Frazier Life Sciences

Jade Biosciences is a biopharmaceutical company dedicated to developing innovative therapies for unmet needs in autoimmune diseases. Its lead asset, JADE-001, targets the APRIL pathway for treating IgA nephropathy, with initial clinical trials planned. Additionally, Jade has two preclinical antibody discovery programs, JADE-002 and JADE-003. The company was founded upon assets licensed from Paragon Therapeutics.

Attovia Therapeutics is a biotherapeutics pipeline developer focused on immune-mediated illness and oncology. The company uses the nanobody platform to create tiny format binders with low picomolar affinity, increased selectivity, quicker internalization, and rapid tissue penetration, thereby assisting cancer patients' treatment.

Trevi Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in New Haven, Connecticut, dedicated to developing and commercializing nalbuphine ER, an oral extended-release formulation. The company is focused on treating serious neurologically mediated conditions, specifically chronic pruritus, chronic cough in patients with idiopathic pulmonary fibrosis, and levodopa-induced dyskinesia in individuals with Parkinson's disease. Trevi is currently conducting a Phase 2b/3 clinical trial, known as the PRISM trial, for nalbuphine ER, targeting severe pruritus associated with prurigo nodularis. The conditions being addressed share similar underlying mechanisms that involve opioid receptors in both the central and peripheral nervous systems. Founded in 2011, Trevi Therapeutics aims to provide innovative treatment options for patients suffering from these challenging health issues.

Palvella Therapeutics, based in Wayne, Pennsylvania, is a clinical-stage biopharmaceutical company established in 2015. It focuses on developing and commercializing targeted therapies for serious and rare genetic skin diseases, specifically genodermatoses. The company's lead product, QTORIN, is a specialized anhydrous gel designed to deliver active ingredients effectively into the skin, addressing the underlying causes of various dermatological conditions. Palvella's pipeline includes candidates aimed at treating microcystic lymphatic malformations and cutaneous venous malformations, utilizing its proprietary QTORIN platform to offer innovative solutions for underserved patient populations.

Maze Therapeutics is a biotechnology company based in South San Francisco, California, focused on developing innovative medicines that leverage the effects of rare genetic variants to address unmet medical needs. Founded in 2017 and originally known as Modulus Therapeutics, the company utilizes its proprietary Compass platform to identify genetic variants linked to diseases and map them to the biological pathways that drive these conditions in specific patient populations. By integrating large-scale human genetics data and functional genomics with various drug discovery methods, Maze Therapeutics aims to reveal modifier genes that offer protective benefits. This approach enhances the understanding of target biology and informs the development of novel therapies, ultimately enabling pharmaceutical companies to create effective treatments.

35Pharma is a biopharmaceutical company focused on designing and developing innovative biologics to address diseases with significant unmet medical needs, particularly pulmonary hypertension and musculoskeletal disorders. The company leverages its expertise in protein engineering and a comprehensive understanding of transforming growth factor (TGF)-beta structure-function relationships to create highly effective ligand traps. These advancements aim to facilitate the development of targeted biologics for treating patients suffering from cardio-pulmonary and metabolic diseases.

Crescent Biopharma is a biotechnology firm focused on developing novel precision-engineered medicines targeting verified biology to improve care for patients with solid tumors.

Eledon Pharmaceuticals, Inc. is a clinical-stage biotechnology company based in Irvine, California, specializing in the development of innovative treatments for patients with disorders of the ear, nose, and throat, as well as for those undergoing organ or cellular transplantation and individuals with autoimmune and neurodegenerative diseases. The company’s lead product, OP0201, is a surfactant-based nasal aerosol designed for patients at risk for or suffering from otitis media. Additionally, Eledon has developed a foam-based drug delivery technology, OP0101 and OP0102, aimed at delivering medications to the ear, nose, and sinus cavities. The company also focuses on the CD40L pathway, with its main compound in development, tegoprubart, being an IgG1 anti-CD40L antibody that shows promise for treating autoimmune diseases and supporting organ transplants. Eledon Pharmaceuticals was formerly known as Novus Therapeutics, Inc. before its rebranding in January 2021.

