Rett syndrome

Anavex Life Sciences to Announce Fiscal 2023 Year End Financial Results on Monday, November 27, 2023

Retrieved on: 
Friday, November 24, 2023

NEW YORK, Nov. 24, 2023 (GLOBE NEWSWIRE) -- Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders including Alzheimer’s disease, Parkinson’s disease, Rett syndrome and other central nervous system (CNS) diseases, today announced that it will issue financial results for its fiscal year ended September 30, 2023, on Monday November 27, 2023.

Key Points: 
  • NEW YORK, Nov. 24, 2023 (GLOBE NEWSWIRE) -- Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders including Alzheimer’s disease, Parkinson’s disease, Rett syndrome and other central nervous system (CNS) diseases, today announced that it will issue financial results for its fiscal year ended September 30, 2023, on Monday November 27, 2023.
  • Management will host a conference call on Monday November 27, 2023, at 8:30 am ET to review financial results and provide an update on the execution of the Company’s growth strategy.
  • Following management’s remarks, there will be a question-and-answer session.
  • A replay of the conference call will also be available on Anavex’s website for up to 30 days.

Anavex Life Sciences Announces Grant of U.S. Patent Covering Blarcamesine (ANAVEX®2-73) for Treatment of Insomnia or Anxiety

Retrieved on: 
Wednesday, November 22, 2023

11,813,242 entitled “A2-73 AS A THERAPEUTIC FOR INSOMNIA, ANXIETY, AND AGITATION”.

Key Points: 
  • 11,813,242 entitled “A2-73 AS A THERAPEUTIC FOR INSOMNIA, ANXIETY, AND AGITATION”.
  • This patent expands Anavex’s existing patent coverage of ANAVEX®2-73 (blarcamesine), including U.S. Patent No.
  • 11,337,953 to cover Anavex’s leading drug candidate, ANAVEX®2-73 (blarcamesine), ANAVEX®1-41 and ANAVEX®19-144, for treating insomnia, anxiety or agitation.
  • Together with Anavex U.S. Patent No.

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

Retrieved on: 
Tuesday, December 19, 2023

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.

Trinity Capital Inc. Provides $40 Million Term Loan to Taysha Gene Therapies

Retrieved on: 
Tuesday, December 19, 2023

PHOENIX, Dec. 19, 2023 /PRNewswire/ -- Trinity Capital Inc. (NASDAQ: TRIN) ("Trinity"), a leading provider of diversified financial solutions to growth-stage companies, today announced the commitment of $40 million in term loans to Taysha Gene Therapies, Inc. (NASDAQ: TSHA) ("Taysha"), a clinical-stage gene therapy company, pursuant to a Loan and Security Agreement dated November 13, 2023, by and among Taysha, the lenders party thereto from time to time (the "Lenders"), and Trinity, as administrative agent and collateral agent for the Lenders.

Key Points: 
  • PHOENIX, Dec. 19, 2023 /PRNewswire/ -- Trinity Capital Inc. (NASDAQ: TRIN) ("Trinity"), a leading provider of diversified financial solutions to growth-stage companies, today announced the commitment of $40 million in term loans to Taysha Gene Therapies, Inc. (NASDAQ: TSHA) ("Taysha"), a clinical-stage gene therapy company, pursuant to a Loan and Security Agreement dated November 13, 2023, by and among Taysha, the lenders party thereto from time to time (the "Lenders"), and Trinity, as administrative agent and collateral agent for the Lenders.
  • Taysha is focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system ("CNS").
  • Its lead clinical program TSHA-102 is in evaluation for Rett syndrome, a rare neurodevelopmental disorder with no approved disease-modifying therapies that treat the root cause of the disease.
  • With the term loan, Taysha believes it will be able to fund its operating expenses and capital requirements into 2026 to support the clinical development of its TSHA-102 program in Rett syndrome.

Taysha Gene Therapies Reports Third Quarter 2023 Financial Results and Provides Corporate and Clinical Updates

Retrieved on: 
Tuesday, November 14, 2023

Following treatment, both patients experienced improvement in key clinical domains impacting activities of daily living, including breathing dysrhythmia, autonomic function, socialization, and gross and fine motor skills.

Key Points: 
  • Following treatment, both patients experienced improvement in key clinical domains impacting activities of daily living, including breathing dysrhythmia, autonomic function, socialization, and gross and fine motor skills.
  • Additional information on available clinical data is available in the Company’s quarterly report on Form 10-Q for the quarter ended September 30, 2023, to be filed with the SEC.
  • Cash and cash equivalents: As of September 30, 2023, the Company had cash and cash equivalents of $164.3 million.
  • Taysha management will hold a conference call and webcast today at 4:30 pm ET to review its financial and operating results and to provide corporate and clinical updates.

Neurogene Doses First Patients in Phase 1/2 Trial of NGN-401 for the Treatment of Female Pediatric Patients with Rett Syndrome

Retrieved on: 
Thursday, November 30, 2023

NGN-401 is an investigational adeno-associated virus (AAV) gene therapy candidate for Rett syndrome purposefully designed and administered to maximize the therapeutic activity while averting transgene overexpression toxicities.

