Intramural sports

ARMGO Pharma Publishes Positive Phase 1b Trial Results of Rycal® ARM210 for the Treatment of Ryanodine Receptor 1 Related Myopathies

Retrieved on: 
Monday, January 29, 2024

The paper reviews data from the Phase 1b study of ARM210 and its novel allosteric mechanism of action (MoA) targeting the root cause of RYR1-RM: mutated Ryanodine Receptor 1 (RYR1).

Key Points: 
  • The paper reviews data from the Phase 1b study of ARM210 and its novel allosteric mechanism of action (MoA) targeting the root cause of RYR1-RM: mutated Ryanodine Receptor 1 (RYR1).
  • These results warrant further development of ARM210 as a potential disease modifying treatment for RYR1-RM in a randomized, placebo-controlled Phase 2 trial.
  • We look forward to the next stages in the development of this important drug.”
    Further information about this Phase 1b trial can be found online at: https://clinicaltrials.gov/study/NCT04141670 .
  • Rycal S48168 (ARM210) for RYR1-related myopathies: a phase one, open-label, dose-escalation trial, Todd et al, eClinicalMedicine https://www.thelancet.com/journals/eclinm/article/PIIS2589-5370(24)00012-9/fulltext

ARMGO Pharma Publishes Positive Phase 1b Trial Results of Rycal® ARM210 for the Treatment of Ryanodine Receptor 1 Related Myopathies

Retrieved on: 
Monday, January 29, 2024

The paper reviews data from the Phase 1b study of ARM210 and its novel allosteric mechanism of action (MoA) targeting the root cause of RYR1-RM: mutated Ryanodine Receptor 1 (RYR1).

Key Points: 
  • The paper reviews data from the Phase 1b study of ARM210 and its novel allosteric mechanism of action (MoA) targeting the root cause of RYR1-RM: mutated Ryanodine Receptor 1 (RYR1).
  • These results warrant further development of ARM210 as a potential disease modifying treatment for RYR1-RM in a randomized, placebo-controlled Phase 2 trial.
  • We look forward to the next stages in the development of this important drug.”
    Further information about this Phase 1b trial can be found online at: https://clinicaltrials.gov/study/NCT04141670 .
  • Rycal S48168 (ARM210) for RYR1-related myopathies: a phase one, open-label, dose-escalation trial, Todd et al, eClinicalMedicine https://www.thelancet.com/journals/eclinm/article/PIIS2589-5370(24)00012-9/fulltext

AbelZeta Announces Formation of Scientific Advisory Board to Support Inflammatory and Immunological Diseases

Retrieved on: 
Wednesday, January 3, 2024

These four industry experts are distinguished scientific leaders with vast experience in the autoimmune disease field."

Key Points: 
  • These four industry experts are distinguished scientific leaders with vast experience in the autoimmune disease field."
  • Members of the SAB include:
    Peter Lipsky, M.D., served on the Board of Directors of the American College of Rheumatology.
  • She was President of the American College of Rheumatology, President of the Henry Kunkel Society, and Chair of the Scientific Advisory Board of the Lupus Research Alliance.
  • "AbelZeta is advancing promising new therapies for inflammatory and immunological diseases through innovative cell therapy platforms that provide custom-tailored treatment for each individual patient.

ImmunoScape Appoints Systems Immunology and Computational Biology Expert Dr. John Tsang to its Scientific Advisory Board

Retrieved on: 
Tuesday, December 12, 2023

ImmunoScape , a biotechnology company focused on next-generation immunotherapies, today announced the appointment of John Tsang, Ph.D. to its Scientific Advisory Board (SAB).

Key Points: 
  • ImmunoScape , a biotechnology company focused on next-generation immunotherapies, today announced the appointment of John Tsang, Ph.D. to its Scientific Advisory Board (SAB).
  • Dr. Tsang joins a distinguished group of leading scientists in immunology and oncology who help to guide ImmunoScape’s scientific strategy.
  • Dr. Tsang was also the Co-Director of the Trans-NIH Center for Human Immunology (CHI) and led its research program in systems human immunology.
  • Dr. Tsang has won multiple awards for his research, including several NIH/NIAID Merit Awards recognizing his scientific leadership in systems immunology, COVID-19, and human immunology research.

MAPS PBC Announces National Institute on Drug Abuse to Share Data for New Drug Application for MDMA-Assisted Therapy for Treatment of PTSD

Retrieved on: 
Wednesday, June 21, 2023

With four decades of independent research looking at its safety, MDMA is one of the most well-studied molecules in clinical development today."

