Autoantigens

Bristol Myers Squibb Presents Late-Breaking Phase 3 Data Demonstrating Health Status Benefits of Mavacamten in Patients with Obstructive Hypertrophic Cardiomyopathy at American College of Cardiology’s 70th Annual Scientific Session

Retrieved on: 
Saturday, May 15, 2021

At 30 weeks, the change in Kansas City Cardiomyopathy Questionnaire Overall Summary Score (KCCQ OSS) was greater in mavacamten patients than placebo, with similar benefits across all KCCQ subscales.

Key Points: 
  • At 30 weeks, the change in Kansas City Cardiomyopathy Questionnaire Overall Summary Score (KCCQ OSS) was greater in mavacamten patients than placebo, with similar benefits across all KCCQ subscales.
  • Moreover, a greater proportion of mavacamten patients achieved a very large, clinically meaningful improvement (\xe2\x89\xa520 points) in the KCCQ OSS, compared to placebo, 36% [33/92] vs. 15% [13/88].
  • A change of at least 5 points is required to be considered clinically significant.
  • Mavacamten reduces cardiac muscle contractility by inhibiting excessive myosin-actin cross-bridge formation that results in hypercontractility, left ventricular hypertrophy and reduced compliance.

Wave Life Sciences Reports Second Quarter 2020 Financial Results and Provides Business Update

Retrieved on: 
Monday, August 10, 2020

ET today

Key Points: 
  • ET today
    CAMBRIDGE, Mass., Aug. 10, 2020 (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 announced financial results for the second quarter ended June 30, 2020 and provided a business update.
  • In the second quarter of 2020, Wave achieved successful editing of ACTB (Beta-actin) mRNA in non-human primates (NHPs) via endogenous ADARs using stereopure GalNAc-conjugated oligonucleotides.
  • ET to discuss the companys second quarter 2020 operating results and provide a business update.
  • The live webcast may be accessed from the investor relations section of the Wave Life Sciences corporate website at ir.wavelifesciences.com .

Protein Glutamine Gamma Glutamyltransferase 2: Pipeline Review, H2 2019 - ResearchAndMarkets.com

Retrieved on: 
Friday, January 3, 2020

The "Protein Glutamine Gamma Glutamyltransferase 2 - Pipeline Review, H2 2019" drug pipelines has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Protein Glutamine Gamma Glutamyltransferase 2 - Pipeline Review, H2 2019" drug pipelines has been added to ResearchAndMarkets.com's offering.
  • According to the recently published report 'Protein Glutamine Gamma Glutamyltransferase 2 - Pipeline Review, H2 2019'; Protein Glutamine Gamma Glutamyltransferase 2 (Tissue Transglutaminase or Transglutaminase C or Transglutaminase H or Transglutaminase 2 or TGase C or TGase H or TGM2 or EC 2.3.2.13) pipeline Target constitutes close to 6 molecules.
  • Protein Glutamine Gamma Glutamyltransferase 2 (Tissue Transglutaminase or Transglutaminase C or Transglutaminase H or Transglutaminase 2 or TGase C or TGase H or TGM2 or EC 2.3.2.13) - Transglutaminase 2 (TG2) is a member of the transglutaminase family that catalyzes calcium-dependent posttranslational modification of proteins by inserting highly stable isopeptide bonds between polypeptide chains or by conjugating polyamines to proteins.
  • TG2 also exhibits GTPase activity and can serve as a signal-transduction G protein.

Krystal Biotech Announces Initiation of Phase 1/2 Study of KB105 in Transglutaminase-1 (“TGM1”) Deficient Autosomal Recessive Congenital Ichthyosis (“ARCI”)

Retrieved on: 
Wednesday, September 4, 2019

PITTSBURGH, Sept. 04, 2019 (GLOBE NEWSWIRE) -- Krystal Biotech Inc. , (Krystal) (NASDAQ: KRYS), a gene therapy company developing medicines to treat rare skin diseases, today announced the initiation of the Phase 1/2 study of KB105 in transglutaminase-1 (TGM1) deficient autosomal recessive congenital ichthyosis (ARCI).

Key Points: 
  • PITTSBURGH, Sept. 04, 2019 (GLOBE NEWSWIRE) -- Krystal Biotech Inc. , (Krystal) (NASDAQ: KRYS), a gene therapy company developing medicines to treat rare skin diseases, today announced the initiation of the Phase 1/2 study of KB105 in transglutaminase-1 (TGM1) deficient autosomal recessive congenital ichthyosis (ARCI).
  • The study, termed GEM-3 study, will examine KB105, an HSV-1 based gene therapy engineered to deliver a human TGM1 gene, in patients with ARCI.
  • Target areas will be imaged and evaluated for safety and efficacy and subjects will be on-trial for approximately 3.5 months.
  • The loss of TGM1 activity results in the severe genetic skin disease autosomal recessive congenital ichthyosis (ARCI).

Oyster Point Pharma to Present at the Cowen and Company 39th Annual Health Care Conference

Retrieved on: 
Tuesday, March 5, 2019

Oyster Point Pharma, Inc., a privately held clinical-stage pharmaceutical company with an initial focus on developing novel therapies for the treatment of Dry Eye Disease, today announced that Jeffrey Nau, Ph.D., M.M.S., President and CEO, is scheduled to present a corporate overview at the Cowen and Company 39th Annual Health Care Conference on Monday, March 11th, 2019 at 5:00 p.m.

Key Points: 
  • Oyster Point Pharma, Inc., a privately held clinical-stage pharmaceutical company with an initial focus on developing novel therapies for the treatment of Dry Eye Disease, today announced that Jeffrey Nau, Ph.D., M.M.S., President and CEO, is scheduled to present a corporate overview at the Cowen and Company 39th Annual Health Care Conference on Monday, March 11th, 2019 at 5:00 p.m.
  • ET at the Boston Marriott Copley Place.
  • Oyster Point Pharma is a clinical-stage pharmaceutical company leveraging neuroscience to discover, develop, and commercialize novel therapies to treat diseases with high unmet needs.
  • Oyster Point is leveraging a class of molecules called nicotinic acetylcholine receptor (nAChR) agonists to bind to receptors on the trigeminal nerve, accessible within the nose, to stimulate natural tear film production.

Oyster Point Pharma to Present at the BIO CEO & Investor Conference

Retrieved on: 
Thursday, January 24, 2019

Oyster Point Pharma, Inc., a privately held clinical-stage pharmaceutical company with an initial focus on developing novel therapies for the treatment of Dry Eye Disease, today announced that Jeffrey Nau, M.M.S., Ph.D., President and CEO, is scheduled to present a corporate overview at the BIO CEO & Investor Conference on Tuesday, February 12th, 2019 at 10:30 a.m. EST in New York.

Key Points: 
  • Oyster Point Pharma, Inc., a privately held clinical-stage pharmaceutical company with an initial focus on developing novel therapies for the treatment of Dry Eye Disease, today announced that Jeffrey Nau, M.M.S., Ph.D., President and CEO, is scheduled to present a corporate overview at the BIO CEO & Investor Conference on Tuesday, February 12th, 2019 at 10:30 a.m. EST in New York.
  • Oyster Point Pharma is a clinical-stage pharmaceutical company leveraging neuroscience to discover, develop, and commercialize novel therapies to treat diseases with high unmet needs.
  • Oyster Point is leveraging a class of molecules called nicotinic acetylcholine receptor (nAChR) agonists to bind receptors on the trigeminal nerve, accessible within the nose, to stimulate natural tear film production.
  • View source version on businesswire.com: https://www.businesswire.com/news/home/20190124005517/en/