Glycogen storage disease

Tim Scott to Succeed Joe Panetta as Biocom California President and CEO

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화요일, 5월 28, 2024
Medical Devices, Health, Research, Pharmaceutical, Science, Biotechnology, Patient, UC, Organization, STEM, Biochemistry, Entrepreneurship, Education, Workforce, University, Therapy, Ecosystem, Incubator, Growth, Human, JD, Drug discovery, Catalent, Pharma, Glycogen storage disease, Multimedia, CDMO, Management

Biocom California, the association representing the California life science industry, today announced that it has named Tim Scott as its president and CEO, effective January 1, 2025.

Key Points: 
  • Biocom California, the association representing the California life science industry, today announced that it has named Tim Scott as its president and CEO, effective January 1, 2025.
  • Mr. Scott will succeed Joe Panetta, Biocom California’s current president and CEO, who has led the organization for 25 years.
  • Chair-elect Sabrina Martucci Johnson, president and CEO of Daré Bioscience, will succeed Mr. Scott as board chair.
  • View the full release here: https://www.businesswire.com/news/home/20240528207819/en/
    Tim Scott, Chairman, Biocom California Board of Directors (Photo: Business Wire)
    Mr. Panetta, who joined Biocom in 1999, has been instrumental in spearheading initiatives that have significantly advanced the biopharma and medical device landscape in California.

Freeline Presents Positive New Data from Phase 1/2 Trial of FLT201, Its Novel Gene Therapy Candidate for Gaucher Disease, in Late-Breaking Oral Presentation at ASGCT 27th Annual Meeting

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목요일, 5월 9, 2024
FDA, Bone marrow, Mutation, Cell, Risk, Blood, News, Research, ERT, White blood cell, Glycogen storage disease, SRT, ALT, GBA1, White, European Medicines Agency, Brain, Enzyme replacement therapy, Regenerative Medicine Advanced Therapy, Therapy, Plasma, Patient, Mouse, Safety, Tissue, Glucocerebrosidase, Substantia nigra, Lysosome, Neuropsychological test, AAV, Dementia, PRIME, Fatigue, EMA, Drug development, Bone pain, Gaucher's disease, FACIT, Disease, Liver, Gene, Society, Standard of care, Annual general meeting, Pharmaceutical industry

LONDON, May 09, 2024 (GLOBE NEWSWIRE) --  Freeline Therapeutics today announced new clinical data from its ongoing Phase 1/2 GALILEO-1 trial of FLT201, its adeno-associated virus (AAV) gene therapy candidate for Gaucher disease, showing substantial reductions in glucosylsphinogsine (lyso-Gb1), one of the best predictors of clinical response, in patients with persistently high levels despite years of treatment with currently approved therapies, as well as early signs of clinical improvements in bone marrow burden and fatigue. FLT201 continues to demonstrate a favorable safety and tolerability profile. These data are being showcased in a late-breaking oral presentation at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting taking place in Baltimore, Maryland.

Key Points: 
  • These data are being showcased in a late-breaking oral presentation at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting taking place in Baltimore, Maryland.
  • Reductions in lyso-Gb1 levels in the blood are highly correlated with substrate reduction in disease-affected tissues and positive clinical outcomes in Gaucher disease.
  • Currently approved treatments have made a significant difference for people with Gaucher disease, but there is not an existing cure.
  • Both the late-breaking oral presentation and the poster presentation are now available on the News & Events section of Freeline’s website.

Denali Therapeutics Reports First Quarter 2024 Financial Results and Business Highlights

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화요일, 5월 7, 2024
FDA, Amyloid beta, Part, Total, PIPE, CDER, Transferrin receptor, Nerve, BBB, CSF, Biomarker, Gamma, Enzyme replacement therapy, Traffic barrier, Hunting, OTV, PTV, Food, MGH, IDS, Conference, MAPT, ALS, Therapy, Cerebrospinal fluid, SAN, MPS II, Hospital, Safety, AFL, SGSH, UC, Alpha-synuclein, LUMA, SNCA, Clinical trial, Glycogen storage disease, Neurodegenerative disease, MPS, COMPASS, Patient, Alfa, Acquisition, ETV, ATV, Hunter syndrome, Sanofi, Massachusetts General Hospital, Pharmaceutical industry

SOUTH SAN FRANCISCO, Calif., May 07, 2024 (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 first quarter ended March 31, 2024, and provided business highlights.

