Genoscience Pharma – new approaches disrupting cancer cell lysosomal functions

Autophagy is a catabolic process which degrades a cellular own component through the lysosomal machinery. The lysosome, at the heart of the autophagy sytem is important in various processes (cancer, infection…).

Genoscience Pharma - new approaches disrupting cancer cell lysosomal functions

In Cancer, they are required in tumor cells for cellular adhesion, motility and signaling, exocytosis, angiogenesis and overall survival, growth, aggressiveness, metastatic potential and drug resistance. Because of their high metabolic rates, rapidly dividing and invasive cancer cells require increased new biomass production to survive. The lysosome is also important for adaptation to nutrient stress as it contains hydrolytic enzymes that play a major role in the degradation of intracellular macromolecules and catabolic (such as autophagy) and anabolic growth. This busy lysosomal behavior leads to alterations in lysosomal structure and function, which, paradoxically, renders cancer cells more sensitive to lysosomal destabilization. In addition, lysosomal enzyme activity is elevated in many tumors compared to adjacent normal tissue, and several reports suggest that lysosomes in tumor cells are more fragile than normal lysosomes. Therefore, lysosome seems to be a target of interest in the fight against cancers. Targeting lysosomes triggers apoptotic and lysosomal cell death pathways.

Genoscience Pharma – new approaches disrupting cancer cell lysosomal functions

In virology, It has been shown that autophagy is activated during virus and bacterial infection and that some viruses can use the autophagy system to facilitate their own replication . Some viruses, such as Coronaviruses are single stranded, positive sense RNA viruses, which induce the rearrangement of cellular membranes upon infection of a host cell. This provides the virus with a platform for the assembly of viral replication complexes, improving efficiency of RNA synthesis. Genoscience Pharma focuses on new approaches disrupting cancer cell lysosomal functions.

Genoscience Pharma was founded in 2001 by Pr Philippe Halfon, a world renowned medical expert on viral diseases, especially on Human Immunodeficient Virus (HIV) and Hepatitis C Virus (HCV). The company was initially focused on the development of anti-HCV agents. Two protease inhibitors were thus developed and out-licensed to BioLineRX. A new scientific direction was taken in 2012 after the discovery of a new chemical family: autophagy inhibitors. Following promising preliminary in vitro and in vivo results from these small molecules, including against cancer stem cells, Genoscience Pharma has decided to take the opportunity to make the difference in Oncology, especially in cancers where medical needs are still unmet. Now, Genoscience Pharma is a clinical stage biopharmaceutical company focused on translating novel scientific insights into medicines for patients with cancer.

Genoscience Pharma – new approaches disrupting cancer cell lysosomal functions

More info: https://www.genosciencepharma.com/

Keywords : lysosomal functions , cancer, oncology , Genoscience Pharma , lysosomes , infectious diseases  , virus , Covid19, leukemia , pancreatic cancer , hepatocarcinoma , small molecules

ARMGO Pharma – developing small molecule Ryanodine Receptor modulators for treating cardiac, musculoskeletal and neurological disorders

ARMGO Pharma is a privately held biopharmaceutical company dedicated to applying original, targeted science to the discovery and development of novel small-molecule therapeutics to treat debilitating cardiac, musculoskeletal, and neurological disorders. The company’s proprietary drugs, known as Rycals®, are a new class of oral agents that repair calcium leaks through the Ryanodine Receptor (RyR).

ARMGO Pharma - developing small molecule Ryanodine Receptor modulators for treating cardiac, musculoskeletal and neurological disorders

ARM210 is a potential disease modifying therapy for genetic diseases, which targets the Ryanodine Receptor calcium channel (RyR), an intracellular calcium-release channel that becomes leaky in these and other diseases. Intracellular calcium leaks via mutant RyR1 channels impair muscle contraction leading to muscle weakness and loss of function, and activate toxic pathways that damage muscle, causing the symptoms in RYR1-RM.

ARM210 is a small molecule that binds to leaky RyR channels and repairs the leak, as demonstrated in vitro in muscle biopsies from RYR1-RM patients. The unique mechanism of action of ARM210 makes it an ideal potential disease modifying therapy for RYR1-RM. Muscle biopsies of the patients in this trial have been previously shown to respond biochemically to ARM210 in vitro. This trial will evaluate the safety and explore the biochemical effect of oral administration of ARM210 in these same patients.

ARMGO Pharma has been awarded an exclusive, worldwide license from Columbia University for its RyR technology based on the research of founding scientist Andrew R. Marks, M.D. In December 2021, ARMGO Pharma raises a $35 million Series B  funding round to progress clinical studies of lead molecule ARM210 in cardiac and skeletal muscle diseases.

ARMGO Pharma – developing small molecule Ryanodine Receptor modulators for treating cardiac, musculoskeletal and neurological disorders

More info : www.armgo.com

Keywords : ARMGO Pharma , small molecule , therapeutics , RyR technology , ARM210 , cardiovascular , musculoskeletal disorders , neurological disorders , CNS , neuroscience , Rycals , Ryanodine Receptor , RyR , Ryanodine Receptor modulators

Biophytis – therapeutics to slow down the degenerative processes associated with aging

Biophytis is a clinical-stage biotechnology company specialized in the development of therapeutics that are aimed at slowing the degenerative processes associated with aging and improving functional outcomes for patients suffering from age-related diseases, including severe respiratoy failure in patients suffering from COVID-19.

Biophytis - therapeutics to slow down the degenerative processes associated with aging

Biophytis SA, founded in 2006, develops drug candidates targeting diseases of aging. Using its technology and know-how, Biophytis has begun clinical development of innovative therapeutics to restore the muscular and visual functions in diseases with significant unmet medical needs. Specifically, the company is advancing two proprietary drug candidates into mid-stage clinical testing this year: Sarconeos (BIO101) to treat sarcopenic obesity and Macuneos (BIO201) to treat dry age-related macular degeneration (AMD). The business model of BIOPHYTIS is to ensure the conduct of the project until clinical activity in the patient is proven, then to license the technologies in order to continue the development in partnership with a pharmaceutical laboratory. The company was founded in partnership with researchers at the UPMC (Pierre and Marie Curie University) and collaborates with scientists at the Institute of Myology, and the Vision Institute

Its lead drug candidate, Sarconeos (BIO101), is an orally administered small molecule in development for the treatment of neuromuscular diseases, including sarcopenia and Duchenne muscular dystrophy (DMD). Our second drug candidate, Macuneos (BIO201), is an orally administered small molecule in development for the treatment of retinal diseases, including dry AMD and Stargardt disease.

Biophytis also has preclinical efficacy data for Sarconeos (BIO101) in Spinal Muscular Atrophy (SMA), a rare neuromuscular disease. Spinal Muscular Atrophy is a rare progressive neurodegenerative disease that robs an individual of the ability to walk, eat and breathe. SMA is the leading genetic cause of death in infants. Symptoms may appear in the first 6 months of life (type 1, the most severe and common), in infancy (types 2 and 3) or in adulthood (type 4, the least common form). SMA affects 1 in 11,000 births in the United States each year, and about 1 in 50 Americans is a genetic carrier.

More info : www.biophytis.com

Keywords for Biophytis :

neuromuscular diseases, dry age-related macular degeneration, AMD, ophthalmology, infectious diseases, COVID19, Asthma, COPD, small molecule, sarcopenia, Stargardt disease, Duchenne muscular dystrophy , DMD, preclinical stage pharma company, clinical stage pharma company , Spinal Muscular Atrophy , rare diseases , aging