Fabry disease

Latest Updates of Viva Biotech's Portfolio Companies

Retrieved on: 
Mercoledì, Marzo 6, 2024
Fabry disease, RMB, PD, Drug development, Strong, Ohio State University, RNA, Insight Partners, Pharmacology, American College, Radiopharmaceutical, VBI, Willebrand, RTW, Pattern hair loss, GMP, Stroke, Good manufacturing practice, Therapy, Skin, GCS, Atopic dermatitis, Safety, Pharmacokinetics, Von Willebrand factor, Investment, AstraZeneca, Innovation, Groundbreaking, Patient, ARCH Venture Partners, Pharmaceutical industry

NEWARK, Calif.-- AceLink Therapeutics, Inc., invested and incubated by Viva BioInnovator (VBI), is a clinical-stage biopharmaceutical company developing next generation oral substrate reduction therapies (SRTs). Recently they announced that the findings from their Phase 1 study of AL01211 in healthy volunteers have been published in the peer-reviewed journal Clinical Pharmacology in Drug Development, a journal of the American College of Clinical Pharmacy. AL01211 is a potent, oral, glucosylceramide synthase (GCS) inhibitor being developed for the treatment of Fabry disease and Type 1 Gaucher disease.

Key Points: 
  • This continuous innovation keeps companies up to date and promotes the evolution of R&D and the success of commercialization.
  • Recently, Viva Biotech's portfolio companies have new updates.
  • NEWARK, Calif.-- AceLink Therapeutics, Inc., invested and incubated by Viva BioInnovator (VBI), is a clinical-stage biopharmaceutical company developing next generation oral substrate reduction therapies (SRTs).
  • Previously, the company had completed a $5.4 million seed funding round, with investors including Rev1 Ventures, Broadview Ventures, and Viva Biotech.

This Rare Disease Day, Feb. 29, CORD celebrates the launch of the Canadian Rare Disease Network - but patients still have no access to the promised $1.4 billion in federal funds for rare disease drugs

Retrieved on: 
Giovedì, Febbraio 29, 2024
Rare disease, Collection, Fabry disease, Health Canada, Research, Investment, Emergency department, Data collection, Calendar, Federation, Organization, Long-term care, Patient, Optimism, CRDN, Disease, Heart, Diagnosis, Science, Summit, Pharmaceutical industry

The federal government promised a Rare Disease Drug Strategy five years ago and announced $1.4 billion in drug funding last March.

Key Points: 
  • The federal government promised a Rare Disease Drug Strategy five years ago and announced $1.4 billion in drug funding last March.
  • CORD is wildly optimistic with the milestone launch of the Canadian Rare Disease Network (CRDN) – a collaboration that brings into reality the vision and strategy that CORD first proposed in 2015.
  • While celebrating the birth of the CRDN, the rare disease community is marking Rare Disease Day with mounting impatience due to unconscionable delays in making available the committed federal government funding for rare disease medicines.
  • CORD has been holding a Rare Disease Day 2024 Summit in Ottawa February 28 and 29.

GC Biopharma Presents Updates on its LSD Treatments at the WORLDSymposium 2024

Retrieved on: 
Mercoledì, Febbraio 14, 2024
Enzyme replacement therapy, Fabry disease, ICV, EMA, Intelligence, European, Glycolipid, ERT, Sanfilippo syndrome, Hunter syndrome, Death, MPS, Prevalence, ODD, Safety, Sanfilippo, IDS, Disorder, Patient, Hanmi Pharmaceutical, Fibrosis, Clinical trial, Knowledge, Kidney disease, Disease, Urine, Heart, Alpha-galactosidase, Phase, Male, Kidney, Glycogen storage disease, Brain, Orphan, Mouse, Pharmaceutical industry

YONGIN, South Korea, Feb. 14, 2024 /PRNewswire/ -- GC Biopharma (CEO, Eun-Chul Huh), a South Korean biopharmaceutical company, announced on Feb. 14th that it has presented the development updates on its LSD (Lysosomal Storage Diseases) medicines at the WORLDSymposium 2024 held on Feb. 4th-9th, 2024 in San Diego, USA.

Key Points: 
  • YONGIN, South Korea, Feb. 14, 2024 /PRNewswire/ -- GC Biopharma (CEO, Eun-Chul Huh), a South Korean biopharmaceutical company, announced on Feb. 14th that it has presented the development updates on its LSD (Lysosomal Storage Diseases) medicines at the WORLDSymposium 2024 held on Feb. 4th-9th, 2024 in San Diego, USA.
  • WorldSymposium 2024 is an international forum for Lysosomal Diseases experts to share and exchange insights for researching better treatment of the disease.
  • In 2012, GC Biopharma succeeded in developing the world's second treatment for Hunter syndrome, "Hunterase" solely using domestic technology.
  • GC Biopharma, together with Hanmi Pharmaceutical, is developing GC1134A/HM15421, a long-acting alpha-galactosidase that can be administered subcutaneously once a month to improve patient convenience.