Alpha9 Theranostics is a biopharma company that focuses on radiotherapeutics for the treatment of solid and hematologic malignancies. The company is creating a strong pipeline of radiopharmaceuticals to target solid and hematologic malignancies. Its systematic approach to radiotherapeutic design is fueled by a toolbox of technologies and chemistries, which offers broad potential for expansion into a multitude of oncology targets. Alpha9 Theranostics was founded in 2019 and is headquartered in Vancouver, Canada.

Egetis Therapeutics AB is a pharmaceutical drug development company based in Stockholm, Sweden, that specializes in late-stage projects targeting serious rare diseases with significant unmet medical needs, particularly within the orphan drug segment. The company is actively developing several innovative therapies, including PledOx, which is undergoing Phase III clinical trials to protect patients from chemotherapy-induced nerve damage while preserving the anticancer effects. Additionally, Egetis is working on Emcitate, a treatment for MCT8 deficiency, a rare genetic condition affecting thyroid hormone signaling, and Aladote, which aims to mitigate the risk of acute liver injury from acetaminophen poisoning. Founded in 2006, Egetis Therapeutics was previously known as PledPharma AB until its name change in December 2020.

GondolaBio researches, develops and manufactures pharmaceutical products.

MBX Biosciences, Inc. is a biotechnology company based in Carmel, Indiana, specializing in the development of therapeutics for rare endocrine disorders. Established in 2018, the company focuses on creating peptides aimed at treating genetic diseases that affect glandular hormones, which play a crucial role in the body’s chemical messaging system. By targeting conditions with inadequate treatment options, MBX Biosciences is dedicated to discovering, developing, and commercializing innovative endocrine therapeutics.

Jade Biosciences is a biopharmaceutical company dedicated to developing innovative therapies for unmet needs in autoimmune diseases. Its lead asset, JADE-001, targets the APRIL pathway for treating IgA nephropathy, with initial clinical trials planned. Additionally, Jade has two preclinical antibody discovery programs, JADE-002 and JADE-003. The company was founded upon assets licensed from Paragon Therapeutics.

Savara is an orphan lung disease company. Savara’s pipeline comprises Molgradex, an inhaled granulocyte-macrophage colony-stimulating factor, or GM-CSF, in Phase 3 development for autoimmune pulmonary alveolar proteinosis, or aPAP, in Phase 2a development for nontuberculous mycobacteria, or NTM, lung infection, and in preparation for Phase 2a development in cystic fibrosis, or CF, affected individuals with chronic NTM lung infection; and AeroVanc, a Phase 3 stage inhaled vancomycin for treatment of persistent methicillin resistant staphylococcus aureus, or MRSA, lung infection in CF. Savara’s strategy involves expanding its pipeline of potentially best-in-class products through indication expansion, strategic development partnerships and product acquisitions, with the goal of becoming a leading company in its field. The most recent acquisition is aerosolized amikacin/fosfomycin, a Phase 2 ready, proprietary combination antibiotic, which has demonstrated potent and broad-spectrum antibacterial activity against highly drug resistant pathogens. Savara’s management team has significant experience in orphan drug development and pulmonary medicine, identifying unmet needs, developing and acquiring new product candidates, and effectively advancing them to approvals and commercialization.

Mereo Biopharma, established in London in 2015, specializes in acquiring and advancing late-stage drug candidates from larger pharmaceutical companies. These candidates, often overlooked due to resource constraints, target rare and specialized diseases with significant unmet medical needs. Mereo's strategy involves rapidly progressing these assets through clinical development, either by partnering or divesting them, or directly commercializing them, particularly in orphan disease indications. The company combines operational efficiency with financial resources, leveraging a unique partnership with a leading global CRO, ICON, to conduct comprehensive clinical studies. Mereo's focus is on transforming the lives of patients worldwide by developing innovative medicines that can significantly improve their quality of life.

