Rett

Rett Syndrome Research Trust's Genetic Medicines Summit Sets the Stage for Accelerated Therapeutic Development

Retrieved on: 
Wednesday, October 11, 2023
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TRUMBULL, Conn., Oct. 11, 2023 /PRNewswire-PRWeb/ -- The Rett Syndrome Research Trust (RSRT) is delighted to announce the successful conclusion of the inaugural Rett Syndrome Genetic Medicines Summit, held September 13 – 15 in Boston, Massachusetts. This landmark event gathered distinguished scientists, clinicians, regulatory experts, and over 40 leading biopharma companies. The goal of the Summit was to foster dialogue among scientific, clinical, industry, and regulatory stakeholders to facilitate collaboration, identify best practices, and refine expectations to expedite pre-clinical and clinical development programs focused on genetic medicines for children and adults with Rett syndrome.

Key Points: 
  • The Rett Syndrome Research Trust (RSRT) is delighted to announce the successful conclusion of the inaugural Rett Syndrome Genetic Medicines Summit, held September 13 – 15 in Boston, Massachusetts.
  • TRUMBULL, Conn., Oct. 11, 2023 /PRNewswire-PRWeb/ -- The Rett Syndrome Research Trust (RSRT) is delighted to announce the successful conclusion of the inaugural Rett Syndrome Genetic Medicines Summit, held September 13 – 15 in Boston, Massachusetts.
  • RSRT's Rett Syndrome Genetic Medicines Summit underscores the organization's unwavering commitment to curing Rett syndrome.
  • RSRT remains steadfast in its mission to accelerate the development of genetic therapies and ultimately find a cure for Rett syndrome.

Taysha Gene Therapies Announces Second Patient Dosed with TSHA-102 in the REVEAL Phase 1/2 Adult Trial for the Treatment of Rett Syndrome

Retrieved on: 
Tuesday, September 26, 2023
Rett, Index, FRCP, Central nervous system, Pediatrics, Health care, IDMC, MECP2, Rett syndrome, Neuroscience, AAV, CNS, Associate, CHU, FAAP, Clinical trial, Investigational New Drug, Patient, Child, MAD, MTD, Principal, FDA, Safety, IND, MHRA, Pharmaceutical industry

DALLAS, Sept. 26, 2023 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS), today announced that the second Rett syndrome patient has been dosed with TSHA-102 in the REVEAL Phase 1/2 adult trial in Canada.

Key Points: 
  • DALLAS, Sept. 26, 2023 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS), today announced that the second Rett syndrome patient has been dosed with TSHA-102 in the REVEAL Phase 1/2 adult trial in Canada.
  • “Dosing the second adult patient in the REVEAL Phase 1/2 adult trial in Canada marks important progress in the ongoing clinical evaluation of TSHA-102 for Rett syndrome,” said Sukumar Nagendran, M.D., President, and Head of R&D of Taysha.
  • The maximum tolerated dose (MTD) or maximum administered dose (MAD) established will then be administered during dose expansion.
  • Enrollment in the low-dose cohort is expected to be complete in the fourth quarter of 2023 with the dosing of the third patient.

Acadia Pharmaceuticals Reports Second Quarter 2023 Financial Results and Operating Overview

Retrieved on: 
Wednesday, August 2, 2023
Science, Biotechnology, Research, Pharmaceutical, Health, Mental Health, Clinical Trials, Other Health, Polyphagia, Prader–Willi syndrome, Senior, Schizophrenia, ACAD, Clinical trial, Rett syndrome, Ariad Pharmaceuticals, Inc. v. Eli Lilly & Co., PIN, Patient, Investment, Pivotal, PWS, FDA, Therapy, Safety, Lotus, Cryptocurrency, Pharmaceutical industry, Nursing, Clothing, Rett, Nature Medicine

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

Key Points: 
  • Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced its financial results for the second quarter ended June 30, 2023.
  • “Our second quarter 2023 results reflect strong performances from both commercial franchises.
  • The Company recently aligned on plans with the FDA to initiate a Phase 3 study in the fourth quarter of 2023.
  • Acadia met with the FDA and aligned on dosing and plans to initiate a Phase 2/3 program in the fourth quarter of 2023.

Long-term Clinical Study Demonstrates Disease Modifying Effects of ANAVEX®2-73 (blarcamesine) for Rett Syndrome

Retrieved on: 
Wednesday, June 28, 2023
Central nervous system, Reduction, Therapy, Classification, Clinical trial, SIGMAR1, Rett syndrome, Fast Track, Doctor of Philosophy, CNS, Trial of the century, Orphan drug, DSM-IV codes, HIV disease progression rates, Patient, FDA, Safety, Syndrome, OLE, Disease, Pharmaceutical industry, Rett

The effect of ANAVEX®2-73 (blarcamesine) in the double-blind part of the U.S. ANAVEX®2-73-RS-001 study was maintained in the open label 12-week extension study.

