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

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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

Nanostics – micro-flow cytometry to quantify extracellular vesicle size, concentration and marker abundance, with advanced machine learning algorithms to determine disease states

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Nanostics is focused on development and commercialization of novel, non-invasive diagnostic tests for cancer. Its core technology is an advanced liquid biopsy platform that can accurately diagnose cancer from a single drop of blood. Its lead product, ClarityDx Prostate, is the most accurate diagnostic test to diagnose aggressive prostate cancer, and is positioned to emerge as the world’s leading diagnostic tool for prostate cancer.

Nanostics - micro-flow cytometry to quantify extracellular vesicle size, concentration and marker abundance, with advanced machine learning algorithms to determine disease states

EVs carrying disease-specific biomarkers like nucleic acid and proteins are continuously released from cells and can be found in biological fluids including blood, urine, semen, and cerebrospinal fluid. The levels of disease-specific EVs are closely related to disease progression making EVs promising targets for minimally invasive diagnostic assays.

Technology designed to accurately detect and measure EVs has the potential to greatly improve liquid biopsy diagnostics. At Nanostics, we use micro-flow cytometry (µFCM) to quantify EV size, concentration, and marker abundance for millions of EVs in minutes. We then use advanced machine learning to analyze the vast amount of data generated using µFCM to provide rapid and precise results that continuously improve with every test.

The EV and machine learning platform ClarityDX® is set to transform the diagnostic landscape and make easy-to-use, minimally invasive predictive tests a reality in the near future.

At Nanostics, we use micro-flow cytometry (µFCM) to quantify EV size, concentration, and marker abundance for millions of EVs in minutes. We then use advanced machine learning to analyze the vast amount of data generated using µFCM to provide rapid and precise results that continuously improve with every test.

The EV and machine learning platform ClarityDX® is set to transform the diagnostic landscape and make easy-to-use, minimally invasive predictive tests a reality in the near future.

The ability to detect and measure EVs is transforming the diagnostic landscape in immunology, neurology, cardiology, and oncology. The ClarityDX® platform technology is well-positioned for future pipeline products including tests for screening, early diagnosis, complementary diagnosis, monitoring, and management for many diseases and medically-related events. Nanostics’ R&D team continues to expand the product pipeline through in-house and partnered projects to include additional liquid biopsy diagnostic tests to improve patient care.

Nanostics – micro-flow cytometry to quantify extracellular vesicle size, concentration and marker abundance, with advanced machine learning algorithms to determine disease states

More info : http://www.nanosticsdx.com

Keywords : machine learning, exosomes, extracellular vesicles, diagnostics, oncology, cancer, prostate cancer, cardiovascular, neuroscience, biofluids , liquid biopsy , micro-flow cytometry

Alpha Cognition – developing novel treatments for neurodegenerative diseases such as Alzheimer’s Dementia and Amyotrophic Lateral Sclerosis

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Alpha Cognition is a clinical stage, biopharmaceutical company dedicated to developing novel treatments for under-served neurodegenerative diseases such as Alzheimer’s Dementia and Amyotrophic Lateral Sclerosis (ALS).

Alpha Cognition - developing novel treatments for neurodegenerative diseases such as Alzheimer’s Dementia and Amyotrophic Lateral Sclerosis

ALPHA-1062, a patented new chemical entity that has demonstrated safety and improved ALPHA-1062, is a patented new chemical entity being developed as a new generation acetylcholinesterase inhibitor for the treatment of Alzheimer’s disease, with expected minimal gastrointestinal side effects. ALPHA-1062’s active metabolite is differentiated from donepezil and rivastigmine in that it binds neuronal nicotinic receptors, most notably the alpha-7 subtype, which is known to have a positive effect on cognition. ALPHA-1062 is also being developed in combination with memantine to treat moderate to severe Alzheimer’s dementia and as an intranasal formulation for traumatic brain injury.

ALPHA-0602 (Progranulin) is expressed in several cell types in the central nervous system and in peripheral tissues, promotes cell survival, regulates certain inflammatory processes, and plays a significant role in regulating lysosomal function and microglial responses to disease. Its intended use for the treatment of neurodegenerative diseases has been patented by the Company and Alpha-0602 has been granted an Orphan Drug Designation for the treatment of Amyotrophic Lateral Sclerosis by the FDA.

ALPHA-0702 and ALPHA-0802 are Granulin Epithelin Motifs, or GEMs, derived from full length progranulin which have therapeutic potential across multiple neurodegenerative diseases. Progranulin is comprised of 7½ Granulin Epithelin Motifs (GEMs). GEMs are important in regulating cell growth, survival, repair, and inflammation. Individual granulin domain peptides are approximately 60 amino acids in length (~7kDa). GEMs are metabolically stable and resistant to proteolysis (long half-life). Evidence suggests that not all GEMs are equally potent at promoting cell survival signaling or reducing proteinopathy, and may compete (e.g., binding to effectors) with active GEMs and effectively decrease their potency. Specific GEM combinations have been shown to be more effective than full length progranulin in promoting Cathepsin D maturation. GEMs have been shown to be important in regulating cell growth, survival, repair, and inflammation. Alpha-0702 and ALPHA-0802 are designed to deliver this with potentially lower toxicity, and greater therapeutic effect.

More info : http://www.alphacognition.com/

Keywords : Alpha Cognition , neurodegenerative diseases , dementia , Alzheimer’s Dementia , Alzheimer Disease , Amyotrophic Lateral Sclerosis , acetylcholinesterase inhibitor , orphan drug , CNS , neuroscience , traumatic brain injury , Granulin Epithelin Motifs , Gene Therapy

Accegen Biotech – complete cell & molecular biology products and services

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AcceGen is an US leading biotech company providing complete and authentic cell & molecular biology products and services in a variety of applications for life science researchers worldwide.