Sanfilippo syndrome

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

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Wednesday, February 14, 2024
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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.

Kyowa Kirin successfully completes acquisition of Orchard Therapeutics, a global gene therapy leader for rare diseases

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Wednesday, January 24, 2024
Patient, MHRA, Therapy, Health care, Biotechnology, Civil service commission, European Commission, Kyowa Kirin, Sanfilippo syndrome, ADS, Food, MLD, Acquisition, Acceleration, CVR, FDA, Priority review, Sanfilippo, HSC, PDUFA, Pharmaceutical industry

TOKYO and LONDON and BOSTON, Jan. 24, 2024 (GLOBE NEWSWIRE) -- Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE: 4151) a Japan-based global specialty pharmaceutical company (J-GSP) discovering and developing novel medicines utilizing the latest biotechnology, and Orchard Therapeutics plc (Orchard Therapeutics, Nasdaq: ORTX), a global gene therapy leader, today announced Kyowa Kirin has successfully completed the acquisition of Orchard Therapeutics.

Key Points: 
  • TOKYO and LONDON and BOSTON, Jan. 24, 2024 (GLOBE NEWSWIRE) -- Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE: 4151) a Japan-based global specialty pharmaceutical company (J-GSP) discovering and developing novel medicines utilizing the latest biotechnology, and Orchard Therapeutics plc (Orchard Therapeutics, Nasdaq: ORTX), a global gene therapy leader, today announced Kyowa Kirin has successfully completed the acquisition of Orchard Therapeutics.
  • The acquisition of all outstanding shares of Orchard Therapeutics by way of a Scheme of Arrangement procedure under the UK Companies Act 2006 was completed on January 24, 2024, and Orchard Therapeutics has become a wholly-owned subsidiary of Kyowa Kirin.
  • “We are truly excited about the acquisition of Orchard Therapeutics, a leading provider of HSC gene therapy.
  • Kyowa Kirin anticipates significant synergies with Orchard Therapeutics and plans to announce its 2024 Financial Guidance in conjunction with its 2023 earnings on 7 February.

Kyowa Kirin Successfully Completes Acquisition of Orchard Therapeutics, a Global Gene Therapy Leader for Rare Diseases

Retrieved on: 
Wednesday, January 24, 2024
Health, Genetics, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Patient, MHRA, Therapy, Health care, Civil service commission, European Commission, Kyowa Kirin, Sanfilippo syndrome, ADS, Food, MLD, Acquisition, Acceleration, CVR, FDA, Priority review, Sanfilippo, HSC, PDUFA, Pharmaceutical industry

Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE: 4151) a Japan-based global specialty pharmaceutical company (J-GSP) discovering and developing novel medicines utilizing the latest biotechnology, and Orchard Therapeutics plc (Orchard Therapeutics, Nasdaq: ORTX), a global gene therapy leader, today announced Kyowa Kirin has successfully completed the acquisition of Orchard Therapeutics.

Key Points: 
  • Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE: 4151) a Japan-based global specialty pharmaceutical company (J-GSP) discovering and developing novel medicines utilizing the latest biotechnology, and Orchard Therapeutics plc (Orchard Therapeutics, Nasdaq: ORTX), a global gene therapy leader, today announced Kyowa Kirin has successfully completed the acquisition of Orchard Therapeutics.
  • The acquisition of all outstanding shares of Orchard Therapeutics by way of a Scheme of Arrangement procedure under the UK Companies Act 2006 was completed on January 24, 2024, and Orchard Therapeutics has become a wholly-owned subsidiary of Kyowa Kirin.
  • “We are truly excited about the acquisition of Orchard Therapeutics, a leading provider of HSC gene therapy.
  • Kyowa Kirin anticipates significant synergies with Orchard Therapeutics and plans to announce its 2024 Financial Guidance in conjunction with its 2023 earnings on 7 February.

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

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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."

