Fabry disease

Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding galactosidase alpha Treatment of Fabry disease, 19/10/2020 Withdrawn

Retrieved on: 
Tuesday, April 9, 2024
Eurostat, Diagnosis, Civil service commission, Fabry disease, Brain, Cell, CD34, Disease, Nervous system, Patient, Committee, Knowledge, Tissue, Stroke, Kidney failure, Fats, Regulation, Autotransplantation, EMA, IRIS, Human, Eye, Sugar, European, Skin, COMP, Heart, Sponsor, European Commission, Enzyme replacement therapy, Blood, PPD, Pain, Safety, Marketing, Bone marrow, Medicine, Pharmaceutical industry

Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding galactosidase alpha Treatment of Fabry disease, 19/10/2020 Withdrawn

Key Points: 


Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding galactosidase alpha Treatment of Fabry disease, 19/10/2020 Withdrawn

CENTOGENE Extends Strategic Partnership With Takeda to Continue Providing Access to Genetic Testing for Patients With Lysosomal Storage Disorders

Retrieved on: 
Tuesday, March 19, 2024
Diagnosis, Gaucher's disease, Disease, Partnership, Fabry, Shire (pharmaceutical company), LSD, Patient, Hunter syndrome, Glycogen storage disease, Fabry disease, Public Investment Fund, Neurodegenerative disease, Pharmaceutical industry

and ROSTOCK, Germany and BERLIN, March 19, 2024 (GLOBE NEWSWIRE) -- Centogene N.V. (Nasdaq: CNTG), the essential life science partner for data-driven answers in rare and neurodegenerative diseases, today announced the extension of an ongoing partnership with Takeda (TSE: 4502/NYSE: TAK) to diagnose patients with Lysosomal Storage Disorders (LSDs).

Key Points: 
  • and ROSTOCK, Germany and BERLIN, March 19, 2024 (GLOBE NEWSWIRE) -- Centogene N.V. (Nasdaq: CNTG), the essential life science partner for data-driven answers in rare and neurodegenerative diseases, today announced the extension of an ongoing partnership with Takeda (TSE: 4502/NYSE: TAK) to diagnose patients with Lysosomal Storage Disorders (LSDs).
  • Under the renewed agreement, CENTOGENE will continue to provide Takeda with access to diagnostic testing for patients around the world.
  • The agreement was established to enhance patient access to rapid and reliable diagnostics for LSDs, including Fabry disease, Gaucher disease, and Hunter syndrome.
  • “Extending our longstanding partnership with Takeda highlights the continuing need to accelerate diagnoses for LSD patients globally and the ability of CENTOGENE to fulfill this vital service,” said Ian Rentsch, CENTOGENE Chief Commercial Officer and General Manager - Pharma.

Sangamo Therapeutics Reports Recent Business Highlights and Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
Wednesday, March 13, 2024
Health, Neurology, Genetics, Clinical Trials, Pharmaceutical, Biotechnology, STAC, Gene, Orphan drug, ALTA, Haemophilia A, BBB, Patient, Health care, Quality of life, IND, Male, ERT, Investment, Therapy, Fabry disease, Plasma, Kite, Gene expression, Partnership, Central nervous system, Novartis, CNS, Pivotal, EMA, Prion, STAAR, Enzyme replacement therapy, Biogen, Tauopathy, Workforce, BLA, AAV, Sanofi, Research, DRG, Neuron, Hematology, FDA, DSM-IV codes, Brain, Sangamo Therapeutics, MAA, Haemophilia, CTA, Mouse, Female, Traffic barrier, Sodium, MHRA, Excipient, Society, Spinal cord, Toxicology, Sigma-Aldrich, Liver, Pharmaceutical industry

Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today reported recent business highlights and fourth quarter and full year 2023 financial results, including meaningful data to support advancement of its neurology pipeline.

Key Points: 
  • Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today reported recent business highlights and fourth quarter and full year 2023 financial results, including meaningful data to support advancement of its neurology pipeline.
  • “In 2023, Sangamo announced the prioritization of its pipeline programs that support our focus as a neurology-focused genomic medicine company,” said Sandy Macrae, Chief Executive Officer of Sangamo.
  • STAC-BBB was well tolerated in NHPs, with no notable treatment related pathological findings in brain, spinal cord or peripheral tissues.
  • Revenues for the fourth quarter ended December 31, 2023 were $2.0 million, compared to $27.2 million for the same period in 2022.

