ERT

Orchard Therapeutics Announces First Patient Randomized in Registrational Trial of OTL-203 for MPS-I Hurler Syndrome

Retrieved on: 
Monday, February 5, 2024
Partnership, Mortality, Hurler, Orchard, Growth, Congress, Cell, Division, Heart, ERT, Glycosaminoglycan, GAG, Health, Disease, Patient, Safety, Transplant, FDA, EMA, Șaeș, San Raffaele Hospital, ESGCT, HSCT, Food, POC, Therapy, MPS, Hurler syndrome, Kyowa Kirin, Acquisition, Enzyme replacement therapy, European Medicines Agency, Gene, University, Eye, IDUA, HSC, RPD, Pharmaceutical industry

TOKYO, LONDON and BOSTON, Feb. 05, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients around the globe, today announced the first patient has been randomized at the M Health Fairview Masonic Children’s Hospital in a registrational trial evaluating the efficacy and safety of OTL-203, an investigational hematopoietic stem cell (HSC) gene therapy, in patients with the Hurler subtype of mucopolysaccharidosis type I (MPS-IH). The trial, referred to as HURCULES, compares treatment with OTL-203 to standard of care with allogeneic hematopoietic stem cell transplant (HSCT), and is expected to enroll 40 MPS-IH patients at sites across the U.S. and Europe.

Key Points: 
  • Approximately 60 percent of children born with MPS-I have the most severe subtype, MPS-IH, also called Hurler syndrome, and rarely live past the age of 10 when untreated.
  • Current treatment options for MPS-IH include allogeneic hematopoietic stem cell transplant (HSCT) and enzyme replacement therapy (ERT), both of which have significant limitations.
  • One patient experienced an acute hypersensitivity reaction that was considered probably related to HSC gene therapy.
  • The viral vector integration profile was comparable with other Orchard Therapeutics lentiviral-based HSC gene therapy studies, and all participants had a stable and highly polyclonal repertoire.

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

Retrieved on: 
Monday, February 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.

JCR Pharmaceuticals to Present at the 20th Annual WORLDSymposium™ 2024

Retrieved on: 
Thursday, January 25, 2024
Health, Other Science, General Health, Research, Science, Pharmaceutical, Biotechnology, Hurler, Patient, Enzyme replacement therapy, PST, Hunter syndrome, MPS II, JCR, Eto, BBB, MPS, Medicine, MHLW, Ikeda, ERT, Syndrome, Safety, Traffic barrier, Glycogen storage disease, Scheie syndrome, Ministry, Pharmaceutical industry

JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) announced today that it will present six presentations at the 20th Annual WORLDSymposium™ 2024, to be held February 4-9, 2024 in San Diego, Calif.

Key Points: 
  • JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) announced today that it will present six presentations at the 20th Annual WORLDSymposium™ 2024, to be held February 4-9, 2024 in San Diego, Calif.
  • In addition to posters being available for each of the presentations, two of them will be shared as oral presentations given by a clinical investigator and a researcher from JCR Pharmaceuticals.
  • A global phase III study of pabinafusp alfa (JR-141) for neuronopathic mucopolysaccharidosis type II: updated study design (Ikeda, et al.)
  • WORLDSymposium™ attendees who would like to receive more information about JCR Pharmaceuticals can visit JCR’s on-site conference booth (#506) or visit its virtual booth on the WORLDSymposium™ conference website.

ConcertAI Adds Healthtech Leader as Senior Vice President and General Manager of Digital Trial Solutions

Retrieved on: 
Wednesday, January 24, 2024
Senior, Philosophy, Form 1099, Patient, Research, Clinical trial, Software as a service, Full, Allegheny College, University, Business, Cancer, MBA, ERT, Generative, Psychology, Doctor of Philosophy, UPMC Hillman Cancer Center, Motivation, DTS, Nursing

CAMBRIDGE, Mass., Jan. 24, 2024 /PRNewswire/ -- ConcertAI, the leader in oncology predictive and generative AI SaaS Solutions and Real-World Data for healthcare and life sciences, today announced that Ryan Kennedy has joined the company as Senior Vice President and General Manager of ConcertAI's Digital Trial Solutions™ (DTS) offering.

