Alpha-1 antitrypsin

Dicerna™ to Begin Clinical Development of DCR-A1AT for Treatment of Patients with Alpha-1 Antitrypsin Deficiency-Associated Liver Disease

Retrieved on: 
Tuesday, July 2, 2019
Biotechnology, FDA, Health, Pharmaceutical, Other Science, Research, General Health, Genetics, Science, Clinical trials, Acute phase proteins, Alpha-1 antitrypsin, Medical specialties, Organ systems, Branches of biology, Liver disease, Alpha 1, Liver, Alpha 1-antitrypsin deficiency, DCR-A1AT and the GalXC™ Technology Platform, Alpha-1 Antitrypsin (A1AT) Deficiency, DicernaTM Pharmaceuticals, Inc.

First, A1AT deficiency-associated liver disease fits with our rare disease strategy as an indication with a significant unmet medical need and a clear biomarker, presenting a rapid development path to approval.

Key Points: 
  • First, A1AT deficiency-associated liver disease fits with our rare disease strategy as an indication with a significant unmet medical need and a clear biomarker, presenting a rapid development path to approval.
  • Dicerna plans to conduct the study in up to 16 sites across Europe, with the first clinical trial site in Sweden.
  • DCR-A1AT is a ribonucleic acid interference (RNAi) therapeutic being investigated for the treatment of liver disease in patients with alpha-1 antitrypsin (A1AT) deficiency.
  • Alpha-1 antitrypsin (A1AT) deficiency is an inherited disorder that can lead to liver disease in children and adults and lung disease in adults.

Arrowhead Pharmaceuticals Receives Fast Track Designation for ARO-AAT for Treatment of Alpha-1 Liver Disease

Retrieved on: 
Thursday, June 27, 2019
Biotechnology, FDA, Health, Pharmaceutical, Oncology, Infectious diseases, General Health, Genetics, Clinical trials, Cardiology, Arrowhead Pharmaceuticals, Health, Medical specialties, Food and Drug Administration, Fast track, Alpha 1-antitrypsin deficiency, Alpha-1 antitrypsin, Fast track, Aro

Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to ARO-AAT, the companys second generation subcutaneously administered RNA interference (RNAi) therapeutic being developed as a treatment for a rare genetic liver disease associated with alpha-1 antitrypsin deficiency (AATD).

Key Points: 
  • Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to ARO-AAT, the companys second generation subcutaneously administered RNA interference (RNAi) therapeutic being developed as a treatment for a rare genetic liver disease associated with alpha-1 antitrypsin deficiency (AATD).
  • We view this FDA Fast Track designation as continued support that ARO-AAT has the potential to address this unmet need.
  • Importantly, Fast Track designation provides a number of important advantages that could expedite the development and review of ARO-AAT.
  • Fast Track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need.

Alpha-1 Antitrypsin Congenital Deficiency Clinical Trial Pipeline Highlights 2019 - Global Forecast to 2024 by Clinical Trial Stage, Drug Mechanism Class, Company - ResearchAndMarkets.com

Retrieved on: 
Friday, June 14, 2019
Health, Clinical trials, Pharmaceutical, Health, Acute phase proteins, Drug discovery, Alpha-1 antitrypsin, Alpha 1, Clinical trial

The "Global Alpha-1 Antitrypsin Congenital Deficiency Clinical Trial Pipeline Highlights - 2019" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Global Alpha-1 Antitrypsin Congenital Deficiency Clinical Trial Pipeline Highlights - 2019" report has been added to ResearchAndMarkets.com's offering.
  • Alpha-1 Antitrypsin Congenital Deficiency Pipeline Highlights - 2019, provides most up-to-date information on key pipeline products in the global Alpha-1 Antitrypsin Congenital Deficiency market.
  • It covers emerging therapies for Alpha-1 Antitrypsin Congenital Deficiency in active clinical development stages including early and late stage clinical trials.
  • The report provides Alpha-1 Antitrypsin Congenital Deficiency pipeline products by clinical trial stages including both early and late stage development - phase 3 clinical trials, phase 2 clinical trials, phase 1 clinical trials, preclinical research, and discovery stage.

