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

Cell-Easy – reducing stem cells manufacturing cost for cell therapies

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Cell-Easy is a cell therapy Contract Development and Manufacturing Organization (CDMO) for preclinical and clinical drug development projects.

Cell-Easy’s aspiration is to dramatically reduce stem cell manufacturing cost, starting with ASC (historical and local strong expertise), to change the affordability challenge currently met by Cell Therapies. Its ASC production process was designed using QbD approach and economical study (Manufacturing Cost model simulation) to ensure cell quality, scalability, compatibility with GMP requirements and economic viability for the entire clinical development INCLUDING the commercialization.

European leading CDMO company specialized in large volume industrialized production of GMP approved cell products

Cell-Easy’s team is committed to its customers to develop & produce ATMPs for clinical advanced therapies. Its ambition combines analytical and process innovations to provide cGMP cells with extended characterization (safety, identity, and performance). Cell-Easy’s experience in stem cells has grown from its original ambition: to make cell therapy accessible to as many patients as possible, by combining an allogeneic approach with a high-performance culture process. Its MSC production process has been designed using a QbD approach and an economic study (simulation of the manufacturing cost model) to ensure cell quality, scalability, compatibility with GMP requirements and economic viability for the entire clinical development, including commercialization.

Analytics-driven Contract Development and Manufacturing Organization (CDMO) specialized in Cell Therapy, Cell-Easy offers customized process development, GMP manufacturing and unique analytical services for adult stem cells (MSCs, iPSCs and Exosome) and immune cells (engineered CAR-T,-NK…cells, and non-engineered cells) in Regenerative Medicine and Immuno-oncology fields.

Cell-Easy facilities comprise 350 sqm of qualified controlled environment suites optimized to develop Manufacturing and Analytical Process, proceed to environmental and material Quality Control and execute cGMP Manufacturing, Fill&Finish, long-term Storage of ATMPs compliant with all the requirements of Regulatory Standards and cGMP guidelines.

Cell-Easy is a recent and fast-growing biotech startup based in Toulouse, France. The Cell-Easy Management team and all collaborators have a strong expertise in the Development and Manufacturing of innovative cell-based therapies.

More info : https://www.cell-easy.com/

Glycorex Transplantation – removing antibodies from blood to improve transplantation and fight autoimmune diseases

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Glycorex’s unique molecular level technology that specifically selects and removes antibodies in the blood has opened up ground-breaking treatments and solutions in healthcare. The company’s medical devices enable transplants across the blood groups saving lives and providing health economic benefits by increasing the number of transplants. The company’s technology to treat blood is applied in several health care settings and a new disruptive product has recently been launched with the potential to increase availability of blood plasma. Several new products for the treatment of autoimmune diseases are under development.

Glycorex Transplantation - removing antibodies from blood to improve transplantation and fight autoimmune diseases

In addition to kidney transplants, our product, Glycosorb® ABO, is used in several other types of transplants, such as liver, heart and stem cell transplants. More than 60 scientific papers with Glycosorb® ABO show excellent short- and long-term results fully comparable to blood group compatible transplants. Thanks to Glycosorb® ABO, blood group incompatible transplants are now routinely performed in many countries.

With our unique technology as a base, we are currently expanding into new interesting applications. In the area of transfusion medicine, we have recently launched a product intended to be used for donated plasma to produce universal blood plasma which can be given regardless of the recipient’s blood group, which means major improvements in logistics, safety and availability.
The focus of our development work is now to develop new treatments for severe autoimmune diseases. The closest to launch is a specific treatment for rheumatoid arthritis (RA) – a disease that has affected around five million people in Europe.

Glycorex Transplantation AB (publ) is based in Lund, Sweden, and its shares are listed on NGM – Main Regulated, Stockholm (NGM: GTAB B).

Glycorex Transplantation – removing antibodies from blood to improve transplantation and fight autoimmune diseases

More info : Glycorex Transplantation AB

Keywords : transplantation, autoimmune diseases, Rheumatoid Arthritis, antibodies hematology

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

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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

Sycai Medical – Early stage diagnosis of pancreatic cancer through Medical Imaging and Artificial Intelligence

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Sycai Medical is a technological startup founded in Barcelona in 2020, born from the motivation of empowering radiologists to improve healthcare through artificial vision and big data. They develop AI-based algorithms, which helps medical professionals in the diagnosisof abdominal injuries and in the prediction of their evolution to increase early-stage cancer detection and improving the quality of life of patients with chronic diseases.

Their product Sycai Medical is an assistant, which helps radiologists to detect pancreatic cystic lesions, predicting their malignant potential and presenting similar cases to verify the diagnosis.

This kind of lesions can evolve into pancreatic cancer, the 4th cause of death by cancer in Europe with an average survival time from diagnosis of less than 5 months and currently without an efficient method or early diagnosis.

The follow-up treatment of the pancreatic cysts leads to repetitive image tests for life, due to the difficulty of predicting which ones are going to be malignant and which are staying benign. The ability of predicting their evolution is the key to increase early detection of pancreatic cancer and to optimize the follow-up treatments.

Their Artificial Intelligence and Big Data based software assists radiologists in the detection and classification of abdominal lesions on CT scans and MRIs. It also provides a prediction of the probability of each lesion to become cancer in the future, which is crucial to diagnose early-stage pancreatic cancer and to improve the life quality of patients.

More info : www.sycaimedical.com

Artificial Intelligence – Big Data – Software – Digital Health – Medical Imaging – Oncology – Pancreatic Cancer – Abdomen – Radiology – Diagnostics – Detection