Fabry disease

Sangamo Therapeutics Reports Recent Business Highlights and First Quarter 2024 Financial Results

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Giovedì, Maggio 9, 2024
Biotechnology, Neurology, Health, Genetics, Pharmaceutical, STAC, FDA, CTA, Haemophilia, Enzyme replacement therapy, Kite, Investment, Nav1.7, Fabry disease, Poster, Disease, ERT, Sigma-Aldrich, STAAR, Health care, MAA, Partnership, Sangamo Therapeutics, MINT, AAV, Ligand Pharmaceuticals, IND, Tauopathy, Novartis, DNA, Patient, Pivotal, DSM-IV codes, EMA, Prion, BBB, Genome, BLA, MHRA, Manuscript, BioRxiv, Capsid, Modularity, Biogen, Therapy, Food and Drug Administration, European Medicines Agency, Gene, Pharmaceutical industry

Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today reported business highlights and first quarter 2024 financial results.

Key Points: 
  • Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today reported business highlights and first quarter 2024 financial results.
  • “We are pleased with the progress being made in business development discussions across our portfolio, including our Fabry disease program.
  • An IND submission is expected in the fourth quarter of 2024.
  • Revenues for the first quarter ended March 31, 2024 were $0.5 million, compared to $158.0 million for the same period in 2023.

uniQure Announces First Quarter 2024 Financial Results and Highlights Recent Company Progress

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Martedì, Maggio 7, 2024
FDA, Phase, Safety, Food, Fabry disease, Research, Immunosuppression, Male, Hand, IRB, Conference, Information technology, RBC Capital Markets, Patient, ALS, Acquisition, CSL, POC, Clinical trial, Temporal lobe epilepsy, SOD1, Pharmaceutical industry

LEXINGTON, Mass. and AMSTERDAM, May 07, 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 first quarter of 2024 and highlighted recent progress across its business.

Key Points: 
  • ~ Clinical trial initiation for Fabry disease on track to begin in second quarter of 2024, followed by refractory mesial temporal lobe epilepsy and SOD1-ALS in third quarter of 2024 ~
    LEXINGTON, Mass.
  • and AMSTERDAM, May 07, 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 first quarter of 2024 and highlighted recent progress across its business.
  • We have obtained IRB approvals for initial sites, which are poised for imminent activation, and have also identified prospective patients.
  • The Company expects cash, cash equivalents and investment securities will fund operations into the second quarter of 2027.

Chiesi Global Rare Diseases Announces Publication of Results from Fabry Disease Patient Survey

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Mercoledì, Aprile 24, 2024
Pain, Enzyme replacement therapy, Hearing loss, Fabry disease, ERT, Genetics, Disease, LGC, Patient, Fatigue, Perception, Quality of life, Pharmaceutical industry

BOSTON, April 24, 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 publication of results from a Fabry disease patient survey in the peer-reviewed Orphanet Journal of Rare Diseases.

Key Points: 
  • - Real-world evidence provides valuable insights about the management and monitoring of patients with Fabry disease for healthcare providers, researchers, and industry partners -
    BOSTON, April 24, 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 publication of results from a Fabry disease patient survey in the peer-reviewed Orphanet Journal of Rare Diseases.
  • "There continues to be a general lack of awareness and a limited appreciation of the numerous unique challenges that rare disease patients face every day, including those who are living with Fabry disease," said Giacomo Chiesi, head of Chiesi Global Rare Diseases.
  • The results of this survey will help us partner with healthcare providers to improve the care of patients with Fabry disease by addressing their most relevant unmet needs."
  • "Our analysis highlights the gap between current standard-of-care in disease monitoring and patient perception of disease progression among patients with Fabry disease."

Chiesi Group's 2023 Revenue Surpasses €3 Billion Mark, Reflecting 10% Growth Year-on-Year (+12% @CER), Underlining Commitment to Innovative, Sustainable Practices

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Lunedì, Aprile 22, 2024
Carbon, Science, Corporation, Enzyme replacement therapy, FDA, AIR, License, International, RARE, Investment, Climate, Fabry disease, Climate change, CER, B Corporation (certification), CDP, Partnership, Empowerment, Alpha-mannosidosis, Parent, CARE, Chronic obstructive pulmonary disease, Environment, Culture, Benefit corporation, Patient, Science Based Targets initiative, Bronchiectasis, Acquisition, Caregiver, Epidermolysis bullosa, GHG, Place of birth, Health, Neonatology, Ethics, Asthma, RDS, Infant respiratory distress syndrome, Growth, Carbon Disclosure Project, COPD, Pharmaceutical industry

PARMA, Italy, April 22, 2024 /PRNewswire/ -- In 2023, Chiesi, a research-oriented international biopharmaceutical group headquartered in Parma (Italy), with 31 affiliates worldwide, showcased the efficacy of its operating model centred around shared value, sustainability, and high ethical standards. Through this approach, Chiesi continues to deliver tangible benefits for patients, people, and the planet.

Key Points: 
  • Chiesi's fixed triple formulation for the treatment of asthma and COPD (Chronic Obstructive Pulmonary Disease) achieved a 27% @CER growth.
  • Also, in 2023 it was introduced in China, presenting a pivotal opportunity for growth and expanding access to treatment for more patients.
  • In March 2023, Chiesi enriched its AIR portfolio thanks to the partnership with Affibody AB to develop and commercialize innovative treatments for respiratory diseases.
  • In November 2023, Chiesi signed a License Agreement with Haisco Pharmaceutical to develop, manufacture, and commercialise a novel solution for bronchiectasis.

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

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Martedì, Aprile 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

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Martedì, Marzo 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: 
Mercoledì, Marzo 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

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Giovedì, Febbraio 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: 
Mercoledì, Febbraio 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

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Lunedì, Febbraio 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.