Denali Therapeutics Announces Key Anticipated 2024 Milestones and Priorities to Further Advance Its Therapeutics Portfolio for Neurodegeneration and Lysosomal Storage Diseases

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Monday, January 8, 2024
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The global Phase 2/3 COMPASS study continues to recruit up to 54 participants with neuronopathic and non-neuronopathic MPS II.

Key Points: 
  • The global Phase 2/3 COMPASS study continues to recruit up to 54 participants with neuronopathic and non-neuronopathic MPS II.
  • Upon completion of the ongoing Phase 1/2 study, and together with data from COMPASS, this combined data package is intended to support registration.
  • Increased RIPK1 activity in the CNS is hypothesized to drive neuroinflammation and cell necroptosis and to contribute to neurodegeneration.
  • Denali will maintain ownership of and continue to advance its current portfolio of clinical stage small molecule programs.

Andelyn Biosciences Selected by Ultragenyx for Late-Stage Process Performance Qualification (PPQ) Manufacturing of Novel Gene Therapy for Sanfilippo Syndrome

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Monday, December 18, 2023
CNS, PPQ, Regenerative medicine, Brain, GMP, MPS, PRIME, CDMO, Sanfilippo syndrome, Regenerative Medicine Advanced Therapy, History, Patient, Research, SGSH, Cell damage, Process performance qualification protocol, EVP, Glycosaminoglycan, Partnership, Development, Orphan drug, Cell, AAV, Pharmaceutical industry, Ultragenyx

COLUMBUS, Ohio, Dec. 18, 2023 /PRNewswire/ -- Andelyn Biosciences , Inc., a pioneering and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), announced that it has been selected by Ultragenyx (NASDAQ: RARE) to perform late-stage Process Performance Qualification (PPQ) manufacturing of the company's UX111 gene therapy for the potential treatment of Sanfilippo Syndrome (MPS IIIA).

Key Points: 
  • COLUMBUS, Ohio, Dec. 18, 2023 /PRNewswire/ -- Andelyn Biosciences , Inc., a pioneering and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), announced that it has been selected by Ultragenyx (NASDAQ: RARE) to perform late-stage Process Performance Qualification (PPQ) manufacturing of the company's UX111 gene therapy for the potential treatment of Sanfilippo Syndrome (MPS IIIA).
  • Andelyn has successfully manufactured GMP supply of UX111 for patient dosing and will be performing the PPQ batches necessary for any future regulatory filings.
  • Andelyn is honored to continue its close partnership to advance the UX111 Program to bring treatment to MPS IIIA patients sooner."
  • With exceptional quality and scalable end-to-end development and manufacturing capabilities across its three Columbus, Ohio facilities, Andelyn is enabling the progression of life-altering gene therapies for rare and prevalent diseases.

Andelyn Biosciences Selected by Ultragenyx for Late-Stage Process Performance Qualification (PPQ) Manufacturing of Novel Gene Therapy for Sanfilippo Syndrome

Retrieved on: 
Monday, December 18, 2023
CNS, PPQ, Regenerative medicine, Brain, GMP, MPS, PRIME, CDMO, Sanfilippo syndrome, Regenerative Medicine Advanced Therapy, History, Patient, Research, SGSH, Cell damage, Process performance qualification protocol, EVP, Glycosaminoglycan, Partnership, Development, Orphan drug, Cell, AAV, Pharmaceutical industry, Ultragenyx

COLUMBUS, Ohio, Dec. 18, 2023 /PRNewswire/ -- Andelyn Biosciences , Inc., a pioneering and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), announced that it has been selected by Ultragenyx (NASDAQ: RARE) to perform late-stage Process Performance Qualification (PPQ) manufacturing of the company's UX111 gene therapy for the potential treatment of Sanfilippo Syndrome (MPS IIIA).

