CHDI

Bridger Aerospace Strengthens Board with the Addition of Seasoned Business Leaders Elizabeth C. Fascitelli and David A. Schellenberg as Independent Directors

Retrieved on: 
星期五, 三月 8, 2024

Jeffrey Kelter, Bridger’s Chairman, commented, “The addition of two proven leaders is a very powerful statement in Bridger’s continuing evolution as a public company.

Key Points: 
  • Jeffrey Kelter, Bridger’s Chairman, commented, “The addition of two proven leaders is a very powerful statement in Bridger’s continuing evolution as a public company.
  • Additionally, Mr. Schellenberg added that "I am honored to join the Board at such an exciting time for Bridger.
  • Ms. Fascitelli earned a Bachelor of Arts from Dartmouth College and a Master’s in Business Administration from Harvard Business School.
  • David A. Schellenberg, age 60, is a seasoned businessman with over 30 years of operating and financial leadership and risk management experience.

uniQure Announces Update on Phase I/II Clinical Trials of AMT-130 Gene Therapy for the Treatment of Huntington’s Disease

Retrieved on: 
星期二, 十二月 19, 2023

The U.S. study consists of a blinded 12-month core study period followed by unblinded long-term follow-up of five years for treated patients.

Key Points: 
  • The U.S. study consists of a blinded 12-month core study period followed by unblinded long-term follow-up of five years for treated patients.
  • Six patients were treated with AMT-130 in the initial low-dose cohort and seven patients were treated in the subsequent high-dose cohort.
  • The cohort includes 31 patients that met the uniQure clinical trial inclusion criteria of Total Functional Capacity, Diagnostic Classification Level and minimum striatal volumes.
  • In mid-2024, uniQure expects to present another clinical update from the ongoing Phase I/II studies of AMT-130, including additional follow-up data from the treated patients in the U.S. and European trials.

uniQure Announces Update on U.S. Phase I/II Clinical Trial of AMT-130 Gene Therapy for the Treatment of Huntington’s Disease

Retrieved on: 
星期三, 六月 21, 2023

Phase I/II clinical trial of AMT-130 for the treatment of Huntington’s disease.

Key Points: 
  • Phase I/II clinical trial of AMT-130 for the treatment of Huntington’s disease.
  • Phase I/II clinical trial of AMT-130, a one-time administered investigational gene therapy for Huntington’s Disease,” stated Ricardo Dolmetsch, Ph.D., president of research and development at uniQure .
  • Phase I/II Trial of AMT-130 in Huntington’s Disease
    A total of 26 patients with early-manifest Huntington’s disease have been enrolled in the multi-center, U.S.
  • Phase I/II clinical trial concluded there are no safety concerns with either dose and recommended continuing clinical development of AMT-130.

Arvinas Reports First Quarter 2023 Financial Results and Provides Corporate Update

Retrieved on: 
星期五, 五月 5, 2023

NEW HAVEN, Conn., May 05, 2023 (GLOBE NEWSWIRE) -- Arvinas, Inc. (Nasdaq: ARVN), a clinical-stage biotechnology company creating a new class of drugs based on targeted protein degradation, today reported financial results for the first quarter ended March 31, 2023 and provided a corporate update.

Key Points: 
  • “We made substantial progress throughout the first quarter across all areas of our early- and late-stage pipeline,” said John Houston, Ph.D., president and chief executive officer at Arvinas.
  • Data are expected to be presented at a medical conference in the second half of 2023.
  • Revenues: Revenues were $32.5 million for the quarter ended March 31, 2023, as compared with $26.5 million for the quarter ended March 31, 2022.
  • Net Loss: Net loss was $81.9 million for the quarter ended March 31, 2023, as compared with $63.4 million for the quarter ended March 31, 2022.

Alnylam Pharmaceuticals Reports First Quarter 2023 Financial Results and Highlights Recent Period Activity

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星期四, 五月 4, 2023

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today reported its consolidated financial results for the first quarter ended March 31, 2023 and reviewed recent business highlights.