Key Points: 
  • CAMBRIDGE, Mass., Jan. 24, 2024 /PRNewswire/ -- ConcertAI, the leader in oncology predictive and generative AI SaaS Solutions and Real-World Data for healthcare and life sciences, today announced that Ryan Kennedy has joined the company as Senior Vice President and General Manager of ConcertAI's Digital Trial Solutions™ (DTS) offering.
  • Ryan brings over two decades of oncology clinical trials and clinical development industry experience in trial management and SaaS solutions.
  • He will lead the DTS business' Software-as-a-Service (SaaS) Suite, which is comprised of the Clinical Trial Optimization™ solution, DTS Patient-to-trial matching, and DTS Full eCRF automations.
  • "I am thrilled to join the ConcertAI team to help bring truly life-changing solutions to our sites, networks, and customers.

European Medicines Agency (EMA) Grants Orphan Drug Designation (ODD) to GC Biopharma's Treatment for Sanfilippo Syndrome (Type A)

Retrieved on: 
Tuesday, January 23, 2024
Patient, ICV, European Medicines Agency, Sanfilippo syndrome, Heparan sulfate, MPS, Enzyme replacement therapy, EMA, Life expectancy, ODD, ERT, Safety, Central nervous system, Drug development, Cerebrospinal fluid, Genetic disorder, Pharmaceutical industry

GC1130A has previously achieved notable milestones by securing both Rare Pediatric Disease designation (RPDD) and Orphan Drug Designation (ODD) from the U.S. FDA in January 2023.

Key Points: 
  • GC1130A has previously achieved notable milestones by securing both Rare Pediatric Disease designation (RPDD) and Orphan Drug Designation (ODD) from the U.S. FDA in January 2023.
  • Sanfilippo Syndrome (type A) is a genetic disorder that triggers central nervous system damage by accumulating Heparan sulfate, leading to a progressive neurodegeneration in pediatric population.
  • "This EMA designation further underlines the potentials of our collaborative pipeline in addressing the disease pathology in upcoming clinical trials.
  • ", said GC Biopharma and added that it will be "committing to expeditiously advancing into clinical trials, acknowledging the urgent, unmet medical needs of Sanfilippo Syndrome patients."

ILUS Acquires the Majority Stake of Samsara Luggage, Inc.

Retrieved on: 
Wednesday, January 10, 2024
Special-purpose entity, Eye, SAML, Social media, NRS, FINRA, OTC, ERT, Acquisition, Mergers and acquisitions, REV, LTD, Growth, Security (finance)

The company confirms that is has acquired the controlling interest in the SEC Reporting OTC listed company, Samsara Luggage, Inc. (OTC: SAML).

Key Points: 
  • The company confirms that is has acquired the controlling interest in the SEC Reporting OTC listed company, Samsara Luggage, Inc. (OTC: SAML).
  • On January 3, 2024, ILUS acquired a convertible note from YAII PN, LTD with outstanding principal and accrued interest of $600,684.93.
  • On January 5, 2024, ILUS converted the note into 150,753,425 shares of common stock in SAML pursuant to the terms of the exchange note which was filed in ILUS’ Schedule 13D.
  • As a result of the conversion, as of January 5, 2024, ILUS acquired control of 91.5% of the outstanding shares in SAML.

Radware Protects Leading European Bank Against Emerging Threat Vectors

Retrieved on: 
Tuesday, January 9, 2024
Cloud, Knowledge, Geography, GEL, Trust, DNS, Partnership, API, Algorithm, Gartner, Denial-of-service attack, Gigaom, Forrester Research, ERT, Quadrant, Organization, Alteon, Machine learning, OWASP, Incident response team, Internet of things

MAHWAH, N.J., Jan. 09, 2024 (GLOBE NEWSWIRE) -- Radware ® (NASDAQ: RDWR), a leading provider of cyber security and application delivery solutions, announced it signed an agreement with a leading European financial institution.

Key Points: 
  • MAHWAH, N.J., Jan. 09, 2024 (GLOBE NEWSWIRE) -- Radware ® (NASDAQ: RDWR), a leading provider of cyber security and application delivery solutions, announced it signed an agreement with a leading European financial institution.
  • Today, the bank, which has offices in more than 50 countries and thousands of employees, delivers a comprehensive set of financial services to private, corporate, and institutional clients.
  • The bank decided to replace its existing security solutions provider to strengthen its protection against emerging threat vectors and enhance data availability and accessibility.
  • Industry analysts such as Aite-Novarica Group, Forrester Research, Gartner, GigaOm, KuppingerCole, and Quadrant Knowledge Solutions continue to recognize Radware as a market leader in cyber security.

