Fabry

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.

Amicus Therapeutics Announces Third Quarter 2023 Financial Results and Corporate Updates

Retrieved on: 
Wednesday, November 8, 2023
Loss, Blackstone Credit, PIN, CER1, Fabry, GAAP, Acquisition, Growth, Depreciation, Amicus Therapeutics, Therapy, Table, Patient, Blackstone, Diagnosis, CER, Pharmaceutical industry, Video game, Cryptocurrency, Bank, Migalastat, EU, Cipaglucosidase alfa

Based on the current operating plan, the Company is on-track to achieve non-GAAP profitability2 in the fourth quarter of 2023, a major milestone for Amicus.

Key Points: 
  • Based on the current operating plan, the Company is on-track to achieve non-GAAP profitability2 in the fourth quarter of 2023, a major milestone for Amicus.
  • Total revenue in the third quarter 2023 was $103.5 million, a year-over-year increase of 27% from total revenue of $81.7 million in the third quarter 2022.
  • ET to discuss the third quarter 2023 financial results and corporate updates.
  • A live audio webcast and related presentation materials can also be accessed via the Investors section of the Amicus Therapeutics corporate website at ir.amicusrx.com .

4DMT Gains Alignment with FDA on Plan to Lift Clinical Hold on Phase 1/2 INGLAXA Clinical Trial for 4D-310 for Fabry Disease Cardiomyopathy

Retrieved on: 
Monday, October 30, 2023
FDA, AAV, Chelsea Handler, Food, IV, Therapy, Fabry, Risk, Atypical hemolytic uremic syndrome, Safety, Patient, Pharmaceutical industry

“We are delighted to have reached an agreement with the FDA on our proposed plan to address the clinical hold and continue trial development,” said David Kirn, M.D., Co-founder and Chief Executive Officer of 4DMT.

Key Points: 
  • “We are delighted to have reached an agreement with the FDA on our proposed plan to address the clinical hold and continue trial development,” said David Kirn, M.D., Co-founder and Chief Executive Officer of 4DMT.
  • Consistent with the Company’s plans, the FDA subsequently notified the Company of a clinical hold in the U.S.
  • The Asia-Pacific clinical trial program was not placed on clinical hold by any regulatory agency.
  • In addition, the INGLAXA clinical trial protocol has been amended to minimize the risk of aHUS following IV 4D-310 dosing, including requiring the R/S immunosuppressive regimen.

Sangamo Therapeutics Announces Strategic Update and Reports Third Quarter 2023 Financial Results

Retrieved on: 
Wednesday, November 1, 2023
Health, General Health, Research, Pharmaceutical, Science, Biotechnology, Regenerative Medicine Advanced Therapy, Ethics, Therapy, Pfizer, Gene, Partnership, Head, Exposition, European Society of Gene and Cell Therapy, ALTA, AAV, Haemophilia, Hope, 65th Street Yard, Workforce, ERT, DSM-IV codes, Cell, CAR, U.S. FDA, Safety, Investment, Patient, Central nervous system, Nav1.7, MAA, 22q13 deletion syndrome, Conference, Enzyme replacement therapy, Hematology, Quality of life, Mace Head Atmospheric Research Station, Novartis, Brain, European Society for Primary Immunodeficiencies, Haemophilia A, Spectrum, FDA, IND, Fabry disease, Prion, Society, Senior, SCN2A, ESGCT, STAAR, Intellectual disability, Sclerosis, BLA, Pharmaceutical industry, Management, Vaccine, Biogen, Neuroscience, Fabry, Sangamo Therapeutics

Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today announced recent business highlights, including progress on its strategic transformation and a corresponding restructuring of operations and workforce reduction, and reported third quarter 2023 financial results.

Key Points: 
  • Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today announced recent business highlights, including progress on its strategic transformation and a corresponding restructuring of operations and workforce reduction, and reported third quarter 2023 financial results.
  • As a result of this restructuring, Sangamo is reducing its US workforce by approximately 40%, or approximately 162 roles.
  • Sangamo expects to incur approximately $8 million-$10 million in one-time restructuring costs in the fourth quarter of 2023.
  • Revenues for the third quarter ended September 30, 2023 were $9.4 million, compared to $26.5 million for the same period in 2022.

uniQure Announces Strategic Reorganization to Reduce Operating Expenses and Support Advancement of Multiple Clinical-Stage Programs

Retrieved on: 
Thursday, October 5, 2023
Neuroscience, ALS, Acquisition, Temporal lobe epilepsy, CSL Behring, CSL, Chief scientific officer, Risk, Fabry disease, IND, Patient, FDA, GMP, Alzheimer's disease, Workforce, Phase, AAV, Rich Porter, MA, CNS, Research, Genetic distance, Construction, Management, Pharmaceutical industry, Fabry

LEXINGTON, Mass. and AMSTERDAM, Oct. 05, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced a strategic reorganization that will significantly reduce operating expenses while supporting focused execution to rapidly advance multiple clinical-stage programs to proof-of-concept.

