LGMD

Vita Therapeutics Closes $31 Million Series B Financing to Develop Cell Therapies for Neuromuscular Diseases and Cancers

Retrieved on: 
Wednesday, October 12, 2022
Stem Cells, Biotechnology, Other Health, Health, Pharmaceutical, Oncology, Other Science, Research, Genetics, Science, Clinical Trials, Quality of life, LinkedIn, Muscle, Chip, SOLVE, Disease, Therapy, MS, IND, Cell engineering, Autosome, US Foods, Muscle weakness, Patient, Facioscapulohumeral muscular dystrophy, Gene editing, Biotechnology, LGMD2A, Twitter, Wasting, HIV disease progression rates, Walking, Research, Vita, Johns Hopkins University, CAR, Technology, Food, Muscular dystrophy, Lululemon Athletica, Company, Disorder, Health, Facial muscles, LGMD, Weakness, IPSC, Doctor of Philosophy, Adult, Immunotherapy, Genetics, Atrophy, Ageing, Ascending limb of loop of Henle, Cancer, Limb–girdle muscular dystrophy, Pharmaceutical industry, Dietary supplement, Vaccine, FSHD

Vita Therapeutics , a cell engineering company harnessing the power of genetics to develop novel cellular therapies to treat muscular dystrophies and cancers, today announced the completion of a $31 million Series B financing.

Key Points: 
  • Vita Therapeutics , a cell engineering company harnessing the power of genetics to develop novel cellular therapies to treat muscular dystrophies and cancers, today announced the completion of a $31 million Series B financing.
  • Vita Therapeutics current pipeline includes lead program, VTA-100 for the treatment of LGMD2A, VTA-120 for the treatment of FSHD, and VTA-300 targeting multiple cancers.
  • Vita Therapeutics is a biotechnology company developing state-of-the-art cellular therapeutics for the treatment of debilitating neuromuscular diseases and cancers.
  • Vita Therapeutics uses induced pluripotent stem cell (iPSC) technology to engineer specific cell types designed to replace those that are defective in patients.

Atamyo Therapeutics Announces First Patient Dosed with ATA-100 Gene Therapy in LGMD-R9 Clinical Trial

Retrieved on: 
Monday, September 26, 2022
Science, Biotechnology, Research, Pharmaceutical, General Health, Health, Genetics, Clinical Trials, Safety, Trial of the century, FR, EudraCT, UMR, AAV, Food, European Medicines Agency, Weakness, Hope, Muscular dystrophy, National Hospital Organization Nagara Medical Center, Lists of diseases, Life, Therapy, Clinical trial, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Patient, FKRP, LGMD, Degenerative disease, Rigshospitalet, Research, Motivation, Pharmaceutical industry, Vaccine

John Vissing, Director of the Copenhagen Neuromuscular Center at the National Hospital, Rigshospitalet, in Copenhagen, where the first patient was dosed, and principal investigator of this trial.

Key Points: 
  • John Vissing, Director of the Copenhagen Neuromuscular Center at the National Hospital, Rigshospitalet, in Copenhagen, where the first patient was dosed, and principal investigator of this trial.
  • Atamyo Therapeutics is a clinical-stage biopharma focused on the development of a new generation of effective and safe gene therapies for neuromuscular diseases.
  • A spin-off of gene therapy pioneer Genethon, Atamyo leverages unique expertise in AAV-based gene therapy and muscular dystrophies from the Progressive Muscular Dystrophies Laboratory at Genethon.
  • Atamyo conveys the spirit of its commitment to improve the life of patients affected by neuromuscular diseases with life-long efficient treatments.

Registration Now Open for 2023 MDA Clinical & Scientific Conference, to Showcase Cutting Edge Research Advancements and Clinical Achievements in Neuromuscular Disease

Retrieved on: 
Monday, September 12, 2022
YouTube, Limb–girdle muscular dystrophy, SMA, Myotonic dystrophy, Glycogen storage disease type II, NMAC, Congenital muscular dystrophy, COVID-19, Muscular Dystrophy Association, Twitter, MDA, FDA, CRISPR, MG, Knowledge, NEW, Organization, LinkedIn, Hilton Anatole, Public policy, Spinal muscular atrophy, Neuromuscular disease, Myasthenia gravis, History, Disease, CEO, Technology, 2021 Essex County Council election, CMT, CMD, LGMD, Doctor of Philosophy, Charcot–Marie–Tooth disease, Episcopal conference, Amyotrophic lateral sclerosis, Degenerative disease, Muscular dystrophy, TikTok, ALS, Research, Lists of diseases, Conference, DMD, Medicine, Multimedia, DM, Duchenne muscular dystrophy, Pharmaceutical industry, Dentistry, Public relations, Facebook, Instagram