Key Points: 
  • COLUMBUS, Ohio, Dec. 18, 2023 /PRNewswire/ -- Andelyn Biosciences , Inc., a pioneering and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), announced that it has been selected by Ultragenyx (NASDAQ: RARE) to perform late-stage Process Performance Qualification (PPQ) manufacturing of the company's UX111 gene therapy for the potential treatment of Sanfilippo Syndrome (MPS IIIA).
  • Andelyn has successfully manufactured GMP supply of UX111 for patient dosing and will be performing the PPQ batches necessary for any future regulatory filings.
  • Andelyn is honored to continue its close partnership to advance the UX111 Program to bring treatment to MPS IIIA patients sooner."
  • With exceptional quality and scalable end-to-end development and manufacturing capabilities across its three Columbus, Ohio facilities, Andelyn is enabling the progression of life-altering gene therapies for rare and prevalent diseases.

Denali Therapeutics Reports Third Quarter 2023 Financial Results and Business Highlights

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Tuesday, November 7, 2023
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SOUTH SAN FRANCISCO, Calif., Nov. 07, 2023 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (Nasdaq: DNLI), a biopharmaceutical company developing a broad portfolio of product candidates engineered to cross the blood-brain barrier (BBB) for the treatment of neurodegenerative diseases and lysosomal storage diseases, today reported financial results for the third quarter ended September 30, 2023, and provided business highlights.

Key Points: 
  • SOUTH SAN FRANCISCO, Calif., Nov. 07, 2023 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (Nasdaq: DNLI), a biopharmaceutical company developing a broad portfolio of product candidates engineered to cross the blood-brain barrier (BBB) for the treatment of neurodegenerative diseases and lysosomal storage diseases, today reported financial results for the third quarter ended September 30, 2023, and provided business highlights.
  • Denali has selected five ASO targets for further development and is focused on advancing two OTV candidates towards clinical development.
  • 2023 Guidance on Operating Expenses:
    Cash, cash equivalents, and marketable securities were approximately $1.12 billion as of September 30, 2023.
  • General and administrative expenses were $25.3 million and $23.3 million for the three months ended September 30, 2023 and 2022, respectively.

QuidelOrtho Reports Third Quarter 2023 Financial Results

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Wednesday, November 1, 2023
Data Management, Medical Supplies, Health, Technology, Health Technology, General Health, Transfusion medicine, Sanfilippo syndrome, EBITDA, QuidelOrtho, EPS, GAAP, COVID-19, Patient, Virus, Pharmaceutical industry

QuidelOrtho Corporation (Nasdaq: QDEL) (the “Company” or “QuidelOrtho”), a global provider of innovative in vitro diagnostic technologies designed for point-of-care settings, clinical labs and transfusion medicine, today announced financial results for the third quarter ended October 1, 2023.

Key Points: 
  • QuidelOrtho Corporation (Nasdaq: QDEL) (the “Company” or “QuidelOrtho”), a global provider of innovative in vitro diagnostic technologies designed for point-of-care settings, clinical labs and transfusion medicine, today announced financial results for the third quarter ended October 1, 2023.
  • Foreign currency translation did not significantly impact third quarter 2023 results.
  • GAAP diluted loss per share for the third quarter of 2023 was $(0.19), compared to diluted EPS of $0.28 for the third quarter of 2022.
  • The Company is maintaining its 2023 financial guidance, provided on August 8, 2023, as follows:

Ultragenyx Announces Program and Pipeline Updates at Analyst Day Including Interim Data from Ongoing Studies in Osteogenesis Imperfecta (OI), Angelman Syndrome (AS) and Wilson Disease

Retrieved on: 
Monday, October 16, 2023
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NOVATO, Calif., Oct. 16, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today will provide updates on its development pipeline, including setrusumab (UX143) for osteogenesis imperfecta (OI), GTX-102 for Angelman syndrome (AS), UX701 in Wilson disease and the rest of the company’s gene therapy portfolio at an Analyst Day held in New York City and by webcast.

Key Points: 
  • Long term data showed patients who stopped and restarted treatment reacquired previously gained developmental skills when they were re-dosed with the current regimen.
  • There have been no additional treatment-related SAEs, including lower extremity weakness, since November 2022.
  • ET on Monday, October 16, 2023 to discuss these data and to provide an update on the company’s development pipeline.
  • An archived version of the remarks will also be available through the Ultragenyx website.