Key Points: 
  • NGN-401 is an investigational adeno-associated virus (AAV) gene therapy candidate for Rett syndrome purposefully designed and administered to maximize the therapeutic activity while averting transgene overexpression toxicities.
  • NGN-401 delivers the full-length human methyl cytosine binding protein 2 (MECP2) gene, providing an optimal gene replacement approach.
  • In non-clinical studies with NGN-401 at clinically relevant doses, cardinal features of Rett syndrome were ameliorated, and no overexpression toxicity was observed.
  • Your resilience, courage, and support not only contribute to the progress of this research, but also inspire hope within the entire Rett syndrome community.

Rett Syndrome Research Trust Launches the Rett Syndrome Global Registry: A Pioneering Parent-Reported Platform Designed to Expedite the Development of Genetic Medicines

Retrieved on: 
Friday, November 10, 2023

TRUMBULL, Conn., Nov. 9, 2023 /PRNewswire-PRWeb/ -- The Rett Syndrome Research Trust (RSRT) is excited to announce the launch of the Rett Syndrome Global Registry, a fully-remote platform for parents to share their knowledge and experiences caring for loved ones with Rett syndrome. Inclusivity is a vital component of the Rett Global Registry. It is accessible to families regardless of geographic location, access to medical facilities, or socioeconomic status. Designed to advance the development of genetic medicines, the Rett Global Registry adheres to FDA guidance documents and complies with regulatory requirements. Biopharmaceutical companies pursuing therapeutic programs for Rett are urgently waiting to analyze data collected from the Rett Global Registry. Parents of children and adults with Rett syndrome are encouraged to register as soon as possible.

Key Points: 
  • Designed to advance the development of genetic medicines, the Rett Global Registry adheres to FDA guidance documents and complies with regulatory requirements.
  • Biopharmaceutical companies pursuing therapeutic programs for Rett are urgently waiting to analyze data collected from the Rett Global Registry.
  • TRUMBULL, Conn., Nov. 9, 2023 /PRNewswire-PRWeb/ -- The Rett Syndrome Research Trust (RSRT) is excited to announce the launch of the Rett Syndrome Global Registry, a fully-remote platform for parents to share their knowledge and experiences caring for loved ones with Rett syndrome.
  • Designed to advance the development of genetic medicines, the Rett Global Registry adheres to FDA guidance documents and complies with regulatory requirements.

Revolutionizing Care: NeuroQure’s Groundbreaking Path to Treating Genetic Intellectual Disabilities

Retrieved on: 
Monday, November 6, 2023

Despite this staggering number, there are currently no federally approved treatments targeting the genetic roots of these disorders.

Key Points: 
  • Despite this staggering number, there are currently no federally approved treatments targeting the genetic roots of these disorders.
  • The goal of developing gene therapy to treat intellectual disabilities fuels both Justus and Dr. Gargus.
  • We envision a future where intellectual disabilities are not barriers but challenges we can overcome,” said Justus.
  • “NeuroQure’s mission is not merely scientific advancement; it’s a beacon of hope for individuals and families affected by these conditions.”

Anavex Life Sciences Appoints Senior VP of Regulatory Affairs

Retrieved on: 
Monday, November 6, 2023

Mr. Goldberger will succeed retiring Senior Vice President Regulatory Affairs, Emmanuel O Fadiran, RPh, MS, PhD.

Key Points: 
  • Mr. Goldberger will succeed retiring Senior Vice President Regulatory Affairs, Emmanuel O Fadiran, RPh, MS, PhD.
  • “We are delighted to welcome Mr. Goldberger to the Anavex team,” said Christopher U Missling, PhD, President and Chief Executive Officer of Anavex.
  • Prior to Otsuka he held senior level positions in Global Regulatory Affairs for Johnson and Johnson (J&J) Pharmaceutical Research and Development in Global Regulatory Affairs.
  • In addition, he led CMC Regulatory Affairs, Labeling, Regulatory Operations/Technology and Medical Writing areas.

Acadia Pharmaceuticals Reports Third Quarter 2023 Financial Results and Operating Overview

Retrieved on: 
Thursday, November 2, 2023

Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced its financial results for the third quarter ended September 30, 2023.

Key Points: 
  • Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced its financial results for the third quarter ended September 30, 2023.
  • The Company plans to initiate a Phase 3 placebo-controlled study of ACP-101 for the treatment of hyperphagia in Prader-Willi syndrome in the fourth quarter of 2023.
  • The Company plans to initiate a Phase 2 study of ACP-204 as a potential treatment for Alzheimer’s disease psychosis in the fourth quarter of 2023.
  • Net product sales of DAYBUE were $66.9 million for the quarter ended September 30, 2023, the first full quarter of commercialization of DAYBUE following the April 17, 2023 launch.