Batten disease

Alcyone Therapeutics Announces Appointment of Dr. Norbert Riedel as Chairman of the Board and Dr. Kathrin Meyer as Chief Scientific Officer and Head of Research & Development

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Mercoledì, Maggio 8, 2024
Doctor of Philosophy, University, MicroRNA, Gene therapy, Hospital, Central nervous system disease, Rett syndrome, Innovation, Jazz Pharmaceuticals, Research, Ohio State University, History, CNS, Baxter International, Therapy, Batten disease, Patient, Acquisition, Gene expression, Nationwide Children's Hospital, Central nervous system, Health, SMARD1, Growth, Medical device

LOWELL, Mass., May 8, 2024 /PRNewswire/ -- Alcyone Therapeutics, a clinical-stage biotechnology company pioneering next-generation precision genetic therapies for complex neurological conditions, today announces appointments of Norbert Riedel, Ph.D., a skilled and experienced biopharmaceutical executive, as the Chairman of its Board of Directors, and Kathrin Meyer, Ph.D., a leader in development and clinical translation of genetic therapies for the central nervous system (CNS), as Alcyone's new Chief Scientific Officer and Head of R&D.

Key Points: 
  • "I am delighted and honored to join the board of Alcyone Therapeutics," said Dr. Riedel.
  • I am very excited about the programs we have in development to address the significant unmet patient needs in several neurological indications."
  • "Developing neurological genetic therapies demands both a highly precise biological mechanism of action and effective delivery to the central nervous system.
  • Dr. Kathrin Meyer is a pioneer in the research and clinical development of novel therapies for the CNS.

Neurogene Reports Fourth Quarter and Full Year 2023 Financial Results and Highlights Recent Updates

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Lunedì, Marzo 18, 2024
Biotechnology, Neurology, Health, Pharmaceutical, Clinical Trials, Toxicity, Administration, Information technology, MECP2, Inc., Patient, Cash, Batten disease, Cohort, Female, Insurance, Rett syndrome, Research and development, Investment, Clinical trial, Net, Data, DSM-IV codes, Pharmaceutical industry

Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced fourth quarter and full year 2023 financial results and highlighted recent corporate updates.

Key Points: 
  • Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced fourth quarter and full year 2023 financial results and highlighted recent corporate updates.
  • We believe this expanded dataset will support future regulatory discussions to align on the design of a potential registrational study,” said Rachel McMinn, Ph.D., Chief Executive Officer and Founder of Neurogene.
  • “NGN-401 has the potential to be a best-in-class treatment option with a targeted route of administration to deliver full-length, functional copies of the MECP2 gene.
  • Net income for the three months ended December 31, 2023 included a one-time $16.4 million bargain purchase gain related to the reverse merger.

Neurogene Announces Business Update and 2024 Outlook

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Venerdì, Gennaio 5, 2024
Science, Neurology, Biotechnology, Research, Pharmaceutical, Health, Genetics, Clinical Trials, DSM-IV codes, Health care, Rett syndrome, Data monitoring committee, FDA, Female, Medication, Brain, MHRA, Investigational New Drug, Natural history, Cohort, GMP, University, Toxicity, Patient, Safety, Batten disease, CTA, Food and Drug Administration, Inc., Pharmaceutical industry

Neurogene Inc. (NASDAQ: NGNE) (“Neurogene” or “the Company”), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, announced today a business update and provided its outlook for 2024.

Key Points: 
  • Neurogene Inc. (NASDAQ: NGNE) (“Neurogene” or “the Company”), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, announced today a business update and provided its outlook for 2024.
  • Key milestones for the Company’s clinical-stage gene therapy product candidates remain on track for 2024, while the Company continues to advance its discovery-stage transgene regulation portfolio.
  • In the fourth quarter of 2023, Neurogene completed a positive meeting with the FDA regarding the future potency assay.
  • Neurogene plans to advance one product from its discovery-stage pipeline into the clinic in 2025.

Neurogene Announces Closing of Merger with Neoleukin Therapeutics and Concurrent Private Placement of $95 Million

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Martedì, Dicembre 19, 2023
Oncology, Health, Genetics, Clinical Trials, Pharmaceutical, Biotechnology, Janus, DSM-IV codes, Food, Cooley LLP, Rett syndrome, FDA, Stifel, Trial of the century, Orphan, SVB Securities, Investment, MECP2, Toxicity, Patient, CLN5, Batten disease, Exercise, Pharmaceutical industry

Neurogene Inc. (NASDAQ: NGNE) (“Neurogene”), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced the closing of its merger with Neoleukin Therapeutics, Inc. (“Neoleukin”).

Key Points: 
  • Neurogene Inc. (NASDAQ: NGNE) (“Neurogene”), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced the closing of its merger with Neoleukin Therapeutics, Inc. (“Neoleukin”).
  • Neurogene shares are expected to begin trading on the NASDAQ Global Market under the ticker “NGNE” beginning today at the market open.
  • In the reverse stock split, every four shares of Neoleukin common stock outstanding were combined and reclassified into one share of Neoleukin common stock.
  • Gibson Dunn & Crutcher LLP served as legal counsel to Neurogene and Cooley LLP served as legal counsel to the placement agents.