Key Points: 
  • and AMSTERDAM, Oct. 05, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced a strategic reorganization that will significantly reduce operating expenses while supporting focused execution to rapidly advance multiple clinical-stage programs to proof-of-concept.
  • “At uniQure, our highest priority is to deliver innovative, life-changing therapies to patients with significant unmet needs.
  • To accomplish our mission and generate near-term value for our stakeholders, we will implement a strategic restructuring of our business,” stated Matt Kapusta, chief executive officer of uniQure .
  • “We are taking important actions today to cut operating expenses while ensuring that we have the necessary resources to advance our prioritized clinical-stage programs as rapidly as possible to proof-of-concept.

Fabry Disease Industry Insights 2023-2032: Epidemiology, Drug Pipeline, Emerging Treatments, Market Trends, Expert Opinions - ResearchAndMarkets.com

Retrieved on: 
Friday, August 25, 2023
Health, Other Health, EU4, Subject-matter expert, Chiesi Farmaceutici, Internet, USD, AAV, Protalix BioTherapeutics, ERT, GLA, Sangamo Therapeutics, Growth, Late onset congenital adrenal hyperplasia, Classic, Enzyme replacement therapy, Epidemiology, PEGylation, SME, Pharmaceutical industry, Vaccine, Video game, Dentistry, Fabry, EU, Fabry disease

This comprehensive analysis offers valuable insights into the Fabry Disease market, including epidemiology, drug pipeline, emerging treatments, market trends, and expert opinions, ultimately assisting stakeholders in understanding the evolving landscape and potential opportunities.

Key Points: 
  • This comprehensive analysis offers valuable insights into the Fabry Disease market, including epidemiology, drug pipeline, emerging treatments, market trends, and expert opinions, ultimately assisting stakeholders in understanding the evolving landscape and potential opportunities.
  • The report provides a comprehensive understanding of Fabry Disease epidemiology, including historical and forecasted data.
  • The Fabry Disease market experienced 15,290 diagnosed prevalent cases in the 7MM in 2022, with 8,355 cases in the US alone.
  • The Fabry Disease market size is projected to increase at a CAGR of 6.3% from 2023 to 2032, driven by awareness and the launch of emerging drugs.

AceLink Opens First Clinical Site in China for Phase 2 Study in Fabry Disease

Retrieved on: 
Thursday, August 10, 2023
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, PK, PD, IV, Aes, Clinical trial, Fabry disease, Pharmacokinetics, Quality of life, Ruijin Hospital, Nephrology, Trust, Patient, CDE, National Medical Products Administration, GCS, Principal, Center for Drug Evaluation and Research, Therapy, Strong, IND, NMPA, Safety, Pharmaceutical industry, Fabry

AceLink Therapeutics, Inc., a clinical stage biopharmaceutical company, today announced the opening of the first clinical trial site in China for its Phase 2, open‑label study of the safety, pharmacokinetics (PK), pharmacodynamics (PD) and preliminary measures of physiological efficacy of AL01211 in males with classic Fabry disease who have not been previously treated with other Fabry disease therapies.

Key Points: 
  • AceLink Therapeutics, Inc., a clinical stage biopharmaceutical company, today announced the opening of the first clinical trial site in China for its Phase 2, open‑label study of the safety, pharmacokinetics (PK), pharmacodynamics (PD) and preliminary measures of physiological efficacy of AL01211 in males with classic Fabry disease who have not been previously treated with other Fabry disease therapies.
  • AL01211 is a novel, oral, non-brain penetrant glucosylceramide synthase (GCS) inhibitor being developed for the treatment of Fabry disease.
  • It has completed a phase 1 clinical trial in healthy volunteers in Australia and a bridging phase 1 trial in China under IND approved by the Center for Drug Evaluation (CDE) of China's National Medical Products Administration (NMPA).
  • With this in mind, we are truly excited about our participation in this trial, as we strive to improve the daily lives of patients with Fabry disease.”