Sangamo Therapeutics Announces U.S. FDA Alignment on Abbreviated Pathway to Potential Approval and EMA Prime Eligibility for ST-920 in Fabry Disease

Retrieved on: 
Lunedì, Febbraio 12, 2024
Research, FDA, Neurology, Genetics, Clinical Trials, Biotechnology, Health, Pharmaceutical, Science, Fabry disease, EMA, Orphan drug, Type D, Investment, Female, ERT, BLA, STAAR, Therapy, PRIME, Sangamo Therapeutics, Patient, Male, Enzyme replacement therapy, Partnership, Pharmaceutical industry

The proposed study would enroll up to 25 patients, both male and female, without the need for a control arm.

Key Points: 
  • The proposed study would enroll up to 25 patients, both male and female, without the need for a control arm.
  • A head-to-head comparison with Enzyme Replacement Therapy (ERT) is not part of the proposed study design deemed acceptable by the FDA.
  • This approach enables a potentially more rapid, efficient and cost-effective pathway to BLA submission than originally anticipated.
  • Sangamo is deferring additional investments in planning for a registrational trial until a collaboration partnership is secured.

4DMT Presents Interim Data from 4D-310 INGLAXA Phase 1/2 Clinical Trials for Fabry Disease Cardiomyopathy at WORLDSymposium™ 2024

Retrieved on: 
Lunedì, Febbraio 12, 2024
C102, Atypical hemolytic uremic syndrome, Fabry disease, CPET, VO2, FDA, Quality of life, Cardiovascular disease, Data, GLS, Safety, MCID, Overalls, Death, Patient, Echocardiography, Chelsea Handler, Pharmaceutical industry

The data was presented in a late-breaking session at WORLDSymposium™ 2024 in San Diego, California on Friday, February 9, 2024.

Key Points: 
  • The data was presented in a late-breaking session at WORLDSymposium™ 2024 in San Diego, California on Friday, February 9, 2024.
  • “We are pleased to see 4D-310 continue to consistently demonstrate clinical activity across multiple important cardiac endpoints including cardiac function, exercise capacity and quality of life,” said Robert Kim, M.D., Chief Medical Officer of 4DMT.
  • “Current therapies do not adequately address Fabry-related cardiovascular manifestations, and cardiovascular disease is the most common cause of death in these patients.
  • “No approved therapy has been shown to definitively clear accumulated Gb3 from cardiomyocytes in patients with Fabry disease, which speaks to 4D-310’s highly differentiated profile.

Chiesi Global Rare Diseases Presents Long-Term Data on Treatment with Velmanase Alfa in Alpha-Mannosidosis and Announces Additional Presentations at the 20th Annual WORLDSymposium™ Research Meeting

Retrieved on: 
Giovedì, Febbraio 8, 2024
Conjunctivitis, Arthralgia, Anaphylaxis, Common, ADAS, Birth, Prevalence, Velmanase alfa, Fabry disease, Human, Diagnosis, Headache, Cough, Erythema, ERT, Glycogen storage disease, Alpha-mannosidosis, DSM-IV codes, Vomiting, Hospital, Incidence, Patient, Rash, Nursing, Data, Chills, Enzyme replacement therapy, Pharmaceutical industry

BOSTON, Feb. 8, 2024 /PRNewswire/ -- Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases, today announced the presentation of long-term data from up to 12 years of treatment with velmanase alfa in patients with alpha-mannosidosis (AM). The company also announced the presentation of immunogenicity and tolerability data from its pegunigalsidase alfa clinical trial program in Fabry disease, as well as analyses of the prevalence, diagnosis, and progression of these rare lysosomal storage disorders. The data are being presented during the 20th Annual WORLDSymposium™ Research Meeting that is being held February 4-9, 2024, in San Diego, California.

Key Points: 
  • The data are being presented during the 20th Annual WORLDSymposium™ Research Meeting that is being held February 4-9, 2024, in San Diego, California.
  • Several poster presentations are highlighting immunogenicity and tolerability data from the pegunigalsidase alfa clinical trial program in Fabry disease.
  • The analysis includes 108 patients, 27 (25%) of whom were ADA+ before receiving their first dose of pegunigalsidase alfa.
  • There was a low incidence of de novo ADAs (n=17) occurring predominantly in the first year of treatment with pegunigalsidase alfa.

Amicus Therapeutics Receives the 2024 New Treatment Award for Pombiliti™ (cipaglucosidase alfa-atga) + Opfolda™ (miglustat) at the 20th Annual WORLDSymposium™

Retrieved on: 
Giovedì, Febbraio 8, 2024
Partnership, Amicus Therapeutics, Cipaglucosidase alfa, Glycogen, Myopathy, ERT, B cell, FDA, GAA, Fabry disease, Food, Physician, Blood, Therapy, Lysosome, Enzyme replacement therapy, Medicine, Pompe, Pharmaceutical industry

Once in the cell, Pombiliti can be properly processed into its most active and mature form to break down glycogen.

