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GenSight Biologics is is a clinical-stage biotechnology company discovering and developing novel therapies for neurodegenerative retinal diseases and diseases of the central nervous system. GenSight Biologics` pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics for retinitis pigmentosa, to help preserve or restore vision in patients suffering from severe degenerative retinal diseases. GenSight Biologics` lead product candidate, GS010, is in Phase III trials in Leber`s Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible low vision and legal blindness in teens and young adults. Using its gene therapy-based approach, GenSight Biologics` product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery.
EpicentRx is a patient-driven immuno-oncology company with minimally toxic therapies that work across diverse patient populations and tumor types for the best possible quality of life during treatment.
Loxo Oncology is committed to the discovery, development, and commercialization of targeted cancer therapies with best-in-class potential. Our diverse pipeline reflects the convergence of proven therapeutic technologies with emerging insights into the underlying susceptibilities of cancer and drug resistance. It is an exciting time to develop cancer drugs. Diagnostic methods are yielding strong clues about which cancer features are actionable, “driver alterations,” in contrast to those merely along for the ride as “passenger alterations.” In the last five years, these insights have transformed the standard of care in melanoma, lung cancer and other cancers. Picking a target is just the first step in building a cancer drug. Good chemists build good drugs. The physical properties of a compound have much to do with its ultimate success or failure. Is it stable? Is it soluble? Is it absorbed? Does it reach its target? Does it engage the target? Does it cause toxicity elsewhere in the body before it achieves maximum efficacy in the cancer? In our opinion, drugs that achieve excellent exposure in the body and are highly specific for their intended targets have the highest chances of success. Once an attractive target is selected and a drug with best-in-class properties is constructed, setting up the right clinical development plan is of utmost importance. Our goal is to generate a clinical path that reflects the underlying scientific hypothesis that made the drug interesting in the first place. This often means developing the drug in a population of patients whose tumors possess a specific vulnerability targeted by the drug. Loxo Oncology was built around a team of full-time professionals and engaged scientific advisors who are aligned in choosing the most actionable targets, and pursuing them through disciplined clinical trial approaches. Our license and collaboration agreement with Array Biopharma and our experienced team allow us to construct drugs with best-in-class properties from the ground up.
Generate Biomedicines is the first drug generation company, pioneering a machine learning-powered generative biology platform with the ability to generate new drugs on demand across a wide range of biologic modalities. The platform can drastically improve the speed at which targets and therapeutics are identified and validated, the specificity of target engagement by generated proteins, and the cost of identifying and developing clinical candidates. The company`s platform represents a potentially fundamental shift in what`s possible in the field of therapeutic development, addressing key challenges of drug discovery and drastically expanding the available search space for novel biomedicines. Generate Biomedicines was founded by Flagship Pioneering after two years of foundational research in its Labs unit and launched in 2020.
Altimmune is a clinical-stage immunotherapeutics company focused on the development of products to stimulate robust and durable immune responses for the prevention and treatment of infectious disease.