4DMT Reports Full Year 2023 Financial Results and Operational Highlights

Retrieved on: 
Thursday, February 29, 2024
Therapy, Atypical hemolytic uremic syndrome, Chelsea Handler, Fabry disease, Respiratory disease, EMA, Injection, Research, CRISPR, Cystic fibrosis, FDA, Partnership, Inflammation, Retinitis pigmentosa, Security (finance), Overalls, Biopsy, Șaeș, PRISM, Conference, Cash, Diabetic retinopathy, Geographic atrophy, Astellas Pharma, CFTR, CNS, PRIME, AMD, Choroideremia, Patient, Safety, Clinical trial, Angiogenesis, VG, DME, AAV, Vaccine

We currently expect cash and cash equivalents to be sufficient to fund operations into the first half of 2027.

Key Points: 
  • We currently expect cash and cash equivalents to be sufficient to fund operations into the first half of 2027.
  • R&D Expenses: Research and development expenses were $97.1 million for 2023, as compared to $80.3 million for 2022.
  • G&A Expenses: General and administrative expenses were $36.5 million for 2023, as compared to $32.9 million for 2022.
  • Net Loss: Net loss was $100.8 million for 2023, as compared to net loss of $107.5 million for 2022.

uniQure Announces 2023 Financial Results and Highlights Recent Company Progress

Retrieved on: 
Wednesday, February 28, 2024
Acquisition, Fabry disease, TMS, Natural history, CSL Behring, ALS, Research, FDA, Partnership, Cerebrospinal fluid, Food, CSL, Etranacogene dezaparvovec, Sale, TFC, Conference, SOD1, Unified, Information technology, AFL, Immunosuppression, Patient, Disease, Phase, Hand, IND, CGMP, State, Temporal lobe epilepsy, Pharmaceutical industry

LEXINGTON, Mass. and AMSTERDAM, Feb. 28, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today reported its financial results for the fourth quarter and full year 2023 and highlighted recent progress across its business.

Key Points: 
  • and AMSTERDAM, Feb. 28, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today reported its financial results for the fourth quarter and full year 2023 and highlighted recent progress across its business.
  • “We are pleased with the progress made across the company in 2023 and are now laser-focused on execution across multiple clinical programs,” stated Matt Kapusta, chief executive officer of uniQure .
  • By the end of 2024, the Company expects to have greater clarity regarding a potential approval pathway for AMT-130.
  • AMT-191 for the treatment of Fabry disease – In November 2023, the Company announced the clearance of an IND for the Phase I/IIa clinical study of AMT-191.

AceLink Therapeutics Announces Publication of Phase 1 Clinical Trial Data Evaluating AL01211 in Healthy Volunteers

Retrieved on: 
Monday, February 26, 2024
Biotechnology, Pharmaceutical, Health, Clinical Trials, Fabry disease, PD, GCS, Drug development, Strong, Risk, Doctor of Philosophy, Safety, Therapy, Pharmacology, Pharmacokinetics, CNS, American College, Development, Patient, Pharmaceutical industry

AL01211 is a potent, oral, glucosylceramide synthase (GCS) inhibitor being developed for the treatment of Fabry disease and Type 1 Gaucher disease.

Key Points: 
  • AL01211 is a potent, oral, glucosylceramide synthase (GCS) inhibitor being developed for the treatment of Fabry disease and Type 1 Gaucher disease.
  • “These results highlight the transformative potential of AL01211 as a best-in-class GCS inhibitor,” said Jerry Shen, Ph.D., Chief Executive Officer and Founder of AceLink Therapeutics.
  • “Our Phase 1 study provided the critical safety and biomarker data to support our ongoing Phase 2 clinical trial in patients with Fabry Disease.
  • “In our trial, AL01211 demonstrated dose-dependent PK and PD effects with a clear correlation between AL01211 exposure and reduction in disease biomarkers.

Latest Updates of Viva Biotech's Portfolio Companies

Retrieved on: 
Wednesday, March 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: 
Thursday, February 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: 
Wednesday, February 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: 
Monday, February 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.