART, aimed at treating certain patients with myasthenia gravis, and FVIII-CAART and DSG3/1-CAART, both in earlier stages of development for different autoimmune conditions. Founded in 2017 and headquartered in Philadelphia, Cabaletta Bio has established collaborations with the University of Pennsylvania and The Regents of the University of California to further its research efforts.
Annexon Biosciences is a clinical-stage biopharmaceutical company focused on developing innovative therapies for autoimmune and neurodegenerative diseases that are influenced by the classical complement pathway. The company’s research revolves around C1q, a key molecule that, when aberrantly activated, contributes to various disease processes. Its lead product candidates include ANX005, a monoclonal antibody designed for intravenous administration, which has completed Phase 1b trials for treating Guillain-Barré syndrome, and ANX007, formulated for intravitreal use, which has also completed Phase 1b trials aimed at glaucoma treatment. Additionally, Annexon is advancing preclinical candidates targeting conditions such as warm autoimmune hemolytic anemia, Huntington's disease, amyotrophic lateral sclerosis, and geographic atrophy. Founded in 2011 and headquartered in South San Francisco, California, the company employs a biomarker-driven strategy to ensure effective engagement of its therapies in the intended patient populations.
Cabaletta Bio is a clinical-stage biotechnology company dedicated to developing engineered T cell therapies for patients with B cell-mediated autoimmune diseases. Utilizing its proprietary technology, the company focuses on chimeric autoantibody receptor (CAAR) T cells, which are engineered to selectively target and eliminate pathogenic B cells responsible for producing harmful autoantibodies while preserving healthy B cells. The company’s lead product candidate, DSG3-CAART, is currently undergoing a Phase I clinical trial for the treatment of mucosal pemphigus vulgaris, a debilitating autoimmune skin disease, as well as Hemophilia A with Factor VIII alloantibodies. Cabaletta's pipeline also includes MuSK-CAART, aimed at treating certain patients with myasthenia gravis, and FVIII-CAART and DSG3/1-CAART, both in earlier stages of development for different autoimmune conditions. Founded in 2017 and headquartered in Philadelphia, Cabaletta Bio has established collaborations with the University of Pennsylvania and The Regents of the University of California to further its research efforts.
Prevail Therapeutics Inc. is a biotechnology company based in New York that specializes in developing gene therapies for neurodegenerative diseases. Founded in 2017 with support from The Silverstein Foundation for Parkinson’s with GBA, REGENXBIO, and OrbiMed, the company is focused on creating innovative adeno-associated virus (AAV)-based treatments. Its lead candidate, PR001, is currently undergoing Phase I/II clinical trials aimed at treating Parkinson's disease linked to GBA1 mutations and neuronopathic Gaucher disease. Additionally, Prevail is developing PR006 for frontotemporal dementia associated with GRN mutations and PR004 for various synucleinopathies. The company is dedicated to advancing therapies that can modify the course of these debilitating conditions.
Engage Therapeutics, based in Summit, New Jersey, is a biopharmaceutical company focused on developing innovative therapies for individuals experiencing uncontrolled epileptic seizures. The company's lead product, Staccato® alprazolam, is a handheld drug-device combination that utilizes an established FDA-approved delivery system to administer alprazolam, an FDA-approved benzodiazepine. This product aims to provide immediate rescue therapy for active seizures, offering a potential solution for patients and their caregivers. In a Phase 2a proof of concept study, Staccato alprazolam demonstrated efficacy in reducing seizures in a photosensitivity model. Engage Therapeutics plans to advance this product into further clinical development through a 505(b)(2) regulatory pathway, positioning it as a promising option for seizure management.
ImaginAb, Inc. is a clinical-stage biotechnology company specializing in immune oncology imaging, with a focus on enhancing precision medicine for cancer treatment. Founded in 2007 and based in Inglewood, California, the company develops engineered antibody fragments known as minibodies, which are designed for diagnostic imaging using Positron Emission Tomography (PET). ImaginAb's portfolio includes precision imaging products targeting CD8 and CD4 for immune-oncology applications, as well as PSMA PET for prostate cancer diagnostics. By creating highly targeted diagnostic imaging agents that retain the specificity of parental antibodies while being optimized for clinical use, ImaginAb addresses critical needs in patient selection and treatment monitoring for cancer immunotherapy.