NEW YORK, Sept. 12, 2022 /PRNewswire/ -- The Muscular Dystrophy Association (MDA) today announced that registration is open for the 2023 MDA Clinical & Scientific Conference being held March 19-22, 2023. MDA is the largest worldwide convener of the neuromuscular disease community of renowned researchers, clinicians, academicians, advocates, and industry leaders both in person at the Hilton Anatole in Dallas, Texas and via live stream.

Key Points: 
  • What is equally important is that we convene MDA's Care Center Network of neuromuscular disease specialists, connecting them to the latest clinical trials and FDA approved treatments.
  • They are also developing new treatments and creating the pipeline of progress that is the hallmark of today's neuromuscular disease frontier."
  • Before the conference, MDA will once again host the annual gathering of the Neuromuscular Advocacy Collaborative (NMAC).
  • For over 70 years, MDA has led the way in accelerating research, advancing care, and advocating for the support of our families.

Selecta Biosciences Reports Second Quarter 2022 Financial Results and Provides Business Update

Retrieved on: 
Thursday, August 4, 2022
Cell, Private Securities Litigation Reform Act, Annual report, Partnership, MMA, Gout, Degenerative disease, IND, Achievement, Immune tolerance, Immunoglobulin A, Intellectual property, Investor, DMD, Anticipation, Poster, II, Development, Safety, Autoimmunity, Disease, Pandemic, LGMD, Intention, GLOBE, Income, Preclinical development, PBC, Antibody, Primary, AAV, Completeness, Glycogen storage disease, Patient, Review, U.S. Securities and Exchange Commission, Primary biliary cholangitis, Autoimmune disease, Administration, Telephone, Clinical trial, Mouse, Nanoparticle, Company, Duchenne muscular dystrophy, Â, Society, Biotechnology, CSO, Research, Muscular dystrophy, NASDAQ, Immunoglobulin G, Pharmaceutical industry, Dentistry, Vaccine, Health insurance, Life insurance, Gene, Swedish Orphan Biovitrum, IgA nephropathy, Selecta

Building on this momentum, we enter the second half of 2022 with a focused portfolio of proprietary programs and an expected financial runway into mid-2024.

Key Points: 
  • Building on this momentum, we enter the second half of 2022 with a focused portfolio of proprietary programs and an expected financial runway into mid-2024.
  • Selecta is working with its partner, Cyrus Biotechnology, to develop a next generation IL-2 molecule to combine with ImmTOR and anticipates selecting an IL-2 candidate by year end 2022.
  • Selecta completed enrollment for DISSOLVE II in June 2022, with 153 study participants, triggering a $10 million milestone payment obligation from Sobi which has been received in Q3 2022.
  • Selecta management will host a conference call at 8:30 AM ET today to provide a corporate update and review the companys second quarter 2022 financial results.

Limb-Girdle Muscular Dystrophy Coalition to Host Externally-Led Drug Development Meeting

Retrieved on: 
Tuesday, July 5, 2022
FDA, Caregiver, ML, Sarepta Therapeutics, Muscle weakness, Muscular dystrophy, LGMD, Prescription Drug User Fee Act, Human voice, Industry, Association, Prevalence, Awareness, Food, Wasting, Degenerative disease, Muscular Dystrophy Association, Organization, PDUFA, The Cyprus Foundation for Muscular Dystrophy Research, Research, Muscle, Patient, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Medical device, Pharmaceutical industry, Coalition

FALLS CHURCH, Va., July 5, 2022 /PRNewswire/ -- On September 23, 2022 at 10am EDT, a coalition of limb-girdle muscular dystrophy (LGMD) nonprofit organizations will lead an Externally-Led Patient Focused Drug Development Meeting (EL-PFDD).