Key Points: 
  • SOUTH SAN FRANCISCO, Calif., May 07, 2024 (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 first quarter ended March 31, 2024, and provided business highlights.
  • There was no collaboration revenue for the quarter ended March 31, 2024, compared to $35.1 million for the quarter ended March 31, 2023.
  • Total research and development expenses were $107.0 million for the quarter ended March 31, 2024, compared to $128.8 million for the quarter ended March 31, 2023.
  • General and administrative expenses were $25.2 million for the quarter ended March 31, 2024, compared to $27.1 million for the quarter ended March 31, 2023.

Orphan designation: Adeno-associated viral vector serotype 9 encoding a codon-optimised human aspartylglucosaminidase transgene Treatment of aspartylglucosaminuria, 19/10/2020 Withdrawn

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화요일, 4월 9, 2024
Eurostat, Diagnosis, Civil service commission, Epilepsy, Aspartylglucosaminuria, Speech, Cell, Disease, Patient, DSM-IV codes, Committee, Knowledge, Tissue, Glycoprotein, Regulation, EMA, IRIS, Glycogen storage disease, Life, Human, Treatment, European, Infection, COMP, Sponsor, European Commission, Spine, Safety, Family, Marketing, Movement, Cell damage, Medicine, Pharmaceutical industry

Orphan designation: Adeno-associated viral vector serotype 9 encoding a codon-optimised human aspartylglucosaminidase transgene Treatment of aspartylglucosaminuria, 19/10/2020 Withdrawn

Key Points: 


Orphan designation: Adeno-associated viral vector serotype 9 encoding a codon-optimised human aspartylglucosaminidase transgene Treatment of aspartylglucosaminuria, 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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화요일, 3월 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.

Natalia Gomez-Ospina, MD, PhD is the Recipient of the 2024 Dr. Michael S. Watson Genetic and Genomic Medicine Innovation Award from the ACMG Foundation for Genetic and Genomic Medicine

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수요일, 3월 13, 2024
Research, Partner, Brain, B cell, FACMG, Gaucher's disease, Assistant, Pediatric Research, American Journal, Patient, Medical genetics, DSM-IV codes, Glycogen storage disease, System, Young Scientist Award, Gene therapy, Doctor of Philosophy, Stanford University, Protein, Genome, American College, Mucopolysaccharidosis, Genomics, Society, Hospital, Genetic, Cell, Young, Genetic distance, Hurler syndrome, Lucile Packard Children's Hospital

BETHESDA, Md., March 13, 2024 /PRNewswire/ -- Natalia Gomez-Ospina, MD, PhD is the recipient of the ACMG Foundation for Genetic and Genomic Medicine's 2024 Dr. Michael S. Watson Genetic and Genomic Medicine Innovation Award—the "Watson Award"—named for the American College of Medical Genetics and Genomics first and longstanding executive director, Michael S. Watson, MS, PhD, FACMG.

Key Points: 
  • BETHESDA, Md., March 13, 2024 /PRNewswire/ -- Natalia Gomez-Ospina, MD, PhD is the recipient of the ACMG Foundation for Genetic and Genomic Medicine's 2024 Dr. Michael S. Watson Genetic and Genomic Medicine Innovation Award—the "Watson Award"—named for the American College of Medical Genetics and Genomics first and longstanding executive director, Michael S. Watson, MS, PhD, FACMG.
  • "I am honored to receive this award in recognition of my dedication to advancing genetic and genomic medicine and my commitment to making a positive difference in the lives of patients with genetic diseases.
  • This award affirms the need for innovative approaches to treat such diseases and the immense potential that genome editing has in this regard.
  • "Dr. Gomez-Espina is a promising physician-scientist whose work is befitting the Dr. Michael Watson Genetic and Genomic Medicine Innovation Award, an award that honors an individual whose work has had a significant impact on genetic and genomic medicine.