Key Points: 
  • The effect of ANAVEX®2-73 (blarcamesine) in the double-blind part of the U.S. ANAVEX®2-73-RS-001 study was maintained in the open label 12-week extension study.
  • Meaning that ANAVEX®2-73 (blarcamesine) exhibited both symptomatic and disease modifying effects in the treatment of Rett syndrome in a clinical setting.
  • Additionally, disease progression, which is defined as change in Rett syndrome disease severity with time, was also reduced with long-term treatment with ANAVEX®2-73 (blarcamesine).
  • We are looking forward to the upcoming read-out of the ANAVEX®2-73 (blarcamesine) EXCELLENCE Phase 2/3 Rett syndrome clinical trial in pediatric patients with Rett syndrome.”

Anavex Life Sciences Reports Publication in Medical Journal Signifying Continued Commitment for Improvement of the Rett Syndrome Caregiver Outcome Measure RSBQ

Retrieved on: 
Monday, June 12, 2023
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The study was partially funded by a grant from the International Rett Syndrome Foundation (IRSF).

Key Points: 
  • The study was partially funded by a grant from the International Rett Syndrome Foundation (IRSF).
  • The RSBQ was developed by a British team of clinicians and researchers to better delineate behavioral problems in children with Rett syndrome.
  • Indeed, most regulated trials include as co-primary efficacy endpoints the RSBQ, as a caregiver measure, and the Clinical Global Impression of Improvement (CGI-I) as an investigator assessment.
  • In the EXCELLENCE Phase 2/3 ANAVEX®2-73-RS-003 Rett syndrome pediatric clinical trial, the characterized Rett Syndrome Behaviour Questionnaire (RBSQ), together with the Clinical Global Impression Improvement Scale (CGI-I), represent the co-primary efficacy endpoints of the study.

Anavex Life Sciences Announces Completion of ANAVEX(®)2-73 (blarcamesine) EXCELLENCE Phase 2/3 Rett Syndrome Clinical Trial

Retrieved on: 
Tuesday, June 6, 2023
Communication, Central nervous system, Therapy, SIGMAR1, Rett syndrome, Fast Track, Doctor of Philosophy, CNS, Trial of the century, AVATAR, Orphan drug, DSM-IV codes, Patient, FDA, Safety, Syndrome, Pharmaceutical industry, Rett

The Company expects to announce topline results from this study in the second half of this year.

Key Points: 
  • The Company expects to announce topline results from this study in the second half of this year.
  • The multi-center, double-blind clinical EXCELLENCE study (ANAVEX®2-73-RS-003)4 in pediatric patients is measuring safety, tolerability, and efficacy of daily oral ANAVEX®2-73 (blarcamesine) doses or placebo.
  • In communication with the FDA, the Company received the Agency’s input on the study endpoints, which were utilized in this clinical study.
  • ANAVEX®2-73 (blarcamesine) had previously received Fast Track designation, Rare Pediatric Disease designation and Orphan Drug designation from the FDA for the treatment of Rett syndrome.

Taysha Gene Therapies Announces First Patient Dosed with TSHA-102 in the REVEAL Phase 1/2 Trial Under Investigation for the Treatment of Rett Syndrome

Retrieved on: 
Monday, June 5, 2023
Communication, Index, FRCP, Central nervous system, Pediatrics, VG, Clinical trial, MECP2, Rett syndrome, Neuroscience, Gene expression, AAV, CNS, Associate, CHU, FAAP, Patient, Child, MAD, MTD, Principal, Safety, Disease, Pharmaceutical industry, Rett

DALLAS, June 05, 2023 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS), today announced that the first patient has been dosed with TSHA-102 in the Phase 1/2 REVEAL trial evaluating the safety and preliminary efficacy of TSHA-102 in adult patients with Rett syndrome. TSHA-102 is a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy that utilizes a novel miRNA-Responsive Auto-Regulatory Element (miRARE) platform designed to regulate cellular MECP2 expression. The study is being conducted at CHU Sainte-Justine, the Université de Montréal mother and child university hospital centre in Montreal, Canada.