Key Points: 
  • Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today reported its consolidated financial results for the first quarter ended March 31, 2023 and reviewed recent business highlights.
  • Attained over 3,160 hATTR amyloidosis patients with polyneuropathy worldwide on commercial treatment with ONPATTRO or AMVUTTRA as of March 31, 2023.
  • Alnylam announces today that it intends to present 18-month results from the APOLLO-B Phase 3 study of patisiran at ESC-HF 2023, being held May 20-23, 2023 in Prague.
  • Financial Results for the Quarter Ended March 31, 2023

Rumi Scientific Announces Data Demonstrating Disease Modifying Effect of BRD9 Inhibition in a Huntington’s Disease Animal Model

Retrieved on: 
星期三, 四月 26, 2023

The research was presented at the CHDI Huntington’s Disease Therapeutics Conference, being held, April 24-27, in Dubrovnik, Croatia.

Key Points: 
  • The research was presented at the CHDI Huntington’s Disease Therapeutics Conference, being held, April 24-27, in Dubrovnik, Croatia.
  • “BRD9 inhibition is a novel target for the treatment of HD identified by our proprietary high throughput organoid phenotypic screening platform,” said Fred Etoc, Chief Scientific Officer of Rumi.
  • “We have previously demonstrated, in vitro, that BRD9 inhibition rescues several of the biochemical dysfunctions known to occur in HD and thus could be disease modifying.
  • These data demonstrate the potential of a novel disease modifying therapeutic target to reverse pathologies in a HD animal model.

Prilenia Shares Preliminary Topline Results from Phase 3 PROOF-HD Clinical Trial in Huntington’s Disease and Data from Phase 2 HEALEY ALS Platform Trial of Pridopidine at the 75th American Academy of Neurology (AAN) Annual Meeting

Retrieved on: 
星期二, 四月 25, 2023

These initial results, along with recently announced findings from the pridopidine arm of the HEALEY ALS Platform Trial, were presented today at the 75th American Academy of Neurology (AAN) Annual Meeting taking place in Boston, MA.

Key Points: 
  • These initial results, along with recently announced findings from the pridopidine arm of the HEALEY ALS Platform Trial, were presented today at the 75th American Academy of Neurology (AAN) Annual Meeting taking place in Boston, MA.
  • Effects on both of these measures were reduced by the use of concomitant medications.
  • Pridopidine was well-tolerated with no serious treatment-related adverse events, with a safety and tolerability profile similar to placebo and consistent with previous clinical studies.
  • “The Company is committed to advancing pridopidine in both HD and ALS, as well as additional indications.”

Molecular Design Platform Accelerating Research For Huntington's Disease

Retrieved on: 
星期四, 七月 30, 2020

SANTA FE, N.M., & NEW YORK, July 30, 2020 /PRNewswire/ --OpenEye Scientific (OpenEye) and CHDI Foundation (CHDI) today announced a research collaboration to identify novel small-molecule therapeutics for Huntington's disease (HD) utilizing Orion, OpenEye's cloud-native molecular design platform.

Key Points: 
  • SANTA FE, N.M., & NEW YORK, July 30, 2020 /PRNewswire/ --OpenEye Scientific (OpenEye) and CHDI Foundation (CHDI) today announced a research collaboration to identify novel small-molecule therapeutics for Huntington's disease (HD) utilizing Orion, OpenEye's cloud-native molecular design platform.
  • Further molecular modeling of selected compounds will be performed in concert by OpenEye and CHDI scientists using Orion.
  • "We are proud to work with CHDI and to use our new cloud platform, Orion, to help combat Huntington's disease," said Dr. Anthony Nicholls, CEO of OpenEye Scientific.
  • OpenEye Scientificis an industry leader in computational molecular design based on decades of delivering rapid, robust, and scalable software, toolkits, and technology and design services across the drug discovery process.

Charles River Laboratories and CHDI Foundation Extend Huntington’s Disease Collaboration

Retrieved on: 
星期二, 四月 9, 2019

Charles River Laboratories International, Inc. (NYSE: CRL) and CHDI Foundation today announced a five-year extension of their ongoing collaboration.

Key Points: 
  • Charles River Laboratories International, Inc. (NYSE: CRL) and CHDI Foundation today announced a five-year extension of their ongoing collaboration.
  • The organizations began working together in 2005 on drug discovery and development for Huntingtons disease (HD) , a genetic neurological disease that causes the progressive dysfunction of nerve cells in the brain.
  • The extension will allow more flexible activity across the different Charles River platforms and entail greater integration into CHDIs programs.
  • Our longstanding partnership with CHDI is built on our mutual commitment to developing novel therapies for Huntingtons disease.