Glycomine, Inc. is a biotechnology company based in San Carlos, California, focused on developing nanomedicines for rare genetic disorders, particularly those related to protein and lipid glycosylation. Founded in 2014, Glycomine aims to address the significant unmet medical needs of patients suffering from approximately 7,000 rare disorders, many of which currently lack FDA-approved treatments. The company specializes in creating orphan drugs that combine replacement therapies—such as substrates, enzymes, and proteins—with innovative intracellular delivery systems. These bio-nano materials and ligands are designed to effectively target and deliver therapeutic molecules to the relevant cells in clinically significant organs, thereby enhancing treatment efficacy for patients with debilitating conditions caused by metabolic disorders and protein misfolding.
Aura Biosciences, Inc. is a clinical-stage biotechnology company based in Cambridge, Massachusetts, focused on developing therapies to target and destroy cancer cells using viral nanoparticle conjugates. The company’s lead product, AU-011, utilizes viral nanoparticles modeled on the human papillomavirus, conjugated with infrared-activated small molecules, specifically for the treatment of ocular melanoma, a primary eye cancer. By employing nanotechnology, Aura aims to safely eliminate cancer locally, particularly in early-stage cases, to prevent progression to more severe forms of the disease, such as metastasis to the liver. Additionally, the company is advancing its bel-sar candidate for treating primary choroidal melanoma and exploring applications in bladder cancer. Founded in 2007, Aura Biosciences is committed to developing precision immunotherapies that preserve the function of organs affected by cancer.
Reneo Pharmaceuticals develops therapies for patients with rare genetic mitochondrial diseases, which impact cellular metabolism and energy production. Founded in 2014 and based in San Diego, California, the company is focused on creating treatments that enhance mitochondrial function, thereby improving muscle health and overall quality of life for affected individuals. Its lead product candidate, REN001, is an oral medication designed to modulate genes essential for energy metabolism and the production of adenosine triphosphate (ATP), the primary energy source for cellular processes. By addressing the underlying metabolic deficiencies in these patients, Reneo aims to prevent muscle injury, weakness, and wasting, ultimately enhancing daily functioning for those with these orphan diseases.
Glycomine, Inc. is a biotechnology company based in San Carlos, California, focused on developing nanomedicines for rare genetic disorders, particularly those related to protein and lipid glycosylation. Founded in 2014, Glycomine aims to address the significant unmet medical needs of patients suffering from approximately 7,000 rare disorders, many of which currently lack FDA-approved treatments. The company specializes in creating orphan drugs that combine replacement therapies—such as substrates, enzymes, and proteins—with innovative intracellular delivery systems. These bio-nano materials and ligands are designed to effectively target and deliver therapeutic molecules to the relevant cells in clinically significant organs, thereby enhancing treatment efficacy for patients with debilitating conditions caused by metabolic disorders and protein misfolding.
Mirum Pharmaceuticals is a biopharmaceutical company focused on the development and commercialization of therapies for debilitating liver diseases. It is involved in the development of Maralixibat for the treatment of pediatric patients with progressive familial intrahepatic cholestasis, or PFIC, and Alagille syndrome, or ALGS. The company is also involved in the development of volixibat for the treatment of adult patients with cholestatic liver diseases.
CuraSen Therapeutics Inc. is a biotechnology company focused on developing therapies for neurodegenerative diseases. The company specializes in small molecule drugs that target a novel mechanism in the brain, aiming to restore function, improve symptoms, and modify the progression of conditions such as Parkinson's disease, Alzheimer's disease, and less common orphan neurodegenerative disorders. Founded in 2016, CuraSen Therapeutics is headquartered in Los Altos, California.
VelosBio, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, focused on developing innovative antibody-drug conjugates (ADCs) to treat hematological cancers and solid tumors. The company specializes in therapies targeting receptor tyrosine kinase-like orphan receptor 1 (ROR1), a cell-surface protein commonly found in various tumors but absent in normal tissues, making it a promising target for cancer treatment. Its lead product, VLS-101, is designed as a ROR1-directed ADC aimed at patients with both hematologic and solid tumor malignancies. Founded in 2017, VelosBio has demonstrated positive preclinical results for its ROR1-directed therapeutics, paving the way for potential use in monotherapy or in combination with other treatments across a wide range of cancers. As of late 2020, VelosBio operates as a subsidiary of Merck & Co., Inc.
Minoryx Therapeutics is a biotechnology company based in Mataro, Spain, founded in 2011. The company specializes in discovering and developing innovative treatments for life-threatening rare diseases, with a particular focus on pediatric neurometabolic conditions of genetic origin. Minoryx is known for its development of pharmacological chaperones, a new generation of small molecule drugs designed to restore protein functionality, thereby addressing unmet medical needs in genetic diseases that severely affect the central nervous system. Additionally, Minoryx engages in repositioning-based projects to explore alternative therapeutic options for these challenging conditions.