Initial Clinical Data of First Pediatric CLN2 Patient Dosed with RGX-181 Presented at SSIEM Annual Symposium

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Mercoledì, Agosto 30, 2023
Seizure, Society, Therapy, TPP1, Neurodegenerative disease, Kia Challenge, Gene expression, Accelerated approval (FDA), Language, AAV, Hunter syndrome, Batten disease, Death, ERT, Patient, Child, Hospital, Enzyme replacement therapy, Safety, CLN2, BLA, Report of the 1st Annual Symposium on Relaxed Improvisation, Peptide, Disease, Pharmaceutical industry, Metabolism

"CLN2 is a debilitating disease caused by mutations in the CLN2 gene resulting in a deficiency of the TPP1 enzyme, which is needed to break down specific peptides associated with cellular waste.

Key Points: 
  • "CLN2 is a debilitating disease caused by mutations in the CLN2 gene resulting in a deficiency of the TPP1 enzyme, which is needed to break down specific peptides associated with cellular waste.
  • "We are encouraged by the initial results demonstrating that RGX-181 is well tolerated and dramatically reduced the number of seizures in the patient enrolled in this trial."
  • "The remarkable decrease in seizures, encouraging safety results and reduction in ERT frequency highlight the potential of this gene therapy to provide a meaningful treatment option to the CLN2 patient community."
  • Today, a physician investigator from the Hospital de Clinicas in Porto Alegre, Brazil reported initial results from a five-year-old child who received a one-time intracisternal dose of RGX-181.

Neurogene and Neoleukin Announce Definitive Merger Agreement

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Martedì, Luglio 18, 2023
Janus, Interim, General counsel, Investment, Rett syndrome, DSM-IV codes, Batten disease, Patient, Pharmaceutical industry

Upon completion of the merger, which is subject to approval by Neurogene and Neoleukin stockholders, the combined company is expected to operate under the name Neurogene Inc. and trade on the Nasdaq Capital Market under the ticker symbol “NGNE”.

Key Points: 
  • Upon completion of the merger, which is subject to approval by Neurogene and Neoleukin stockholders, the combined company is expected to operate under the name Neurogene Inc. and trade on the Nasdaq Capital Market under the ticker symbol “NGNE”.
  • “We are excited to announce our planned merger with Neoleukin, which we believe is a transformative step forward in our mission to bring life-changing genetic medicines to the patients and families impacted by devastating neurological diseases,” said Rachel McMinn, Ph.D., Founder and Chief Executive Officer of Neurogene.
  • This capital will also support our internal manufacturing capabilities, which we expect will continue to provide significant financial and strategic flexibility.
  • We are grateful to our current and former employees who contributed to Neoleukin’s efforts and look forward to the combined company’s continued progress and success.”

LEXEO Therapeutics to Present Clinical and Preclinical Data at Upcoming Scientific Conferences

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Giovedì, Ottobre 27, 2022
University, CNS, Drug development, LinkedIn, Interim, FA, ICAR, Patient, Clinical trial, Weill Cornell Medicine, Public, Trial of the century, Peripheral nervous system, Batten disease, International Congress on Tuberculosis, Degenerative disease, GLOBE, University of California, San Diego Performance-Based Skills Assessment, Safety, Therapy, AAV, American Heart Association, Central nervous system, Pharmaceutical industry, Online shopping

NEW YORK, Oct. 27, 2022 (GLOBE NEWSWIRE) -- LEXEO Therapeutics (LEXEO), a clinical-stage biotech company advancing a pipeline of adeno-associated virus (AAV)-based gene therapy candidates for cardiovascular and central nervous system (CNS) diseases, today announced it will be presenting clinical and preclinical data at three upcoming scientific meetings. LEXEO will present at the inaugural International Congress for Ataxia Research (ICAR) held live in Dallas, Texas from November 1-4, 2022, the American Heart Association Scientific Sessions held live in Chicago, Illinois and virtually from November 5-7, 2022, and the Clinical Trials for Alzheimer’s Disease (CTAD) conference held live in San Francisco, California from November 29-December 2, 2022.

Key Points: 
  • NEW YORK, Oct. 27, 2022 (GLOBE NEWSWIRE) -- LEXEO Therapeutics (LEXEO), a clinical-stage biotech company advancing a pipeline of adeno-associated virus (AAV)-based gene therapy candidates for cardiovascular and central nervous system (CNS) diseases, today announced it will be presenting clinical and preclinical data at three upcoming scientific meetings.
  • To view full event programming, please visit the AHA Scientific Sessions website .
  • LEXEO Therapeutics is a New York City-based, clinical-stage gene therapy company focused on addressing some of the most devastating genetically defined cardiovascular and central nervous system diseases affecting both larger-rare and prevalent patient populations.
  • LEXEOs foundational science stems from partnerships and exclusive licenses with leading academic laboratories at Weill Cornell Medicine and the University of California, San Diego.