Sangamo Therapeutics Reports Recent Business Highlights and Second Quarter 2023 Financial Results

Retrieved on: 
Tuesday, August 8, 2023
Biotechnology, Pharmaceutical, Genetics, Health, Pfizer, Central nervous system, Therapy, Gene, Haemophilia A, BLA, Biogen, Safety, Fabry disease, Phytoene synthase, Research, Brain, AAV, Science, Fast track (FDA), U.S. FDA, Sodium, Phase 3, Nav1.7, Novartis, Patient, Prion, Primate, Annual general meeting, Cell, American Society for Cell Biology, Eli Lilly and Company, MAA, FDA, STAAR, Enzyme replacement therapy, IND, Neurology, Haemophilia, Society, Pharmaceutical industry, Medical device, Vaccine, Fabry, Chroma, Sanofi, Sangamo Therapeutics

Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today reported recent business highlights and second quarter 2023 financial results.

Key Points: 
  • Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today reported recent business highlights and second quarter 2023 financial results.
  • We are pleased to have executed several business development deals, demonstrating the excitement in Sangamo’s science and platform,” said Sandy Macrae, Chief Executive Officer of Sangamo.
  • Revenues for the second quarter ended June 30, 2023 were $6.8 million, compared to $29.4 million for the same period in 2022.
  • Additionally, revenues relating to our collaboration agreement with Kite decreased by $5.1 million, reflecting a reduction in collaboration activities during the quarter.

Amicus Therapeutics Announces Second Quarter 2023 Financial Results and Corporate Updates

Retrieved on: 
Tuesday, August 8, 2023
Fabry, Loss, Table, Civil service commission, CER, CER1, Amicus, GAAP, PIN, Retirement, Acquisition, Patient, Cipaglucosidase alfa, Growth, CFO, Depreciation, Diagnosis, Commercial, Amicus Therapeutics, Therapy, European Commission, Pharmaceutical industry, Video game, Cryptocurrency, EU, Migalastat

The Company continues to expect regulatory approvals of AT-GAA in both the U.S. and U.K. in the third quarter of 2023.

Key Points: 
  • The Company continues to expect regulatory approvals of AT-GAA in both the U.S. and U.K. in the third quarter of 2023.
  • Total revenue in the second quarter 2023 was $94.5 million, a year-over-year increase of 17% from total revenue of $80.7 million in the second quarter 2022.
  • Total GAAP operating expenses of $104.2 million for the second quarter 2023 decreased as compared to $133.1 million for the second quarter 2022.
  • ET to discuss the second quarter 2023 financial results and corporate updates.

Protalix BioTherapeutics Reports Second Quarter 2023 Financial and Business Results

Retrieved on: 
Monday, August 7, 2023
Protalix BioTherapeutics, Medsafe, European Medicines Agency, Tax, MD, Patient, European, Fabry disease, BLA, NYSE, Calendar, Gout, Half-life, Pharmacokinetics, Research, EMA, Russell, FIH, Growth, FDA, PEGylation, Biologics license application, Safety, Civil service commission, Health, Baylor University Medical Center, Israel Innovation Authority, FASN, Webcast, MAA, HDEC, European Commission, KOL, Clinical trial, EDT, Marketing, ERT, EC, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Food, Vaccine, Pharmaceutical industry, Drug, Life insurance, Online shopping, EU, Fabry, Chiesi Farmaceutici

CARMIEL, Israel, Aug. 7, 2023 /PRNewswire/ -- Protalix BioTherapeutics, Inc. (NYSE American: PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx® plant cell-based protein expression system, today reported financial results for the second quarter ended June 30, 2023 and provided a business update on recent regulatory, clinical and corporate developments.

Key Points: 
  • "2023 has been a transformational year for Protalix thus far," said Dror Bashan, Protalix's President and Chief Executive Officer.
  • On May 5, 2023, the European Commission (EC) granted marketing authorization to Elfabrio (pegunigalsidase alfa) in the European Union.
  • On June 26, 2023, the Company was included in the broad-market Russell 3000® Index at the conclusion of the 2023 Russell indexes annual reconstitution.
  • Financial expenses, net were $0.8 million for the three months ended June 30, 2023, compared to financial income, net of $0.2 million for the three months ended June 30, 2022.