Key Points: 
  • FALLS CHURCH, Va., July 5, 2022 /PRNewswire/ -- On September 23, 2022 at 10am EDT, a coalition of limb-girdle muscular dystrophy (LGMD) nonprofit organizations will lead an Externally-Led Patient Focused Drug Development Meeting (EL-PFDD).
  • The meeting will be summarized in a Voice of the Patient report which, along with recorded footage of the meeting, will be publicly available.
  • The LGMD Coalition is grateful for support from our sponsors, AskBio, Edgewise Therapeutics, ML BioSolutions, Sarepta Therapeutics, and Vita Therapeutics.
  • We also acknowledge our Advocacy Partners, the Muscular Dystrophy Association and the LGMD Awareness Foundation.

The LGMD Community Celebrates Adoption of ICD-10 Diagnostic Codes for LGMD

Retrieved on: 
Wednesday, June 29, 2022
Department, Diagnosis, Department of Neurology, Xuanwu hospital, View, Patient, Neurology, TikTok, LinkedIn, Public policy, Research, Adoption, Muscular dystrophy, ALS, Prevalence, Awareness, Muscular Dystrophy Association, Degenerative disease, Organization, FAAN, Incidence, NEW, MDA, Twitter, Physician, LGMD, Medical device, Pharmaceutical industry, MSCI, Facebook, Instagram, MD

The implementation of these ICD-10 codes represents a culmination of two-years of collaborative efforts within the LGMD patient, clinical, and research communities.

Key Points: 
  • The implementation of these ICD-10 codes represents a culmination of two-years of collaborative efforts within the LGMD patient, clinical, and research communities.
  • These diagnostic codes will be fully implemented and ready for use starting this October.
  • This significant advance could well shorten the diagnostic odyssey for individuals with LGMD, facilitate the targeted care the community needs, and accelerate surveillance and epidemiological research.
  • This milestone is the culmination of over two years of collaborative efforts between LGMD advocacy organizations and leading LGMD clinicians and researchers.

Selecta Biosciences Announces Partnership Advancements and Clinical Trial Updates

Retrieved on: 
Monday, June 13, 2022
Kidney, Degenerative disease, Achievement, Duchenne muscular dystrophy, Company, Intellectual property, Gout, Sarepta Therapeutics, Java Research License, Elevated railway, Annual report, United, Muscular dystrophy, II, Partnership, Security (finance), Methylmalonic acidemia, U.S. Securities and Exchange Commission, ADAS, AAV, Metabolism, MMA, DMD, Randomness, Wastewater treatment, SRPT, Sarepta, GLOBE, Inflammatory arthritis, Private Securities Litigation Reform Act, LGMD, Pandemic, Incidence, Quality of life, NASDAQ, Autoimmune disease, Clinical trial, Flare, Heart, SUA, Tissue, Uric acid, Joint, Safety, Vaccine, Pharmaceutical industry, Selecta, Swedish Orphan Biovitrum, Greater Boston, Patient

WATERTOWN, Mass., June 13, 2022 (GLOBE NEWSWIRE) -- Selecta Biosciences, Inc. (NASDAQ: SELB), a biotechnology company leveraging its clinically validated ImmTOR® platform to develop tolerogenic therapies for autoimmune diseases, enhance gene therapies and mitigate unwanted immune responses to biologics, today announced several pipeline advancements and partnership updates.

Key Points: 
  • Additionally, Selecta is eligible to receive an additional $4 million payment upon the achievement of certain near-term preclinical milestones by Sarepta.
  • DISSOLVE II, the second of two Phase 3 clinical studies evaluating SEL-212 for chronic refractory gout, continues to progress.
  • The completion of enrollment in DISSOLVE I & II will result in a $10 million milestone payment obligation from Sobi to Selecta.
  • Selecta expects to start a Phase 1 clinical trial in Q4 2022.

Muscular Dystrophy Association Announces MDA Rivals, A Streaming Event Featuring Special Guests Including Indianapolis Colts Running Back Nyheim Hines and More

Retrieved on: 
Wednesday, June 8, 2022
Neuromuscular disease, Research, Muscular Dystrophy Association, LinkedIn, Indianapolis Colts, NFL, Degenerative disease, Fall Guys, Play, MDA, Awareness, PSAS, Multimedia, Muscular dystrophy, LGMD, ALS, Duchenne muscular dystrophy, NEW, Twitter, Rocket League, TikTok, View, Public relations, Online gambling, Video game console, Facebook, Instagram

NEW YORK, June 8, 2022 /PRNewswire/ -- What/Where/When: The Muscular Dystrophy Association (MDA) will host "MDA Rivals," a one-day streaming event featuring the stars of the MDA's Let's Play gaming community and professional streamers, including Indianapolis Colts running back and the MDA 2022 National Spokesperson Nyheim Hines, on Saturday, June 18, 2022 from 7-8pm ET on Twitch.