SteadyMed Ltd. is a medical-device company pursuing innovative devices for delivering injectable therapeutic drugs for pain relief, diabetes and other chronic conditions.
The company's products are pre-filled drug-infusion patches that will be sold in conjunction with a series of pharma and device companies. Specific applications of the device will be tailored in accordance with the requirements of the company's partners.
Protagonist Pty Ltd is a biotechnology company based in Brisbane, Australia, focused on developing innovative therapeutics for inflammatory and metabolic conditions. The company specializes in discovering peptide and small molecule compounds that mimic or inhibit protein-protein interactions relevant to therapeutic applications. Protagonist Pty Ltd also aims to identify drug candidates for targets that have traditionally been resistant to small molecule discovery, creating arrays of molecules that explore biologically-relevant chemical diversity. Originally founded as Cytokine Mimetics Pty Ltd in 2001, it operates as a subsidiary of Protagonist Therapeutics, Inc., a clinical-stage biopharmaceutical company that develops peptide-based products to address unmet medical needs, particularly in hematology and gastroenterology. Protagonist Therapeutics has a portfolio that includes drug candidates in various stages of clinical trials for conditions such as beta-thalassemia and inflammatory bowel disease.
Aimmune Therapeutics is a company operating in the United States biotechnology industry. Its main focus is in the development of product candidates to combat peanut and other food allergies. The company's therapeutic approach, which is referred to as Characterized Oral Desensitization Immunotherapy, is a system designed to desensitize patients to food allergens using characterized biologic products, defined treatment protocols, and support services.
Advaxis, Inc. is a clinical-stage biotechnology company based in Princeton, New Jersey, focused on developing immunotherapies for cancer and infectious diseases. The company utilizes a unique platform based on live, attenuated bacteria engineered to deliver antigen/adjuvant fusion proteins, which aim to enhance the immune response against cancer. Advaxis is actively developing therapies for HPV-related cancers, including axalimogene filolisbac, currently in a Phase II clinical trial for head and neck cancer. Other products in development include ADXS-PSA for prostate cancer, ADXS-NEO for individualized treatment using neoantigens from a patient’s tumor, ADXS-HOT for generating robust anti-cancer immunity, and ADXS-HER2 targeting HER2-positive tumors. Advaxis has established collaborations and licensing agreements with various organizations to further advance its research and development efforts. The company was founded in 2002 and continues to progress its innovative treatments through multiple clinical trials.
Adverum Biotechnologies is a clinical-stage gene therapy company based in Redwood City, California, focused on developing innovative therapies for ocular and rare diseases. Its primary product candidate, ADVM-022, is designed for the treatment of wet age-related macular degeneration through a single intravitreal injection that aims to provide long-lasting therapeutic effects. The company's pipeline also includes ADVM-043, targeting alpha-1 antitrypsin deficiency, and ADVM-053, which is in preclinical development for hereditary angioedema. Adverum collaborates with Editas Medicine to utilize its proprietary AAV vectors for genome editing in inherited retinal diseases and has a partnership with Regeneron Pharmaceuticals to explore multiple ocular therapeutic targets. Originally known as Avalanche Biotechnologies, the company rebranded in May 2016 following a merger with Annapurna Therapeutics, further advancing its mission to deliver effective treatments for chronic and debilitating conditions.
Nexvet Australia Pty Ltd, a biopharmaceutical company, develops biological therapies for companion animals. It develops drugs in the areas of chronic pain, immune oncology, chronic inflammation, and allergy. Nexvet Australia Pty Ltd was formerly known as Nexvet Biopharma Pty Ltd. The company was founded in 2010 and is based in Melbourne, Australia with a biopharmaceutical manufacturing facility in Tullamore, Ireland. Nexvet Australia Pty Ltd operates as a subsidiary of Zoetis Inc.
Lumena Pharmaceuticals focuses on developing oral therapeutics for rare liver diseases, aiming to enhance liver function and alleviate disease symptoms. The company's clinical-stage product candidates are designed to selectively target a transporter in the intestine, minimizing systemic absorption and reducing the risk of toxicities. Lumena's lead candidate, LUM001, has been thoroughly evaluated in over 12 clinical studies involving more than 1,400 subjects, positioning it for rapid advancement in clinical trials for both pediatric and adult patients with various forms of cholestatic liver disease. In addition to its primary objective of creating novel treatments for rare liver conditions, Lumena's innovative therapeutic approach also holds potential for addressing metabolic diseases affecting the liver, which represent a significant and growing health concern among both children and adults.