Key Points: 
  • Once in the cell, Pombiliti can be properly processed into its most active and mature form to break down glycogen.
  • Late-onset Pompe disease is a rare, debilitating, and life-threatening lysosomal disorder caused by a deficiency of the enzyme acid alpha-glucosidase (GAA).
  • This is the second WORLDSymposium™ New Treatment Award presented to Amicus.
  • Amicus received the award in 2017 for the first and only oral therapy approved for people living with Fabry disease.

Sangamo Therapeutics Announces Updated Phase 1/2 STAAR Study Data in Fabry Disease Showing Sustained Benefit and Differentiated Safety Profile

Retrieved on: 
Lunedì, Febbraio 5, 2024
Pharmaceutical, Genetics, Health, Clinical Trials, Partnership, COVID-19, Steroid, Trial of the century, ERT, LFT, Pharmacology, Patient, Safety, FDA, Antibody, Glomerular filtration rate, Investment, Common, Fatigue, Fabry disease, Neutralizing antibody, Physical, STAAR, Therapy, Quality of life, Kidney, Headache, EGFR, Sangamo Therapeutics, Plasma, Form, Pharmaceutical industry

Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced updated preliminary data from the Phase 1/2 STAAR clinical study evaluating isaralgagene civaparvovec, or ST-920, a wholly owned gene therapy product candidate for the treatment of Fabry disease.

Key Points: 
  • Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced updated preliminary data from the Phase 1/2 STAAR clinical study evaluating isaralgagene civaparvovec, or ST-920, a wholly owned gene therapy product candidate for the treatment of Fabry disease.
  • “Despite the availability of ERT and chaperone therapies, Fabry disease treatment is burdensome, with some patients still developing disease progression.
  • The most common adverse events were pyrexia, headache, COVID-19, fatigue and nasopharyngitis (majority Grade 1/2, with one Grade 3 pyrexia).
  • The data demonstrated supraphysiological plasma and liver α-Gal A activity in mouse models, supporting Phase 1/2 and potential Phase 3 clinical dosing.

Amicus Therapeutics Announces Presentations and Posters at the 20th Annual WORLDSymposium™ 2024

Retrieved on: 
Giovedì, Febbraio 1, 2024
Prevalence, Poster, Patient, Amicus Therapeutics, Fabry disease, Therapy

PRINCETON, N.J., Feb. 01, 2024 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), today announced that one oral presentation and 11 posters across its development programs will be included at the 20th Annual WORLDSymposium™ 2024 , being held February 4-9, 2024 in San Diego, CA.

Key Points: 
  • PRINCETON, N.J., Feb. 01, 2024 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), today announced that one oral presentation and 11 posters across its development programs will be included at the 20th Annual WORLDSymposium™ 2024 , being held February 4-9, 2024 in San Diego, CA.
  • Abstract Title: Multiorgan involvement in females with Fabry disease: Results from two Phase III trials and the followME registry (Poster #246)
    Abstract Title: Prevalence of migalastat-amenable mutations in patients with Fabry disease from Brazil, Argentina and Colombia (Poster #262)
    Presenter: Juan Politei, MD, Foundation for the Study of Neurometabolic Diseases, Buenos Aires, Argentina
    Abstract Title: Treatment-related benefit and satisfaction in patients with Fabry disease: Insight into patients’ expectations and preferences from the SATIS-Fab study (Poster #189)
    Presenter: Mark Roberts, MD, Salford Royal NHS Foundation Trust, Greater Manchester Neurosciences Centre, Manchester, U.K.
    Presenter: Benedikt Schoser, MD, Ludwig Maximilian University of Munich, Munich, Germany

uniQure Announces FDA Clearance of Investigational New Drug Application for AMT-191 Gene Therapy for Fabry Disease

Retrieved on: 
Mercoledì, Novembre 29, 2023
Phase, Liver, GLA, Food, IND, Fabry disease, FDA, Investigational New Drug, Haemophilia, Patient, Safety, Pharmaceutical industry

and AMSTERDAM, Nov. 29, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for AMT-191, the Company’s gene therapy candidate for Fabry disease .

Key Points: 
  • and AMSTERDAM, Nov. 29, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for AMT-191, the Company’s gene therapy candidate for Fabry disease .
  • AMT-191 comprises an AAV5 vector that delivers an α-galactosidase A (GLA) transgene designed to target the liver and produce the deficient GLA protein.
  • “AMT-191 has the potential to be a differentiated gene therapy for the one-time treatment of Fabry disease, incorporating a proprietary promoter and leveraging our validated AAV5 technology comprised within HEMGENIX®, an approved liver-directed gene therapy for the treatment of hemophilia B developed by uniQure.
  • The multicenter, open-label trial consists of two dose-escalating cohorts of three patients each to assess safety, tolerability, and efficacy of AMT-191 in patients with Fabry disease.