Transthyretin

Intellia Therapeutics to Present Updated Interim Clinical Data from Ongoing Phase 1 Study of NTLA-2001 for the Treatment of Transthyretin (ATTR) Amyloidosis at the International Liver Congress™ 2022

Retrieved on: 
Wednesday, June 8, 2022

The presentation will include new data from the ongoing Phase 1 study evaluating NTLA-2001 in people living with hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN).

Key Points: 
  • The presentation will include new data from the ongoing Phase 1 study evaluating NTLA-2001 in people living with hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN).
  • Data supporting the fixed dose selection for Part 2, the single-dose expansion cohort of the polyneuropathy arm, will also be presented.
  • There are an estimated 50,000 people worldwide living with ATTRv amyloidosis and between 200,000 and 500,000 people with ATTRwt amyloidosis.
  • Intellia Therapeutics, a leading clinical-stage genome editing company, is developing novel, potentially curative therapeutics leveraging CRISPR-based technologies.

Intellia Therapeutics Receives U.S. FDA Orphan Drug Designation for NTLA-2001, an Investigational CRISPR Therapy for the Treatment of Transthyretin (ATTR) Amyloidosis

Retrieved on: 
Thursday, October 21, 2021

This investigational therapy is the first CRISPR therapy to be administered systemically to edit a disease-causing gene inside the human body.

Key Points: 
  • This investigational therapy is the first CRISPR therapy to be administered systemically to edit a disease-causing gene inside the human body.
  • Orphan drug designation underscores the FDAs recognition of NTLA-2001s potential promise as a single-dose, novel therapy for the treatment of ATTR amyloidosis, said Intellia President and Chief Executive Officer John Leonard, M.D.
  • NTLA-2001 is currently being studied in a Phase 1 trial in adults with hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN).
  • The decision by the FDA follows a March 2021 decision by the European Commission (EC) to also grant NTLA-2001 orphan drug designation for the treatment of ATTR amyloidosis.

Attralus Appoints Jake Bauer to Board of Directors

Retrieved on: 
Wednesday, July 28, 2021

SOUTH SAN FRANCISCO, Calif., July 28, 2021 (GLOBE NEWSWIRE) -- Attralus, Inc., a clinical stage biopharmaceutical company developing transformative medicines to improve the lives of patients with systemic amyloidosis, today announced that Jake Bauer joined the companys Board of Directors as an independent director.

Key Points: 
  • SOUTH SAN FRANCISCO, Calif., July 28, 2021 (GLOBE NEWSWIRE) -- Attralus, Inc., a clinical stage biopharmaceutical company developing transformative medicines to improve the lives of patients with systemic amyloidosis, today announced that Jake Bauer joined the companys Board of Directors as an independent director.
  • We are delighted to welcome Mr. Bauer to our Board of Directors.
  • Mr. Bauer currently serves as a member of the Board of Directors for Enliven Therapeutics, Phoenix Tissue Repair, Inc. and Arya Sciences Acquisition Corp V.
    It is a privilege to join Attralus Board at this exciting time in the companys evolution, said Mr. Bauer.
  • Attralus was founded by scientific experts in the field of amyloidosis and is headquartered in South San Francisco, California.

Prothena and Novo Nordisk Announce Acquisition Agreement for Prothena’s ATTR Amyloidosis Programme

Retrieved on: 
Monday, July 12, 2021

PRX004 is a phase 2 ready anti-amyloid immunotherapy designed to deplete the amyloid deposits that are associated with the disease pathology of ATTR amyloidosis.

Key Points: 
  • PRX004 is a phase 2 ready anti-amyloid immunotherapy designed to deplete the amyloid deposits that are associated with the disease pathology of ATTR amyloidosis.
  • Novo Nordisk will initially focus on the clinical development of PRX004 in ATTR cardiomyopathy - an underdiagnosed and potentially fatal form of ATTR amyloidosis characterised by build-up of amyloid deposits in cardiac tissue.
  • Under the terms of the definitive purchase agreement, Novo Nordisk acquires Prothenas wholly-owned subsidiary and gains full worldwide rights to the intellectual property and related rights of Prothenas ATTR amyloidosis business and pipeline.
  • This acquisition is a testament to Prothenas pioneering work in ATTR amyloidosis and Novo Nordisks dedication to advancing new disease-modifying therapies for the benefit of people with cardiovascular diseases which are the worlds leading cause of death.

Intellia and Regeneron Announce Landmark Clinical Data Showing Deep Reduction in Disease-Causing Protein After Single Infusion of NTLA-2001, an Investigational CRISPR Therapy for Transthyretin (ATTR) Amyloidosis

Retrieved on: 
Saturday, June 26, 2021

These are the first ever clinical data suggesting that we can precisely edit target cells within the body to treat genetic disease with a single intravenous infusion of CRISPR.

Key Points: 
  • These are the first ever clinical data suggesting that we can precisely edit target cells within the body to treat genetic disease with a single intravenous infusion of CRISPR.
  • By contrast, the standard of care for ATTRv-PN, which requires chronic treatment, typically yields TTR reductions of approximately 80%.
  • Robust preclinical data, showing deep and long-lasting transthyretin (TTR) reduction following in vivo inactivation of the target gene, supports NTLA-2001s potential as a single-administration therapeutic.
  • Interim Phase 1 clinical data released in June 2021 confirm substantial, dose-dependent reduction of TTR protein following a single dose of NTLA-2001.

