MECP2

Guidehouse Awarded Department of Defense Contract for Army Prognostics & Predictive Maintenance Prototype

Thursday, October 14, 2021 - 2:00pm

WASHINGTON, Oct. 14, 2021 /PRNewswire/ -- Guidehouse , a leading global provider of consulting and managed services to public sector and commercial clients, today announced it has been awarded a contract by the US Department of Defense fora prototype Prognostics & Predictive Maintenance (PPMx) analytical capability for the US Army that will provide actionable analysis for fleet management, deployment, supply chain, and maintenance to improve Army operations.

Key Points: 
  • WASHINGTON, Oct. 14, 2021 /PRNewswire/ -- Guidehouse , a leading global provider of consulting and managed services to public sector and commercial clients, today announced it has been awarded a contract by the US Department of Defense fora prototype Prognostics & Predictive Maintenance (PPMx) analytical capability for the US Army that will provide actionable analysis for fleet management, deployment, supply chain, and maintenance to improve Army operations.
  • The PPMx initiative will modernize Army supply and maintenance processes by providing analysis, predictions, and prescriptive recommendations.
  • Guidehouse brings advanced analytics capabilities to the project, coupled with extensive knowledge of Army processes, data, systems, and culture to help the Army improve equipment availability and fleet readiness across the force.
  • Partnering with TIBCO , a global leader in enterprise data and analytics software, Guidehouse will provide an on-demand prognostic and predictive maintenance analytical capability using multiple data sources and provide recommendations for improvement.

Wave Life Sciences Announces New Data for Leading RNA Editing Capability Across Multiple Tissues and Provides Update on AATD Program During Analyst and Investor Research Webcast

Tuesday, September 28, 2021 - 9:05pm

CAMBRIDGE, Mass., Sept. 28, 2021 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases, today presented new data for its ADAR-mediated RNA editing capability (ADAR editing), including new preclinical editing data across multiple tissues, as well as an update on its discovery-stage alpha-1 antitrypsin deficiency (AATD) program during the company’s 2021 Analyst and Investor Research Webcast. The webcast also included updates on the company’s PRISM™ platform and initial results from the application of ADAR editing to neurology targets.

Key Points: 
  • The webcast also included updates on the companys PRISMplatform and initial results from the application of ADAR editing to neurology targets.
  • A replay of the Analyst and Investor Research Webcast is available on Waves Investor Relations website.
  • Additionally, in vitro data were presented demonstrating the potential to target protein-protein interactions and upregulate downstream gene expression with AIMers.
  • Wave undertakes no obligation to update the information contained in this press release to reflect subsequently occurring events or circumstances.

Taysha Receives Orphan Drug Designation from the European Commission for TSHA-102 for the Treatment of Rett Syndrome

Wednesday, September 22, 2021 - 12:00pm

TSHA-102 is a self-complementary intrathecally delivered AAV9 gene replacement therapy under development for the treatment of Rett syndrome.

Key Points: 
  • TSHA-102 is a self-complementary intrathecally delivered AAV9 gene replacement therapy under development for the treatment of Rett syndrome.
  • TSHA-102 has previously received rare pediatric disease designation and orphan drug designation from the U.S. Food and Drug Administration (FDA).
  • The European Commission grants orphan drug designation for medicines being developed for the diagnosis, prevention or treatment of treat life-threatening or chronically debilitating conditions that affect fewer than five in 10,000 people in the European Union.
  • Orphan designation in the European Union includes benefits such as protocol assistance, reduced regulatory fees and market exclusivity.

Anavex Life Sciences Announces ANAVEX®2-73 (Blarcamesine) Biomarker Correlated with Efficacy Endpoints in Placebo-Controlled U.S. Phase 2 Clinical Trial for the Treatment of Adult Patients with Rett Syndrome

Monday, June 21, 2021 - 12:00pm

Rett syndrome is caused by mutations in the MECP2 gene and strikes all racial and ethnic groups.

Key Points: 
  • Rett syndrome is caused by mutations in the MECP2 gene and strikes all racial and ethnic groups.
  • The population of patients with Rett syndrome is estimated to be approximately 11,000 patients in the U.S.
  • The Phase 2 trial is a randomized double-blind, placebo-controlled safety, tolerability, pharmacokinetic and efficacy study of oral liquid ANAVEX2-73 to treat Rett syndrome in a total of 31 adult patients with Rett syndrome over a 7-weeks treatment period (End of Trial, EOT) were evaluated incorporating precision medicine biomarkers.
  • Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof.

Taysha Gene Therapies Announces Publication of Preclinical Data for TSHA-102 in Rett Syndrome in Brain, a Highly Esteemed Neurological Science Peer-Reviewed Journal

Monday, May 10, 2021 - 12:00pm

\xe2\x80\x9cHistorically, unregulated gene replacement of MECP2 resulted in overt adverse events, including death in wild type mice due to overexpression of the MECP2 protein.