4D Molecular Therapeutics is a clinical-stage gene therapy company based in Emeryville, California, that specializes in developing targeted gene therapy products using evolved adeno-associated virus vectors. The company has a diverse portfolio focusing on three main therapeutic areas: ophthalmology, cardiology, and pulmonology. Key product candidates include 4D-125, currently in a Phase I/II clinical trial for treating X-linked retinitis pigmentosa; 4D-110, in a Phase I trial for choroideremia; and 4D-310, also in a Phase I/II trial for Fabry disease. Additionally, 4D Molecular Therapeutics is developing investigational new drug candidates such as 4D-150 for wet age-related macular degeneration and 4D-710 for cystic fibrosis lung disease. The company aims to address unmet medical needs through its innovative gene therapies.
Aura Biosciences, Inc. is a clinical-stage biotechnology company based in Cambridge, Massachusetts, focused on developing therapies to target and destroy cancer cells using viral nanoparticle conjugates. The company’s lead product, AU-011, utilizes viral nanoparticles modeled on the human papillomavirus, conjugated with infrared-activated small molecules, specifically for the treatment of ocular melanoma, a primary eye cancer. By employing nanotechnology, Aura aims to safely eliminate cancer locally, particularly in early-stage cases, to prevent progression to more severe forms of the disease, such as metastasis to the liver. Additionally, the company is advancing its bel-sar candidate for treating primary choroidal melanoma and exploring applications in bladder cancer. Founded in 2007, Aura Biosciences is committed to developing precision immunotherapies that preserve the function of organs affected by cancer.
Kezar Life Sciences, Inc. is a clinical-stage biotechnology company based in South San Francisco, California, focused on developing novel small molecule therapeutics for unmet medical needs in autoimmunity and cancer. The company’s lead candidate, KZR-616, is a selective immunoproteasome inhibitor currently undergoing Phase 2 clinical trials for multiple autoimmune conditions, including lupus nephritis and autoimmune hemolytic anemia, as well as Phase 1b/2 trials for systemic lupus erythematosus. In addition to KZR-616, Kezar is advancing preclinical products, including KZR-TBD, aimed at treating oncology and autoimmune disorders. Founded in 2015, Kezar Life Sciences leverages insights into protein homeostasis, particularly the interplay between protein degradation and secretion, to drive its drug discovery efforts.
Sentien Biotechnologies, Inc. develops proprietary cell compositions and GMP manufacturing methods to advance combination products that unlock the potential of cell therapy. It provides the Sentinel, a device that actively treats patients during a dialysis dose by supplementing patient blood with MSC-derived secretions, a combinational mixture of natural anti-inflammatory and trophic molecules that can attenuate the immune dysfunction of acute renal failure while promoting a tissue repairing response. The company was founded in 2008 and is headquartered in Medford, Massachusetts.
Sentieon, Inc. is a biotechnology company founded in 2014 and headquartered in Mountain View, California, specializing in cloud-based bioinformatics solutions for the life sciences and clinical markets. The company develops a suite of bioinformatics tools designed for the efficient processing of genomics data, focusing on high accuracy and computational efficiency. Key products include Sentieon DNAseq, which facilitates germline DNA analysis, and Sentieon TNseq, used for detecting somatic variants in tumor-normal samples. Sentieon's software solutions are characterized by their scalability, deployability, and upgradability, allowing them to adapt to various user needs. The company's advanced algorithms and robust software implementation ensure rapid turnaround times and consistent results, benefiting scientists, clinicians, and patients by enhancing precision in genomic data analysis.
Glycomine, Inc. is a biotechnology company based in San Carlos, California, focused on developing nanomedicines for rare genetic disorders, particularly those related to protein and lipid glycosylation. Founded in 2014, Glycomine aims to address the significant unmet medical needs of patients suffering from approximately 7,000 rare disorders, many of which currently lack FDA-approved treatments. The company specializes in creating orphan drugs that combine replacement therapies—such as substrates, enzymes, and proteins—with innovative intracellular delivery systems. These bio-nano materials and ligands are designed to effectively target and deliver therapeutic molecules to the relevant cells in clinically significant organs, thereby enhancing treatment efficacy for patients with debilitating conditions caused by metabolic disorders and protein misfolding.
Minoryx Therapeutics is a biotechnology company based in Mataro, Spain, founded in 2011. The company specializes in discovering and developing innovative treatments for life-threatening rare diseases, with a particular focus on pediatric neurometabolic conditions of genetic origin. Minoryx is known for its development of pharmacological chaperones, a new generation of small molecule drugs designed to restore protein functionality, thereby addressing unmet medical needs in genetic diseases that severely affect the central nervous system. Additionally, Minoryx engages in repositioning-based projects to explore alternative therapeutic options for these challenging conditions.
Aura Biosciences, Inc. is a clinical-stage biotechnology company based in Cambridge, Massachusetts, focused on developing therapies to target and destroy cancer cells using viral nanoparticle conjugates. The company’s lead product, AU-011, utilizes viral nanoparticles modeled on the human papillomavirus, conjugated with infrared-activated small molecules, specifically for the treatment of ocular melanoma, a primary eye cancer. By employing nanotechnology, Aura aims to safely eliminate cancer locally, particularly in early-stage cases, to prevent progression to more severe forms of the disease, such as metastasis to the liver. Additionally, the company is advancing its bel-sar candidate for treating primary choroidal melanoma and exploring applications in bladder cancer. Founded in 2007, Aura Biosciences is committed to developing precision immunotherapies that preserve the function of organs affected by cancer.
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