Attralus Announces Presentation of Clinical Data on AT-01, a Novel Diagnostic for Systemic Amyloidosis, at the 2021 Society of Nuclear Molecular Imaging Meeting

Retrieved on: 
Tuesday, June 15, 2021

An oral presentation and posters described results from a clinical study evaluating AT-01 (124I-p5+14), a novel, radiolabeled, synthetic amyloid-targeting peptide designed for imaging systemic amyloid deposits.

Key Points: 
  • An oral presentation and posters described results from a clinical study evaluating AT-01 (124I-p5+14), a novel, radiolabeled, synthetic amyloid-targeting peptide designed for imaging systemic amyloid deposits.
  • Currently, a definitive diagnosis of systemic amyloidosis is often made following histological evaluation of a tissue biopsy, said Dr. Wall.
  • In addition, this imaging modality may provide further clinical information to help improve patient management and disease understanding.
  • Attralus is a clinical stage biopharmaceutical company focused on creating transformative medicines to improve the lives of patients with systemic amyloidosis.

Prothena to Participate in JMP Securities Life Sciences Conference on June 16

Retrieved on: 
Thursday, June 10, 2021

Following the live presentation, a replay of the webcast will be available on the Company's website for at least 90 days following the presentation date.

Key Points: 
  • Following the live presentation, a replay of the webcast will be available on the Company's website for at least 90 days following the presentation date.
  • Prothena Corporation plc is a late-stage clinical company with a robust pipeline of novel investigational therapeutics, built on protein dysregulation expertise, with the potential to change the course of devastating rare peripheral amyloid and neurodegenerative diseases.
  • Prothenas pipeline includes both wholly-owned and partnered programs being developed for the potential treatment of diseases including AL amyloidosis, ATTR amyloidosis, Alzheimers disease, Parkinsons disease and a number of other neurodegenerative diseases.
  • For more information, please visit the Companys website at www.prothena.com and follow the Company on Twitter @ProthenaCorp.

Intellia Therapeutics to Present Interim Clinical Data from Ongoing Phase 1 Study of NTLA-2001 for the Treatment of Transthyretin (ATTR) Amyloidosis at the 2021 Peripheral Nerve Society Annual Meeting

Retrieved on: 
Friday, June 4, 2021

The presentation will include interim data from the ongoing dose-escalation portion of Intellias Phase 1 trial evaluating NTLA-2001 in people living with hereditary ATTR amyloidosis with polyneuropathy (hATTR-PN).

Key Points: 
  • The presentation will include interim data from the ongoing dose-escalation portion of Intellias Phase 1 trial evaluating NTLA-2001 in people living with hereditary ATTR amyloidosis with polyneuropathy (hATTR-PN).
  • Following safety assessment and dose optimization, Intellia intends to further evaluate NTLA-2001 in a broader population of people with ATTR amyloidosis, including those with cardiomyopathy.
  • Intellia will host a live webcast on Monday, June 28, 2021 at 8:00 a.m. E.T.
  • Intellias global Phase 1 trial is an open-label, multi-center, two-part study of NTLA-2001 in adults with hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN).

Alnylam Completes Enrollment in APOLLO-B Phase 3 Study of Patisiran in Transthyretin-Mediated (ATTR) Amyloidosis Patients with Cardiomyopathy

Retrieved on: 
Tuesday, June 1, 2021

Enrollment was completed with over 300 ATTR amyloidosis patients across 90 sites in more than 20 countries.

Key Points: 
  • Enrollment was completed with over 300 ATTR amyloidosis patients across 90 sites in more than 20 countries.
  • The APOLLO-B trial was initiated based on encouraging data from previous studies that support further investigation of patisiran in ATTR amyloidosis patients with cardiomyopathy.
  • Specifically, encouraging results were obtained from the landmark Phase 3 APOLLO study in hATTR amyloidosis patients with polyneuropathy on a number of exploratory cardiac endpoints.
  • The APOLLO-B study is a Phase 3, randomized, double-blind, placebo-controlled multicenter global study designed to evaluate the efficacy and safety of patisiran in patients with transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy, which enrolled over 300 adult patients with ATTR amyloidosis (hereditary or wild-type) with cardiomyopathy.

Attralus Announces Presentation of Clinical Data on AT-01, a Novel, Potential First-in-Class Diagnostic for Systemic Amyloidosis at the 2021 American College of Cardiology Annual Meeting

Retrieved on: 
Monday, May 17, 2021

AT-01 is a novel diagnostic that provides the potential to change the treatment paradigm by enabling detection of all types of systemic amyloidosis at all stages of disease.\xe2\x80\x9d\nCurrently, there are no FDA-approved imaging agents as diagnostics for systemic amyloidosis.

Key Points: 
  • AT-01 is a novel diagnostic that provides the potential to change the treatment paradigm by enabling detection of all types of systemic amyloidosis at all stages of disease.\xe2\x80\x9d\nCurrently, there are no FDA-approved imaging agents as diagnostics for systemic amyloidosis.
  • Systemic amyloidosis is significantly underdiagnosed due to low awareness, lack of specific symptoms, and no current disease-specific diagnostics.
  • The two most common forms of systemic amyloidosis are immunoglobulin light-chain (AL) amyloidosis and transthyretin amyloidosis (ATTR).\nAttralus is a clinical stage biopharmaceutical company focused on creating transformative medicines to improve the lives of patients with systemic amyloidosis.
  • By targeting the universal disease-causing pathology in systemic amyloidosis diseases, PAR therapeutics have the potential to treat and reverse disease in patients with all types and stages of systemic amyloidosis.