Key Points: 
  • Participants will earn prizes and have the opportunity to win sponsored product giveaways including signed jerseys and footballs from Hines.
  • Nyheim Hines , Indianapolis Colts Running Back, 2022 MDA National Spokesperson
    MDA partners with Hines, who has a personal connection to the critical need to drive awareness, improve care, advance research, and advocate for people living with muscular dystrophy, ALS, and related neuromuscular diseases.
  • Hines' mother lives with limb-girdle muscular dystrophy (LGMD) and shares his story in the video and audio PSAs with MDA National Ambassador Ethan LyBrand, who lives with Duchenne muscular dystrophy.
  • Muscular Dystrophy Association (MDA) is the #1 voluntary health organization in the United States for people living with muscular dystrophy, ALS, and related neuromuscular diseases.

Atamyo Therapeutics Announces Significant Milestones for ATA-100 and ATA-200, its Gene Therapy Programs to Treat Limb-Girdle Muscular Dystrophy 2I/R9 and 2C/R5

Retrieved on: 
Monday, May 16, 2022
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, Muscular dystrophy, ANSM, Health care, EST, EMA, FKRP, Master of Science, European Society of Gene and Cell Therapy, Therapy, Research, National Medicinal Drugs Policy, CMC, Lists of diseases, Orphan, Quality, Supply chain, Vaccine, Patient, Degenerative disease, CTA, European Medicines Agency, Cell, MBA, Sanofi Pasteur, CNRS, LGMD, Poster, MHRA, Date, Technology, CTO, Orphan drug, Hope, Gene, Industry, AAV, Life, Weakness, Master of Engineering, CEO, Pharmaceutical industry, Management

ATA-100, a single-administration gene therapy candidate for LGMD2I/R9, delivers a normal copy of the gene for production of FKRP proteins.

Key Points: 
  • ATA-100, a single-administration gene therapy candidate for LGMD2I/R9, delivers a normal copy of the gene for production of FKRP proteins.
  • Atamyo Therapeutics is focused on the development of a new generation of effective and safe gene therapies for neuromuscular diseases.
  • A spin-off of gene therapy pioneer Genethon, Atamyo leverages unique expertise in AAV-based gene therapy and muscular dystrophies from the Progressive Muscular Dystrophies Laboratory at Genethon.
  • Atamyo conveys the spirit of its commitment to improve the life of patients affected by neuromuscular diseases with life-long efficient treatments.

Vita Therapeutics Relocates to University of Maryland BioPark

Retrieved on: 
Monday, May 2, 2022
Catalent, Muscular dystrophy, University, Senior, University of Maryland, College Park, Johns Hopkins University, LGMD, ABQ BioPark Botanic Garden, Company, Biotechnology, MS, Cell engineering, Food, IPSC, Patient, US Foods, Health, BD, History of the University of Maryland, College Park, UM, Genetics, Technology, Economic development, Series, Cancer, Therapy, Pharmaceutical industry, Illumina

BALTIMORE, May 2, 2022 /PRNewswire/ -- The University of Maryland (UM) BioPark announced today that Vita Therapeutics, a cell engineering company harnessing the power of genetics to develop cellular therapies to treat muscular dystrophies and solid tumors, has established offices and laboratory space within approximately 10,000 square feet of leased space in the BioPark. Vita Therapeutics will leverage the new space to bring together its 25+-person team under one roof for the first time with the goal of progressing its co-lead programs, VTA-100 and VTA-300. The company uses induced pluripotent stem cell (iPSC) technology to engineer specific cell types designed to replace those that are defective in patients.

Key Points: 
  • "We proudly welcome Vita Therapeutics as the newest tenant within the BioPark," said James Hughes, President of the UM BioPark and Senior Vice President and Chief Enterprise and Economic Development Officerat University of Maryland, Baltimore.
  • Vita Therapeutics has completed Series A financing of $32 million and is currently working with numerous partners, including PanCella, Wyss Institute, and Johns Hopkins University, to advance clinical programs.
  • Vita Therapeutics is a biotechnology company generating state-of-the-art cellular therapeutics for the treatment of debilitating neuromuscular diseases and cancers.
  • The UM BioPark offers high-quality lab and office space solutions for companies of all sizes, especially high-growth companies seeking connection with its academic partner, University of Maryland, Baltimore.