LEXEO Therapeutics Receives Orphan Drug Designation for LX1004 from European Commission

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Martedì, Ottobre 18, 2022
LinkedIn, University of California, San Diego Performance-Based Skills Assessment, CLN2, Therapy, Royal commission, Weill Cornell Medicine, Patient, Drug development, Government budget balance, Seizure, Severe cognitive impairment, University, NEW, Mutation, AAV, Peripheral nervous system, CNS, GLOBE, Degenerative disease, Death, TPP1, FDA, Batten disease, Central nervous system, European Commission, Pharmaceutical industry

NEW YORK, Oct. 18, 2022 (GLOBE NEWSWIRE) -- LEXEO Therapeutics (LEXEO), a clinical-stage biotech company advancing a pipeline of adeno-associated virus (AAV)-based gene therapy candidates for genetically defined cardiovascular and central nervous system (CNS) diseases, today announced that the European Commission has granted Orphan Drug Designation to LX1004 for the treatment of CLN2 Batten disease. LX1004 is an AAV-mediated gene therapy designed to deliver a fully functional CLN2 gene to the CNS via intracisternal injection to restore TPP1, the secreted protein that is deficient in patients with CLN2 Batten disease.

Key Points: 
  • LX1004 is an AAV-mediated gene therapy designed to deliver a fully functional CLN2 gene to the CNS via intracisternal injection to restore TPP1, the secreted protein that is deficient in patients with CLN2 Batten disease.
  • An End of Phase 1/2 Meeting with the FDA will be held by the end of 2022.
  • LEXEO has previously been granted Rare Pediatric Disease and Orphan Drug designations by the FDA.
  • LEXEO Therapeutics is a New York City-based, clinical-stage gene therapy company focused on addressing some of the most devastating genetically defined cardiovascular and central nervous system diseases affecting both larger-rare and prevalent patient populations.

LEXEO Therapeutics to Present New Clinical Data from its Investigational Gene Therapy LX1001 for APOE4-Associated Alzheimer’s Disease at the 29th European Society of Gene & Cell Therapy Annual Meeting

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Mercoledì, Ottobre 5, 2022
LinkedIn, University of California, San Diego Performance-Based Skills Assessment, ESGCT, European Society of Gene and Cell Therapy, Therapy, Weill Cornell Medicine, Cell, Patient, Drug development, University, APOE2, AAV, Peripheral nervous system, APOE4, CNS, GLOBE, Degenerative disease, Batten disease, Central nervous system, Pharmaceutical industry

In an oral presentation, LEXEO will present clinical data from the ongoing study of LX1001 for APOE4-associated Alzheimers disease.

Key Points: 
  • In an oral presentation, LEXEO will present clinical data from the ongoing study of LX1001 for APOE4-associated Alzheimers disease.
  • LX1001 is an AAV-based investigational gene therapy designed to deliver the apolipoprotein E2 (APOE2) gene into the CNS of APOE4 homozygous Alzheimers disease patients to halt or slow disease progression.
  • This marks the first clinical data from a gene therapy program targeting APOE4-associated Alzheimers disease presented at a scientific meeting.
  • LEXEOs foundational science stems from partnerships and exclusive licenses with leading academic institutions at Weill Cornell Medicine and the University of California, San Diego.

HaemaLogiX strengthens Board with appointment of Dr. Geoff Nichol

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Giovedì, Luglio 7, 2022
Patient, Carmine, Achondroplasia, CEO, Medarex, B cell, Novartis, Pulmonology, Physiology, BMS, Amoxicillin/clavulanic acid, Bay, BioMarin Pharmaceutical, Nobel Prize, Senior advisor, Formoterol, SA, BMN, Nobel Prize in Physiology or Medicine, CMO, MBA, Non-executive director, GlaxoSmithKline, SVP, Bristol Myers Squibb, Haemophilia, EVP, Physician, Royal Australasian College, Royal college, Sangamo Therapeutics, VP, Board, Multiple myeloma, Drug development, Batten disease, Lymphatic system, Pharmaceutical industry, Pegvaliase, Medicine, Omalizumab, Phenylketonuria

Bryce Carmine, HaemaLogiX's Chairman and CEO commented: "Geoff's impressive, globally recognised background in drug development, and in particular, in antibody development, makes him a valuable contribution to the HaemaLogiX board.

Key Points: 
  • Bryce Carmine, HaemaLogiX's Chairman and CEO commented: "Geoff's impressive, globally recognised background in drug development, and in particular, in antibody development, makes him a valuable contribution to the HaemaLogiX board.
  • Importantly, during his time at Medarex, Geoff oversaw the preclinical and clinical development of KappaMab, our multiple myeloma drug candidate.
  • He brings outstanding industry knowledge and a highly translatable skillset to our Board mix.
  • Dr. Nichol brings nearly 30 years' experience in drug development.