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Crinetics Pharmaceuticals is a clinical stage pharmaceutical company focused on the discovery, development, and commercialization of novel therapeutics for rare endocrine diseases and endocrine-related tumors. The company`s lead product candidate, paltusotine (formerly CRN00808), is an investigational, oral, selective nonpeptide somatostatin receptor type 2 biased agonist for the treatment of acromegaly, an orphan disease affecting more than 26,000 people in the United States. A Phase 3 program in acromegaly with paltusotine is underway. Crinetics also plans to advance paltusotine into a Phase 2 trial for the treatment of carcinoid syndrome associated with neuroendocrine tumors. The company is also developing CRN04777, an investigational, oral, nonpeptide somatostatin receptor type 5 (SST5) agonist for congenital hyperinsulinism, as well as CRN04894, an investigational, oral, nonpeptide ACTH antagonist for the treatment of Cushing`s disease, congenital adrenal hyperplasia and other diseases of excess ACTH. All of the company`s drug candidates are new chemical entities resulting from in-house drug discovery efforts and are wholly owned by the company.
Intellia Therapeutics is a leading genome editing company whose mission is to develop potentially curative gene editing treatments that can positively transform the lives of people living with severe and life-threatening diseases. We are focused on the development of proprietary therapeutics using a recently developed biological tool known as the CRISPR/Cas9 system. The promise of the CRISPR/Cas9 system is the driving force behind the creation of Intellia. Our founders have a shared belief that the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course. This technology offers the potential for us to develop curative therapeutic options for patients with chronic diseases by addressing the underlying cause of the disease. It can bring new hope and cures to people who now have nowhere to turn for help. Our combination of deep scientific expertise and clinical development experience, along with our leading intellectual property portfolio, puts Intellia in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Intellia has the right people, assets and ambitious vision needed to take full advantage of the CRISPR/Cas9 technology`s attributes – high potency, specificity, simplicity of use, broad applicability, and multifunctional programmability – and accelerate the advancement of curative products into the clinic.
Freeline is a clinical-stage, fully integrated, next generation, systemic AAV-based gene therapy company with the ambition of transforming the lives of patients suffering from inherited systemic debilitating diseases. We are headquartered in the UK and we also have operations in Germany and the US. Freeline was founded in 2015 and builds upon the pioneering work by our Clinical and Scientific Advisor and Director, Professor Amit Nathwani, Professor of Haematology at UCL. With our liver-based investigational gene therapy technology, we aim to improve the lives of people with chronic systemic diseases and realise the potential of gene therapy treatments.
Fastgen Corporation is a San Mateo, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
TriNetX is the global health research network enabling healthcare organizations, biopharmaceutical companies and contract research organizations (CROs) to collaborate, enhance trial design, accelerate recruitment and bring new therapies to market faster. TriNetX combines EMR data such as demographics, diagnoses, procedures, medications, labs, genomics, and deep oncology data with data derived from clinical documentation including discharge summaries, radiology reports, pathology reports, and others, to deliver the industry`s most comprehensive data set for protocol design, feasibility, site selection and patient identification.