Alpha- Antitrypsin Deficiency Pipeline Review, H1 2019 - ResearchAndMarkets.com

Retrieved on: 
Thursday, April 25, 2019
Health, Pharmaceutical, Alpha-1 antitrypsin

The "Alpha- Antitrypsin Deficiency - Pipeline Review, H1 2019" drug pipelines has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Alpha- Antitrypsin Deficiency - Pipeline Review, H1 2019" drug pipelines has been added to ResearchAndMarkets.com's offering.
  • Alpha- Antitrypsin Deficiency - Pipeline Review, H1 2019, provides comprehensive information on the therapeutics under development for Alpha- Antitrypsin Deficiency (Genetic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type.
  • The Alpha- Antitrypsin Deficiency (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Alpha- Antitrypsin Deficiency and features dormant and discontinued projects.
  • The pipeline guide reviews pipeline therapeutics for Alpha- Antitrypsin Deficiency (Genetic Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.

CSL Behring Delivers on its Continued Commitment to Alpha 1 Patients with FDA Approval of Convenient Single-Vial Dosing for ZEMAIRA® [Alpha1-Proteinase Inhibitor (Human)]

Retrieved on: 
Thursday, April 25, 2019
Acute phase proteins, Alpha-1 antitrypsin, Alpha 1, Medical specialties, Organ systems, Branches of biology, Alpha 1-antitrypsin deficiency, Elastase

This approval is significant for the Alpha 1 community as ZEMAIRA was previously available only in a 1-gram vial.

Key Points: 
  • This approval is significant for the Alpha 1 community as ZEMAIRA was previously available only in a 1-gram vial.
  • "Many Alpha 1 patients already experience the burden of weekly infusions," said Dr. Robert Sandhaus, MD, PhD, FCCP, Clinical Director, Alpha 1 Foundation and Medical Director, AlphaNet.
  • ZEMAIRA, a natural Alpha 1 protein derived from human plasma, has been proven to raise and maintain serum levels of alpha 1 antitrypsin protein (AAT) in patients.
  • Alpha1-Proteinase Inhibitor (Human), ZEMAIRA is indicated to raise the plasma level of alpha1-proteinase inhibitor (A1-PI) in patients with A1-PI deficiency and related emphysema.

Global Plasma Fractionation Markets, 2019-2024 - Increasing Focus of Plasma Fractionators Towards Manufacturing and Developing Recombinant Factors

Retrieved on: 
Monday, April 8, 2019
Branches of biology, Medical specialties, Anatomy, Blood, Transfusion medicine, Hematology, Blood plasma fractionation, Fractionation, Blood plasma, Plasma, Antibody, Alpha-1 antitrypsin

The global plasma fractionation market is projected to reach USD 34.9 billion by 2024 from USD 25.4 billion in 2019, at a CAGR of 6.6% during the forecast period.

Key Points: 
  • The global plasma fractionation market is projected to reach USD 34.9 billion by 2024 from USD 25.4 billion in 2019, at a CAGR of 6.6% during the forecast period.
  • Growth in the plasma fractionation market can largely be attributed to factors such as the growing use of immunoglobulins in various therapeutic areas, increase in plasma collection (and the number of plasma collection centers), and the increasing use of alpha-1-antitrypsin.
  • By product, immunoglobulin market is expected to hold the largest share of the plasma fractionation market in 2019.
  • The immunoglobulin segment is expected to dominate the plasma fractionation market during the forecast period.

Global Alpha-1 Antitrypsin Drugs Market 2018-2022 - One Driver Influencing this Market is the Improving Diagnosis of Alpha-1 Antitrypsin Deficiency

Retrieved on: 
Tuesday, August 28, 2018
Acute phase proteins, Medicine, Alpha-1 antitrypsin, Hepatology, Anatomy, Alpha 1-antitrypsin deficiency, Alpha 1, Clinical medicine, Trypsin

The "Global Alpha-1 Antitrypsin Drugs Market 2018-2022" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Global Alpha-1 Antitrypsin Drugs Market 2018-2022" report has been added to ResearchAndMarkets.com's offering.
  • The Global Alpha-1 Antitrypsin Drugs Market to grow at a CAGR of 10.64% during the period 2018-2022.
  • The report, Alpha-1 Antitrypsin Drugs Market 2018-2022, has been prepared based on an in-depth market analysis with inputs from industry experts.
  • According to the report, one driver influencing this market is the improving diagnosis of alpha-1 antitrypsin deficiency.