Key Points: 
  • The study is being conducted at CHU Sainte-Justine, the Université de Montréal mother and child university hospital centre in Montreal, Canada.
  • This is a significant milestone that furthers our quest to bring a potentially transformational gene therapy to patients and families living with Rett syndrome.
  • The dosing of the first patient in this important clinical trial represents a critical advancement in evaluating the potential of gene therapy for Rett syndrome.
  • We’re pleased to collaborate with Taysha Gene Therapies in an effort to bring a gene therapy treatment that could meaningfully change the lives of patients and their caregivers.”

Taysha Gene Therapies Presents Preclinical Data on TSHA-102 for Rett Syndrome Demonstrating Cellular Regulation of MeCP2 Expression in Key Mouse Models at the American Society of Gene and Cell Therapy 26th Annual Meeting

Retrieved on: 
Friday, May 19, 2023
University of Texas Southwestern Medical Center, Central nervous system, University, Gene, Civil service commission, Gait, Survival, Rett syndrome, Cell, AAV, CNS, Female, Orphan drug, Patient, Risk, Annual general meeting, American Society of Gene and Cell Therapy, Growth, FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Safety, Society, Food, European Commission, Vaccine, Bridge, Rett, DALLAS, MECP2

DALLAS, May 19, 2023 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS), today presents preclinical data from neonatal mouse models on TSHA-102 for Rett syndrome, including new data in wild-type mice, at the American Society of Gene and Cell Therapy (ASGCT) 26th Annual Meeting. TSHA-102 utilizes a miniMECP2 gene and a novel miRNA-Responsive Auto-Regulatory Element (miRARE) technology designed to regulate cellular MECP2 expression. In a Taysha-sponsored study, the safety and efficacy of TSHA-102 were explored in both neonatal wild-type and Mecp2–/Y knockout mice, respectively. Preclinical in-life data on early intervention of TSHA-102 in neonatal mice suggest miRARE enables the expression of the MeCP2 protein in deficient CNS cells while preventing toxic overexpression within cells expressing normal levels of MeCP2.

Key Points: 
  • TSHA-102 utilizes a miniMECP2 gene and a novel miRNA-Responsive Auto-Regulatory Element (miRARE) technology designed to regulate cellular MECP2 expression.
  • In a Taysha-sponsored study, the safety and efficacy of TSHA-102 were explored in both neonatal wild-type and Mecp2–/Y knockout mice, respectively.
  • Preclinical in-life data on early intervention of TSHA-102 in neonatal mice suggest miRARE enables the expression of the MeCP2 protein in deficient CNS cells while preventing toxic overexpression within cells expressing normal levels of MeCP2.
  • We believe these new data from neonatal wild-type mice support the potential of miRARE to enable the optimal amount of MeCP2.

Acadia Pharmaceuticals Reports First Quarter 2023 Financial Results and Operating Overview

Retrieved on: 
Monday, May 8, 2023
Mental Health, Health, General Health, Research, Science, Pharmaceutical, Biotechnology, Schizophrenia, ACAD, Food, Rett syndrome, Ariad Pharmaceuticals, Inc. v. Eli Lilly & Co., PIN, Patient, PDP, Investment, Growth, FDA, Therapy, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Pimavanserin, Pharmaceutical industry, Cryptocurrency, Rett

Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced its financial results for the first quarter ended March 31, 2023.

Key Points: 
  • Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced its financial results for the first quarter ended March 31, 2023.
  • In connection with the FDA approval of DAYBUE, Acadia received a Rare Pediatric Disease Priority Review Voucher.
  • At March 31, 2023, Acadia’s cash, cash equivalents, and investment securities totaled $402.9 million, compared to $416.8 million at December 31, 2022.
  • Acadia is reiterating all of its 2023 guidance provided on February 27, 2023.

Acadia Pharmaceuticals Announces U.S. FDA Approval of DAYBUE™ (trofinetide) for the Treatment of Rett Syndrome in Adult and Pediatric Patients Two Years of Age and Older

Retrieved on: 
Saturday, March 11, 2023
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Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that the U.S. Food and Drug Administration (FDA) has approved DAYBUE™ (trofinetide) for the treatment of Rett syndrome in adult and pediatric patients two years of age and older.

Key Points: 
  • Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that the U.S. Food and Drug Administration (FDA) has approved DAYBUE™ (trofinetide) for the treatment of Rett syndrome in adult and pediatric patients two years of age and older.
  • DAYBUE is the first and only drug approved for the treatment of Rett syndrome.
  • View the full release here: https://www.businesswire.com/news/home/20230303005382/en/
    "Today marks an important milestone for the Rett community and Acadia.
  • Acadia management will discuss the FDA approval of DAYBUE for the treatment of Rett syndrome via conference call and webcast on Monday, March 13, 2023 at 8:30 a.m. Eastern Time.