Disc Medicine, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative treatments for severe hematologic disorders. Founded in 2017, the company specializes in human genetics-based therapeutics that aim to improve red blood cell production in patients suffering from various hematologic diseases. Disc Medicine's pipeline includes therapeutic candidates targeting critical pathways involved in red blood cell biology, such as heme biosynthesis and iron homeostasis. Notable programs include bitopertin, which is being developed for conditions like erythropoietic porphyrias and Diamond-Blackfan anemia. Through its research and development efforts, Disc Medicine seeks to transform the lives of patients affected by these challenging disorders.

Attovia Therapeutics is a biotherapeutics pipeline developer focused on immune-mediated illness and oncology. The company uses the nanobody platform to create tiny format binders with low picomolar affinity, increased selectivity, quicker internalization, and rapid tissue penetration, thereby assisting cancer patients' treatment.

Enlaza Therapeutics is a biotechnology research company founded in 2020 and based in La Jolla, California. The company focuses on developing novel protein therapeutics aimed at providing more effective and safer treatment options for patients. Utilizing advanced synthetic biology technology, Enlaza's platform enables site-specific covalent coupling driven by drug binding. This innovative approach allows for the creation of first-in-class covalent biologics, which are designed to offer enhanced efficacy and improved safety profiles compared to existing treatments.

Diagonal Therapeutics is a biotechnology company specializing in the discovery and development of agonist antibodies. Its innovative platform integrates proprietary computational and experimental methods to overcome historical challenges in agonist antibody drug development, enabling the delivery of therapies for conditions such as hereditary hemorrhagic telangiectasia and pulmonary arterial hypertension.

Applied Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in New York, focused on developing innovative therapies for serious medical conditions with significant unmet needs. The company's lead product candidate, AT-007, is in phase I/II clinical trials for the treatment of galactosemia, while AT-001 is undergoing phase II trials for diabetic cardiomyopathy and phase I trials for diabetic peripheral neuropathy. Additionally, AT-003 is in phase I trials for diabetic retinopathy. The company also has preclinical candidates, such as AT-104, aimed at treating orphan hematological oncology conditions. Founded in 2016, Applied Therapeutics leverages advancements in technology and regulatory processes to create drugs targeting well-established molecular mechanisms.

Sudo Biosciences is a biopharmaceutical company focused on developing innovative medicines that target the tyrosine kinase 2 (TYK2) pseudokinase domain, an important component in cytokine signaling pathways associated with various immune-mediated inflammatory conditions. The company has a pipeline of next-generation TYK2 inhibitors, which includes a candidate designed to penetrate the brain for the treatment of multiple sclerosis and neurodegenerative diseases characterized by neuroinflammation, as well as a topical candidate aimed at addressing immune-mediated dermatologic diseases. Sudo Biosciences aims to provide effective and affordable treatment options for patients suffering from these conditions.

PepGen is a clinical-stage biotechnology company based in Oxford, United Kingdom, focused on advancing nucleic acid therapeutics, particularly antisense oligonucleotides. Founded in 2018, the company aims to enhance the delivery and efficacy of these therapies through its proprietary Enhanced Delivery Oligonucleotide (EDO) platform. This innovative platform utilizes engineered peptides designed to improve tissue penetration, cellular uptake, and nuclear delivery of oligonucleotides. PepGen's technology has shown promise in preclinical studies, demonstrating the ability to transport therapeutics effectively into various target tissues, including smooth, skeletal, and cardiac muscle, as well as the central nervous system. The company's mission is to unlock the full potential of nucleic acid therapeutics to transform the treatment landscape for severe neuromuscular and neurological diseases.

Adverum Biotechnologies is a clinical-stage gene therapy company focused on developing innovative treatments for ocular and rare diseases. Headquartered in Redwood City, California, the company aims to establish gene therapy as a new standard of care, aspiring to provide functional cures that restore vision and prevent blindness. Its pipeline includes several product candidates, notably ADVM-022, which targets wet age-related macular degeneration through a single intravitreal injection designed to deliver long-term therapeutic effects. Other candidates include ADVM-043 for alpha-1 antitrypsin deficiency and ADVM-053 for hereditary angioedema. Adverum collaborates with partners like Editas Medicine and Regeneron Pharmaceuticals to leverage advanced AAV vectors and develop treatments for various inherited retinal diseases and ocular therapeutic targets. Founded in 2006, the company was previously known as Avalanche Biotechnologies and rebranded in 2016.