NEC Achieves TX-RAMP Level 2 Certification for Suite of Biometric and Face Recognition Solutions

Retrieved on: 
Friday, January 12, 2024
Security, Air, White House, Federal Government, Audio, Video, Transport, State, Local, Software, Networks, Internet, Homeland Security, Data Management, Technology, Mobile, Wireless, Public Policy, Government, Law Enforcement, Emergency Services, Other Travel, Transportation, Travel, Certification, Privacy, Database, Cloud computing, DIR, NEC, NFR, ERT, NEC Corporation of America, FedRAMP, Web browser, ARS, Archive, Image, Information, Medical device

The certification encompasses Integra-ID®, NeoFace® Reveal (NFR), and Insights for NEC Advanced Recognition Systems (ARS).

Key Points: 
  • The certification encompasses Integra-ID®, NeoFace® Reveal (NFR), and Insights for NEC Advanced Recognition Systems (ARS).
  • This expanded certification underscores NEC's commitment to delivering cutting-edge solutions with a rigorous security-by-design approach to ensure privacy for all.
  • "We are proud to receive Level 2 Certification from TX-RAMP for our ARS Law Enforcement Biometric Product Suite.
  • This recognition reflects NEC's unwavering commitment to providing advanced and secure solutions while prioritizing privacy,” said Gary Lac, NEC Vice President of Solutions Development, ARS.

Immusoft To Present on ISP-001 Clinical Trial at WORLDSymposium™ 2024 Conference

Retrieved on: 
Wednesday, January 3, 2024
Research, Genetics, Clinical Trials, Stem Cells, Other Health, Biotechnology, Pharmaceutical, Health, Science, Other Science, Immunosuppression, Lysosomal storage disease, Conference, Risk, Infection, Iduronidase, Bone marrow, Hair loss, Infertility, Disease, Enzyme replacement therapy, Nausea, MPS I, Immune system, Clinical trial, MPS, University, ERT, Corporation, IDUA, ISP, Cell, Patient, Cell biology, Genetic engineering, Vaccine

Immusoft Corporation , a cell therapy company dedicated to improving the lives of patients with rare diseases, today announced that it will present at the upcoming WORLDSymposium 2024 Conference, held in San Diego, February 4-9, 2024.

Key Points: 
  • Immusoft Corporation , a cell therapy company dedicated to improving the lives of patients with rare diseases, today announced that it will present at the upcoming WORLDSymposium 2024 Conference, held in San Diego, February 4-9, 2024.
  • Immusoft is developing a new modality of gene encoded medicine that holds the promise of being durable and redosable.
  • The Company’s lead investigational drug candidate ISP-001, is a first-in-class investigational treatment for mucopolysaccharidosis type I (MPS I), a rare lysosomal storage disease.
  • In this first-in-human clinical trial, the patient was dosed without the need for a preconditioning regimen (required for gene modified stem cells) or immunosuppression (required for most systemic virus-delivered gene therapy).

ILUS Completes Acquisition and Provides Dividend, Merger and Subsidiary Update

Retrieved on: 
Friday, December 29, 2023
Replay Solutions, Sale, Acquisition, Emergency, S-1, Mining, ERT, Exchange, Growth, Mergers and acquisitions, OTC, Security (finance), Shareholder Meeting

An update is provided on several matters of importance for Shareholders including the acquisition, an associated dividend for ILUS Shareholders, merger agreement negotiations, subsidiaries, and financing.

Key Points: 
  • An update is provided on several matters of importance for Shareholders including the acquisition, an associated dividend for ILUS Shareholders, merger agreement negotiations, subsidiaries, and financing.
  • Following ERT’s acquisition of Emergency Response assets from ILUS, it intends to pay a special equity dividend to ILUS Shareholders and ERT has appointed its own legal counsel with whom it is finalizing the matter.
  • The structure of the dividend is such that following its intended payment to ILUS Shareholders, ILUS will retain its controlling interest in ERT.
  • Having been approached, the ILUS management team is currently in discussions regarding a merger with a NASDAQ listed company.