Key Points: 
  • \xe2\x80\x9cHistorically, unregulated gene replacement of MECP2 resulted in overt adverse events, including death in wild type mice due to overexpression of the MECP2 protein.
  • With the built-in regulatory element, miRARE, TSHA-102 provided a statistically significant survival extension by 56% in 4- to 5-week-old knockout Rett mice.
  • We see the potential for broadening the miRARE platform to other CNS diseases requiring regulated gene expression.
  • Additional information will be made available in other filings that we make from time to time with the SEC.

DiamiR Awarded $3.86M in Two Grants from the NIH

Thursday, October 1, 2020 - 1:02pm

The second award of $498,572 over a year and a half was granted to DiamiR by the National Institute for Neurological Disorders and Stroke (NINDS) of the NIH for a project entitled "Circulating Organ-enriched microRNAs as biomarkers of Rett Syndrome."

Key Points: 
  • The second award of $498,572 over a year and a half was granted to DiamiR by the National Institute for Neurological Disorders and Stroke (NINDS) of the NIH for a project entitled "Circulating Organ-enriched microRNAs as biomarkers of Rett Syndrome."
  • Rett syndrome (RTT) is a rare X-linked neurodevelopmental disorder caused by mutations in the MECP2 gene that affects almost exclusively females.
  • "We are delighted to receive these highly competitive awards from the NIH," added Kira Sheinerman, PhD, CEO of DiamiR.
  • These statements are predictions and are subject to risks and uncertainties that could cause the actual events or results to differ materially.

Genetic Editing Milestone in Mouse Model of Rett Syndrome

Wednesday, July 15, 2020 - 4:00am

The new research, published July 14 in the journal Cell Reports, shows that RNA editing may repair the underlying cause of Rett Syndrome in a mouse model.

Key Points: 
  • The new research, published July 14 in the journal Cell Reports, shows that RNA editing may repair the underlying cause of Rett Syndrome in a mouse model.
  • The results represent a promising early step in using RNA editing to treat Rett Syndrome, a disorder that affects about 350,000 individuals worldwide.
  • "The study is the first example of RNA editing in a mouse model of a neurological disease, and therefore a considerable step forward in the potential of RNA editing becoming a therapeutic for Rett Syndrome," says Monica Coenraads, executive director of Rett Syndrome Research Trust, which helped fund the study.
  • The Rett Syndrome Research Trust (RSRT) is a non-profit organization with a highly personal and urgent mission: a cure for Rett Syndrome and related MECP2 disorders.

Anavex Life Sciences Receives Rare Pediatric Disease Designation from FDA for ANAVEX®2-73 (blarcamesine) for the Treatment of Rett Syndrome

Thursday, November 14, 2019 - 12:00pm

The RPD designation provides priority review by theFDAto encourage treatments for rare pediatric diseases.

Key Points: 
  • The RPD designation provides priority review by theFDAto encourage treatments for rare pediatric diseases.
  • We are pleased to have received the rare pediatric designation from the U.S. FDA for ANAVEX2-73 (blarcamesine) for young patients with Rett syndrome, said Christopher U. Missling, Ph.D., Chief Executive Officer of Anavex.
  • Our goal is to advance a potential treatment for Rett syndrome in order to bring medicines to patients as soon as possible.
  • Rett syndrome is caused by mutations in the MECP2 gene and strikes all racial and ethnic groups.

Rett Syndrome Research Trust Names Timothy A. Riley Chief Scientific Officer and Chief Business Officer; Randall Carpenter Transitions to Chief Medical Officer

Wednesday, September 26, 2018 - 3:00pm

TRUMBULL, Conn., Sept. 26, 2018 /PRNewswire-PRWeb/ -- The Rett Syndrome Research Trust (RSRT) is delighted to announce that Timothy A. Riley, Ph.D. has joined the organization in the role of Chief Scientific Officer and Chief Business Officer.

Key Points: 
  • TRUMBULL, Conn., Sept. 26, 2018 /PRNewswire-PRWeb/ -- The Rett Syndrome Research Trust (RSRT) is delighted to announce that Timothy A. Riley, Ph.D. has joined the organization in the role of Chief Scientific Officer and Chief Business Officer.
  • who previously held the position of Chief Scientific Officer is transitioning to Chief Medical Officer.
  • The decision to expand the research team is in response to numerous recent scientific breakthroughs that enable potentially transformative therapeutics for individuals with Rett Syndrome.
  • The Rett Syndrome Research Trust (RSRT) is a nonprofit organization with a highly personal and urgent mission: a cure for Rett Syndrome and related MECP2 disorders.