ZS Pharma, Inc. is a biopharmaceutical company based in Coppell, Texas, focused on developing non-absorbed drugs for renal, cardiovascular, liver, and metabolic diseases. Established in 2008, the company specializes in highly selective ion-trap therapies, particularly its lead candidate, ZS-9. This innovative treatment employs proprietary zirconium silicate technology to create ion traps that can effectively reduce excess potassium levels in patients suffering from hyperkalemia, a serious condition often associated with chronic kidney disease, hypertension, diabetes, and heart failure. ZS-9 is currently undergoing late-stage clinical trials to evaluate its efficacy and safety. As of December 2015, ZS Pharma operates as a subsidiary of Zeneca, Inc.
Calithera Biosciences, Inc. is a clinical-stage biopharmaceutical company based in South San Francisco, California, dedicated to discovering and developing small molecule therapeutics for cancer treatment. The company focuses on targeting tumor metabolism and tumor immunology to create innovative anti-cancer drugs. Its lead product candidate, CB-839, is an inhibitor of glutaminase currently undergoing Phase II clinical trials for solid tumors. Additionally, Calithera is developing CB-1158, an oral arginase inhibitor in Phase I/II trials for hematology and oncology indications. The company is also working on other candidates, including CB-280 for cystic fibrosis and CB-708, an inhibitor of CD73. Calithera has established collaborations with major pharmaceutical firms, including Bristol-Myers Squibb and Incyte Corporation, to advance its research and development efforts. Founded in 2010, Calithera aims to address critical challenges in cancer therapy through its specialized drug development pipeline.
Private Equity Round in 2013
PTC Therapeutics, Inc. is a biopharmaceutical company dedicated to the discovery, development, and commercialization of orally administered small-molecule therapeutics for rare disorders and oncology. Notably, it offers Translarna and Emflaza for treating nonsense mutation Duchenne muscular dystrophy in ambulatory patients. The company is advancing several clinical programs, including Translarna for nonsense mutation aniridia and Dravet syndrome/CDKL5, as well as RG7916 and RO7034067 for spinal muscular atrophy. Additionally, PTC Therapeutics is developing PTC596 and PTC299, a small molecule targeting cancer treatment, and a gene therapy candidate, PTC-AADC, for Aromatic L-amino acid decarboxylase deficiency. Collaborations with various organizations, including F. Hoffman-La Roche and the Spinal Muscular Atrophy Foundation, enhance its research and commercialization efforts. Founded in 1998, PTC Therapeutics is headquartered in South Plainfield, New Jersey.
Ultragenyx Pharmaceutical is a biopharmaceutical company based in the United States that focuses on developing therapeutics for rare and ultra-rare diseases, particularly serious genetic conditions. The company identifies, acquires, and commercializes novel treatments, leveraging existing scientific knowledge to address unmet medical needs in this challenging area. Its portfolio includes several medicines such as Crysvita, which treats X-linked hypophosphatemia in both adults and pediatric patients, Dojolvi, and Mepsevii, indicated for Mucopolysaccharidosis VII. Ultragenyx aims to create impactful therapeutics by employing effective development strategies tailored to the complexities of rare diseases.
Private Equity Round in 2011
Puma Biotechnology is a biopharmaceutical company dedicated to the development and commercialization of innovative products aimed at improving cancer care. The company focuses on acquiring and advancing drug candidates that have completed initial clinical testing. Its primary product, NERLYNX, is an oral formulation of neratinib, which is used for treating HER2-positive breast cancer. In addition to neratinib, Puma is developing PB357, an irreversible tyrosine kinase inhibitor that targets epidermal growth factor receptors HER1, HER2, and HER4. The company has established a licensing agreement with Pfizer for the development and commercialization of these drug candidates. Founded in 2010 and headquartered in Los Angeles, California, Puma Biotechnology is committed to enhancing treatment outcomes for patients with various forms of cancer.