Silence Therapeutics plc is a biotechnology company based in London, specializing in the discovery and development of innovative RNA therapeutics. The company focuses on utilizing short interfering RNA (siRNA) technology to modulate gene expression and address various medical conditions, including hematology, cardiovascular diseases, and rare metabolic disorders. Its pipeline includes several product candidates, such as SLN124, aimed at treating iron overload disorders by silencing the TMPRSS6 gene, and SLN360, which targets the LPA gene to reduce the risk of cardiovascular diseases. Silence Therapeutics has established strategic collaborations with major organizations, including AstraZeneca, to advance the development of siRNA therapeutics across multiple disease areas. The company leverages its proprietary genetic toolkit and delivery systems to optimize therapeutic outcomes, harnessing the body's natural biological mechanisms for effective treatment.

Sudo Biosciences is a biopharmaceutical company focused on developing innovative medicines that target the tyrosine kinase 2 (TYK2) pseudokinase domain, an important component in cytokine signaling pathways associated with various immune-mediated inflammatory conditions. The company has a pipeline of next-generation TYK2 inhibitors, which includes a candidate designed to penetrate the brain for the treatment of multiple sclerosis and neurodegenerative diseases characterized by neuroinflammation, as well as a topical candidate aimed at addressing immune-mediated dermatologic diseases. Sudo Biosciences aims to provide effective and affordable treatment options for patients suffering from these conditions.

Lassen Therapeutics specializes in developing monoclonal antibodies aimed at treating fibrosis, rare diseases, and oncology. The company’s primary candidate, LASN01, targets the IL-11 receptor alpha, a critical component in the IL-6 family of cytokines. IL-11 is a significant mediator of fibrosis and plays an essential role in tumor growth and the organization of the tumor microenvironment. By blocking IL-11's activity, Lassen Therapeutics seeks to provide more effective treatment options compared to traditional approaches that target other factors like TGF-β and CTGF. Through innovative antibody technologies, the company aims to deliver breakthrough therapeutics that improve the lives of patients facing serious health challenges.

Egetis Therapeutics AB is a pharmaceutical drug development company based in Stockholm, Sweden, that specializes in late-stage projects targeting serious rare diseases with significant unmet medical needs, particularly within the orphan drug segment. The company is actively developing several innovative therapies, including PledOx, which is undergoing Phase III clinical trials to protect patients from chemotherapy-induced nerve damage while preserving the anticancer effects. Additionally, Egetis is working on Emcitate, a treatment for MCT8 deficiency, a rare genetic condition affecting thyroid hormone signaling, and Aladote, which aims to mitigate the risk of acute liver injury from acetaminophen poisoning. Founded in 2006, Egetis Therapeutics was previously known as PledPharma AB until its name change in December 2020.

Soleno Therapeutics, Inc. is a clinical-stage biopharmaceutical company dedicated to the development and commercialization of innovative therapeutics for rare diseases. The company's primary focus is on its lead candidate, Diazoxide Choline Controlled Release (DCCR), a once-daily oral tablet designed for the treatment of Prader-Willi Syndrome (PWS). DCCR contains diazoxide choline, a choline salt of diazoxide, which is a benzothiadiazine. Currently, the product is being evaluated in a Phase III clinical development program. Established in 1999 and headquartered in Redwood City, California, Soleno Therapeutics was formerly known as Capnia, Inc. before rebranding in May 2017.

AN2 Therapeutics, Inc. is a biopharmaceutical company based in Menlo Park, California, founded in 2017. The company is dedicated to the research, development, and commercialization of innovative medicines aimed at addressing infectious diseases. AN2 Therapeutics is focused on developing epetraborole, a once-daily oral treatment for patients suffering from chronic non-tuberculous mycobacterial lung disease, which represents a significant unmet medical need. Additionally, the company has established a strategic partnership with Brii Biosciences to enhance its development efforts.

