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Decibel Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, one of the largest areas of unmet need in medicine. Decibel has built a proprietary platform that integrates single-cell genomics and bioinformatic analyses, precision gene therapy technologies and expertise in inner ear biology. Decibel is leveraging its platform to advance gene therapies designed to selectively replace genes for the treatment of congenital, monogenic hearing loss and to regenerate inner ear hair cells for the treatment of acquired hearing and balance disorders. Decibel`s pipeline, including its lead gene therapy program, DB-OTO, to treat congenital, monogenic hearing loss, is designed to deliver on our vision of a world in which the privileges of hearing and balance are available to all.
Brain diseases collectively represent one of the greatest challenges of our generation, affecting upwards of 1.5 billion people. However, treatments for many of the most prevalent brain disorders have been dominated by a single class of drugs, such as serotonin targeting antidepressants for MDD, leaving patients with a high degree of unmet medical need given the lack of diverse treatment options and mechanisms of action. We are taking a fundamentally different approach to the way treatments for brain diseases are developed. Our pipeline is comprised of programs pursuing therapeutically relevant targets implicated in Central Nervous System (CNS) diseases, each targeting a novel mechanism of action with best-in-class pharmacology. Our work is supported by our Precision Toolbox, which integrates a suite of translational, clinical, and computational tools to generate insights that can enable precision medicine approaches. Neumora is relentless in its commitment to discovering, developing and commercializing novel therapies for the 1.5 billion people living with brain diseases.
GangaGen Inc is a Palo Alto, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Spruce is a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for rare endocrine disorders with significant unmet medical need. Our wholly-owned product candidate, tildacerfont, is a CRF1 receptor antagonist currently in late-stage trials in adult patients with classic congenital adrenal hyperplasia (CAH), with a planned Phase 2 trial in pediatric classic CAH. We aim to advance the treatment paradigm for classic CAH with a well-tolerated, non-steroidal approach designed to offer markedly improved disease control and reduced steroid burden for patients. Tildacerfont may also benefit patients with other disorders characterized by elevated levels of or hyperresponsiveness to adrenocorticotropic hormone (ACTH), a hormone involved in the production of cortisol, including a rare form of polycystic ovary syndrome (PCOS).
Zynerba (NASDAQ: ZYNE) is dedicated to improving the lives of people with rare and near rare neuropsychiatric disorders where there is a high unmet medical need by pioneering the development and commercialization of next-generation pharmaceutically-produced cannabinoid therapeutics formulated for transdermal delivery. Cannabinoids interact with specific receptors throughout the body to produce pharmacologic effects, primarily in the CNS and immune systems. Transdermal delivery has the potential to reduce adverse effects associated with oral dosing because it allows the drug to be absorbed through the skin directly into the bloodstream, avoiding the gastrointestinal tract and lessening the opportunity for GI-related adverse events. It also avoids first-pass liver metabolism, potentially enabling lower dosage levels of active pharmaceutical ingredients with a higher bioavailability and an improved safety profile. Zynerba`s lead patent-protected product candidate in clinical development is ZYN002: ZYN002 is the first and only pharmaceutically-produced cannabidiol (CBD) formulated as a permeation-enhanced gel for transdermal delivery. Through a proprietary combination of our pharmaceutically-produced CBD and permeation enhancers, we believe we can effectively deliver CBD through the layers of the epidermis and into the circulatory system. The company is currently assessing ZYN002 in Fragile X syndrome and certain adult and pediatric refractory epilepsies. These include adult refractory focal epilepsy and developmental and epileptic encephalopathies (DEE), a heterogeneous group of rare and ultra-rare epilepsy syndromes associated with severe cognitive impairment and behavioral disturbances.