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

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화요일, 2월 27, 2024
MPS II, Hearing, ARIA, Antibody, LRRK2, PTV, IDS, Transferrin receptor, MAPT, Traffic barrier, ALS, Hunting, Dermatan sulfate, Therapy, BBB, SAN, Total, Amyloid-related imaging abnormalities, Alpha-synuclein, COMPASS, ETV, Hunter syndrome, Neurodegenerative disease, Conference, MPS, Safety, Biomarker, OTV, LUMA, AFL, SNCA, Amyloid beta, CSF, PIPE, Glycogen storage disease, Research, ATV, FDA, Food, Risk, Sanofi, UC, CD98, Patient, Cognition, Oligonucleotide, Science, Alfa, Pharmaceutical industry

Enrollment continues in the global Phase 2/3 COMPASS study and is expected to be completed in 2024.

Key Points: 
  • Enrollment continues in the global Phase 2/3 COMPASS study and is expected to be completed in 2024.
  • Sanofi intends to present the detailed efficacy and safety results of the ALS Phase 2 HIMALAYA study at a future scientific forum.
  • There was no collaboration revenue for the quarter ended December 31, 2023, compared to $10.3 million for the quarter ended December 31, 2022.
  • Further, for the quarter ended December 31, 2023, there was also a decrease in other unallocated research and development expenses as a result of reduced facility costs.

Denali Therapeutics Announces $500 million Private Placement Equity Financing

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화요일, 2월 27, 2024
BBB, SAN, Traffic barrier, Prospectus, Investment, Sale, Exercise, Therapy, Acceleration, PIPE, Glycogen storage disease, Research, Security (finance)

Denali anticipates the gross proceeds from the PIPE to be approximately $500 million.

Key Points: 
  • Denali anticipates the gross proceeds from the PIPE to be approximately $500 million.
  • The financing is expected to close on February 29, 2024, subject to customary closing conditions.
  • The financing was led by a U.S.-based healthcare-focused investor, with participation from global asset managers based in Boston and on the West Coast.
  • This cash estimate is a preliminary estimate and based on information currently available to management, and these estimates could change.

JCR Pharmaceuticals’ Research Presentations at WORLDSymposium™ 2024 Showcase JR-141 (Pabinafusp Alfa) and Other Investigational Treatments for Lysosomal Storage Disorders

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수요일, 2월 14, 2024
Science, Other Science, Biotechnology, Research, Pharmaceutical, General Health, Health, Clinical Trials, Enzyme replacement therapy, BBB, JCR, MPS II, Traffic barrier, Glycogen storage disease, Ministry, MHLW, ERT, Hunter syndrome, Syndrome, Scheie syndrome, MPS, Safety, Developmental psychology, Hurler syndrome, Child, Patient, Hurler–Scheie syndrome, Trial of the century, Consciousness, DSM-IV codes, Hypotension, Mouse, Pharmaceutical industry

JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) today announced the presentation of several datasets demonstrating the potential benefits of its investigational therapies for lysosomal storage disorders (LSDs).

Key Points: 
  • JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) today announced the presentation of several datasets demonstrating the potential benefits of its investigational therapies for lysosomal storage disorders (LSDs).
  • JCR is dedicated to address the unmet medical needs for this community,” said Shin Ashida, President and CEO of JCR Pharmaceuticals.
  • Finally, the third JR-141 presentation is pre-clinical and highlights the recovery of retinal function in mice with MPS II.
  • “We are pleased to be able to offer IZCARGO® to patients with MPS II in Japan,” said Yoshikatsu Eto, M.D., Ph.D., Institute of Neurological Disorders, Advanced Clinical Research Center, Kanagawa, Japan.

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

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수요일, 2월 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.