Radionetics Oncology is a biopharmaceutical company dedicated to the discovery and development of innovative radiotherapeutics aimed at treating various oncology indications. The company specializes in creating non-peptide and small molecule agents that facilitate the delivery of therapeutic radioisotopes, targeting solid tumors. By advancing these novel treatments, Radionetics Oncology aims to enhance the options available to medical professionals in combating cancer.

ORIC Pharmaceuticals is a clinical-stage biopharmaceutical company focused on developing therapies to overcome resistance in cancer treatments. Founded in 2014 and based in South San Francisco, the company’s lead product candidate, ORIC-101, is a small molecule antagonist of the glucocorticoid receptor, which is associated with resistance to various cancer therapies in solid tumors. Its second candidate, ORIC-533, is an orally bioavailable small molecule inhibitor of CD73, targeting a critical component of the adenosine pathway linked to resistance against chemotherapy and immunotherapy. In addition to these products, ORIC is advancing multiple precision medicines aimed at addressing other mechanisms of cancer resistance. The company’s founders, Charles Sawyers and Scott Lowe, have extensive experience in identifying novel cancer targets that have led to innovative therapies.

Attovia Therapeutics is a biotherapeutics pipeline developer focused on immune-mediated illness and oncology. The company uses the nanobody platform to create tiny format binders with low picomolar affinity, increased selectivity, quicker internalization, and rapid tissue penetration, thereby assisting cancer patients' treatment.

Carmot Therapeutics, Inc. is a clinical-stage biotechnology company based in Berkeley, California, with an additional office in San Francisco. Founded in 2008, the company focuses on discovering and developing therapies for metabolic diseases, including obesity and diabetes. Carmot employs a proprietary drug discovery approach known as Chemotype Evolution, which enables the exploration of novel chemical and biological frontiers. The company is advancing a portfolio of clinical-stage drug candidates, including CT-388, a once-weekly injectable dual GLP-1/GIP receptor agonist; CT-996, a once-daily oral GLP-1 receptor agonist; and CT-868, a once-daily injectable dual GLP-1/GIP receptor agonist for treating type 1 diabetes in patients with overweight or obesity. These innovative therapies aim to enhance glucose clearance and promote weight loss, addressing the fundamental causes of metabolic diseases.

Krystal Biotech is a biopharmaceutical company based in Pittsburgh, Pennsylvania, focused on developing gene therapies for patients with rare skin diseases in the United States. Its primary candidate, beremagene geperpavec (B-VEC), is designed to treat dystrophic epidermolysis bullosa and has completed Phase I/II clinical trials. Additionally, the company is advancing KB105, which is in Phase I/II trials for autosomal recessive congenital ichthyosis, while KB301 and KB104 are in preclinical stages targeting aesthetic skin conditions and Netherton Syndrome, respectively. Krystal Biotech utilizes its proprietary STAR-D gene therapy platform to create innovative treatments that offer significant clinical benefits. Founded in 2015, the company aims to broaden its pipeline to address various dermatological conditions in the future.

Alpha9 Theranostics is a biopharma company that focuses on radiotherapeutics for the treatment of solid and hematologic malignancies. The company is creating a strong pipeline of radiopharmaceuticals to target solid and hematologic malignancies. Its systematic approach to radiotherapeutic design is fueled by a toolbox of technologies and chemistries, which offers broad potential for expansion into a multitude of oncology targets. Alpha9 Theranostics was founded in 2019 and is headquartered in Vancouver, Canada.

MBX Biosciences, Inc. is a biotechnology company based in Carmel, Indiana, specializing in the development of therapeutics for rare endocrine disorders. Established in 2018, the company focuses on creating peptides aimed at treating genetic diseases that affect glandular hormones, which play a crucial role in the body’s chemical messaging system. By targeting conditions with inadequate treatment options, MBX Biosciences is dedicated to discovering, developing, and commercializing innovative endocrine therapeutics.