Key Points: 
  • With four decades of independent research looking at its safety, MDMA is one of the most well-studied molecules in clinical development today."
  • MAPS PBC expects to submit the new drug application to the FDA in the second half of 2023.
  • The safety and efficacy of MDMA-assisted therapy have not been established for the treatment of PTSD.
  • This content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.

BioAegis Therapeutics Announces Two Studies Exploring the Role of ‘Inflammation Regulator’ Gelsolin in Type 2 Diabetes

Retrieved on: 
Thursday, April 13, 2023

NORTH BRUNSWICK, N.J., April 13, 2023 (GLOBE NEWSWIRE) -- BioAegis Therapeutics, Inc., a clinical-stage company developing therapies for inflammatory diseases through a portfolio built around plasma gelsolin (pGSN), a highly conserved and abundant endogenous human immune regulatory protein, announces publication of two new research studies exploring gelsolin’s role in type 2 diabetes, an inflammation-related metabolic disease.

Key Points: 
  • Low levels of gelsolin identified in individuals with type 2 diabetes living below poverty.
  • It explored the role of microparticles (MPs) which drive inflammation in individuals with diabetes.
  • Type 2 diabetic subjects with foot ulcers were shown to have up to 14-fold elevations of blood-borne F-actin-coated microparticles.
  • White adults with diabetes had lower levels compared to White adults without diabetes and to African American individuals either with or without diabetes.

Induced Pluripotent Stem Cell (iPSC) Global Market Report 2023: Sector to Reach $4.36 Billion by 2027 at a 10.1% CAGR - ResearchAndMarkets.com

Retrieved on: 
Tuesday, February 21, 2023

The "Induced Pluripotent Stem Cell (iPSC) Global Market Report 2023" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Induced Pluripotent Stem Cell (iPSC) Global Market Report 2023" report has been added to ResearchAndMarkets.com's offering.
  • The regions covered in the induced pluripotent stem cell (iPSC) market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East and Africa.
  • An increase in the prevalence of chronic disorders is one of the major factors that is driving the growth of the Induced pluripotent stem cell market.
  • The potential risk of tumour is one of the major restraints on the growth of the Induced pluripotent stem cell market.

New Study Presents Novel Solution to Increasing Diagnostic Yield and Accelerating Diagnosis of Rare Disease

Retrieved on: 
Monday, October 31, 2022

ANN ARBOR, Mich. , Oct. 31, 2022   /PRNewswire-PRWeb/ -- Genomenon, Inc., an AI-driven genomics company, announced the publication of a paper in Human Mutation summarizing current knowledge of clinical and genetic findings in patients with ENPP1 Deficiency. The study, completed in collaboration with Inozyme Pharma, is the largest ever gathered, and demonstrates the necessity of comprehensive genomic data for accelerating the diagnosis of rare disease.

Key Points: 
  • The study, completed in collaboration with Inozyme Pharma, is the largest ever gathered, and demonstrates the necessity of comprehensive genomic data for accelerating the diagnosis of rare disease.
  • The data has been made available to doctors, researchers, and clinicians through Genomenon's Mastermind Genomic Search Engine with the purpose of increasing awareness and diagnosis of the disease and related clinical trials.
  • Inozyme Pharma, Inc. (Nasdaq: INZY) is a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases impacting the vasculature, soft tissue, and skeleton.
  • INZ-701 is currently in Phase 1/2 clinical trials for the treatment of ENPP1 Deficiency and ABCC6 Deficiency.

National Institute of Health Revitalizes Data-Entry Procedures Amid OIG Reprimand

Retrieved on: 
Wednesday, September 14, 2022

Washington, D.C.--(Newsfile Corp. - September 14, 2022) - The National Institute of Health (NIH) has been reprimanded by the US Department of Health and Human Services (HHS) for failing to uphold transparency guideline requirements, according to a recent statement from the Office of the Inspector General (OIG).

Key Points: 
  • Washington, D.C.--(Newsfile Corp. - September 14, 2022) - The National Institute of Health (NIH) has been reprimanded by the US Department of Health and Human Services (HHS) for failing to uphold transparency guideline requirements, according to a recent statement from the Office of the Inspector General (OIG).
  • The HHS and OIG have made recommendations to the NIH to ensure transparency moving forward.
  • Published earlier this month by the OIG, the statement outlined specifics on the reprimand and mistakes the NIH made in inadequately enforcing federal reporting requirements for the various studies that it funds.
  • In response to this report, the NIH has agreed to update procedures to ensure strict implementation of guidelines.