VectivBio AG is a clinical-stage biotechnology company based in Basel, Switzerland, founded in 2019 as a spinout from Therachon. The company specializes in the discovery, development, and commercialization of innovative treatments for severe rare diseases, particularly those with significant unmet medical needs. Its lead product candidate, Apraglutide, is a synthetic GLP-2 analog designed to enhance the intestine’s ability to absorb fluids and nutrients, thereby reducing the reliance on parenteral support in patients with short bowel syndrome. VectivBio is dedicated to developing best-in-disease therapies that aim to meaningfully improve the lives of patients and their families, providing more than just incremental advancements over existing standard-of-care options.

Enlaza Therapeutics is a biotechnology research company founded in 2020 and based in La Jolla, California. The company focuses on developing novel protein therapeutics aimed at providing more effective and safer treatment options for patients. Utilizing advanced synthetic biology technology, Enlaza's platform enables site-specific covalent coupling driven by drug binding. This innovative approach allows for the creation of first-in-class covalent biologics, which are designed to offer enhanced efficacy and improved safety profiles compared to existing treatments.

Immunovant, headquartered in New York, is a clinical-stage biopharmaceutical company dedicated to improving the lives of patients with autoimmune diseases. Its primary focus is on developing monoclonal antibodies, notably IMVT-1401, a fully human antibody that targets the neonatal Fc receptor (FcRn). This investigational product is designed for subcutaneous injection and aims to address various IgG-mediated autoimmune conditions. IMVT-1401 is currently in Phase IIa clinical trials for myasthenia gravis and thyroid eye disease, with Phase II trials also initiated for warm autoimmune hemolytic anemia. Additionally, the company is developing batoclimab and IMVT-1402, both targeting FcRn.

Sudo Biosciences is a biopharmaceutical company focused on developing innovative medicines that target the tyrosine kinase 2 (TYK2) pseudokinase domain, an important component in cytokine signaling pathways associated with various immune-mediated inflammatory conditions. The company has a pipeline of next-generation TYK2 inhibitors, which includes a candidate designed to penetrate the brain for the treatment of multiple sclerosis and neurodegenerative diseases characterized by neuroinflammation, as well as a topical candidate aimed at addressing immune-mediated dermatologic diseases. Sudo Biosciences aims to provide effective and affordable treatment options for patients suffering from these conditions.

Allakos Inc. is a clinical-stage biopharmaceutical company based in Redwood City, California, founded in 2012. The company focuses on developing antibody-based therapeutics aimed at treating various conditions linked to dysregulation of the T-helper type 2 immune response, such as allergic diseases and chronic inflammation. Its lead product, antolimab (AK002), targets conditions including eosinophilic gastritis, eosinophilic gastroenteritis, chronic urticaria, indolent systemic mastocytosis, and severe allergic conjunctivitis. Additionally, Allakos is developing AK006, an antibody that targets Siglec-6, an inhibitory receptor found on mast cells. By binding to Siglec-6, AK006 is designed to enhance the receptor's natural inhibitory function, thereby reducing mast cell activation and associated inflammatory responses.

Trevi Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in New Haven, Connecticut, dedicated to developing and commercializing nalbuphine ER, an oral extended-release formulation. The company is focused on treating serious neurologically mediated conditions, specifically chronic pruritus, chronic cough in patients with idiopathic pulmonary fibrosis, and levodopa-induced dyskinesia in individuals with Parkinson's disease. Trevi is currently conducting a Phase 2b/3 clinical trial, known as the PRISM trial, for nalbuphine ER, targeting severe pruritus associated with prurigo nodularis. The conditions being addressed share similar underlying mechanisms that involve opioid receptors in both the central and peripheral nervous systems. Founded in 2011, Trevi Therapeutics aims to provide innovative treatment options for patients suffering from these challenging health issues.

Lexeo Therapeutics is a biotechnology company specializing in adeno-associated virus (AAV)-mediated therapies for both rare and common diseases caused by single gene mutations. Headquartered in New York, the company maintains an integrated pipeline developed in collaboration with Weill Cornell Medicine's Department of Genetic Medicine. Lexeo focuses on advancing its clinical programs towards commercialization while continuing to research new therapies for various patient populations and unmet medical needs.