Alpha-1 antitrypsin

Dicerna Announces Interim Results From Phase 1 Trial of Belcesiran for Treatment of Alpha-1 Antitrypsin Deficiency-Associated Liver Disease

Retrieved on: 
Wednesday, July 21, 2021
Biotechnology, Health, Pharmaceutical, Clinical trials, Oncology, Anatomy, Acute-phase proteins, Branches of biology, Alpha-1 antitrypsin, Medical specialties, Aat, Serpin, Alpha 1, Liver disease, Alpha-1 antitrypsin deficiency, Alpha-1 Antitrypsin Deficiency and Alpha-1 Antitrypsin Deficiency-Associated Liver Disease (AATLD), Belcesiran, RNAi and Dicerna’s GalXC™ RNAi Platform, Dicerna Pharmaceuticals, Inc., ALPHA-1 ANTITRYPSIN DEFICIENCY AND ALPHA-1 ANTITRYPSIN DEFICIENCY-ASSOCIATED LIVER DISEASE (AATLD), BELCESIRAN, RNAI AND DICERNA’S GALXC™ RNAI PLATFORM, DICERNA PHARMACEUTICALS, INC.

Data from this interim analysis showed dose-dependent reductions in serum alpha-1 antitrypsin (AAT) with administration of a single dose of belcesiran.

Key Points: 
  • Data from this interim analysis showed dose-dependent reductions in serum alpha-1 antitrypsin (AAT) with administration of a single dose of belcesiran.
  • The primary treatment evaluation period for the final dose cohort (12.0 mg/kg) of belcesiran in the Phase 1 trial is ongoing.
  • Alpha-1 antitrypsin (AAT) deficiency is a rare genetic condition caused by mutations in the SERPINA1 gene that results in disease of the liver and lungs.
  • Belcesiran is a clinical-stage, subcutaneously administered, investigational GalXC RNAi therapy targeting alpha-1 antitrypsin (AAT) that is in development for the treatment of AAT deficiency-associated liver disease (AATLD).

Optime Care’s Patient-First Strategy Helps Physicians Optimize Care for Rare and Orphan Disease Patients with Pulmonary Issues During COVID-19 Pandemic

Retrieved on: 
Wednesday, June 9, 2021
Technology, Nursing, Infectious diseases, Hospitals, Genetics, Practice Management, Managed care, Pharmaceutical, Health, Data management, Anatomy, Medical specialties, Organs anatomy), Hepatology, Acute-phase proteins, Lung disorders, Alcohol and health, Alpha-1 antitrypsin, Alpha 1, Liver disease

This is especially important for patients with Alpha-1 Antitrypsin Deficiency (Alpha-1), a genetic condition that may result in serious lung disease in adults and/or liver disease at any age.

Key Points: 
  • This is especially important for patients with Alpha-1 Antitrypsin Deficiency (Alpha-1), a genetic condition that may result in serious lung disease in adults and/or liver disease at any age.
  • Optime Cares patient-first approach offers patients a proactive, process-driven telehealth program which mitigates the transmission of infection, such as coronavirus, for these vulnerable individuals.
  • This focus addresses all variables around collecting data, while maintaining frequent communication with patients and their families to ensure compliance and positive outcomes.
  • Listen to Optime Cares Rare Voices podcast episode with Dr. Miskoff here .

Global Alpha 1 Antitrypsin Deficiency Treatment Market Report 2021-2027 - ResearchAndMarkets.com

Retrieved on: 
Tuesday, April 20, 2021
Health, Pharmaceutical, Alpha-1 antitrypsin, Compound annual growth rate, Draft:ByteBridge.io

b'The "Alpha 1 Antitrypsin Deficiency Treatment - Global Market Trajectory & Analytics" report has been added to ResearchAndMarkets.com\'s offering.\nAmid the COVID-19 crisis, the global market for Alpha 1 Antitrypsin Deficiency Treatment estimated at US$1.6 Billion in the year 2020, is projected to reach a revised size of US$2.9 Billion by 2027, growing at a CAGR of 8.9% over the analysis period 2020-2027.\nAugmentation Therapy, one of the segments analyzed in the report, is projected to record a 9.7% CAGR and reach US$1.4 Billion by the end of the analysis period.

Key Points: 
  • b'The "Alpha 1 Antitrypsin Deficiency Treatment - Global Market Trajectory & Analytics" report has been added to ResearchAndMarkets.com\'s offering.\nAmid the COVID-19 crisis, the global market for Alpha 1 Antitrypsin Deficiency Treatment estimated at US$1.6 Billion in the year 2020, is projected to reach a revised size of US$2.9 Billion by 2027, growing at a CAGR of 8.9% over the analysis period 2020-2027.\nAugmentation Therapy, one of the segments analyzed in the report, is projected to record a 9.7% CAGR and reach US$1.4 Billion by the end of the analysis period.
  • China, the world\'s second largest economy, is forecast to reach a projected market size of US$493.1 Million by the year 2027 trailing a CAGR of 8.3% over the analysis period 2020 to 2027.
  • Among the other noteworthy geographic markets are Japan and Canada, each forecast to grow at 8.4% and 7.2% respectively over the 2020-2027 period.
  • Led by countries such as Australia, India, and South Korea, the market in Asia-Pacific is forecast to reach US$328.4 Million by the year 2027.\n'

Global Alpha-1 Antitrypsin Drugs Market Trajectory & Analytics Report 2021: Market to Reach $2 Billion by 2027 - U.S. Market is Estimated at $319.3 Million, While China is Forecast to Grow at 9% CAGR - ResearchAndMarkets.com

Retrieved on: 
Monday, April 19, 2021
Health, Pharmaceutical, Actuarial science, Compound annual growth rate, Mathematical finance, Alpha-1 antitrypsin

b'The "Alpha-1 Antitrypsin Drugs - Global Market Trajectory & Analytics" report has been added to ResearchAndMarkets.com\'s offering.\nAmid the COVID-19 crisis, the global market for Alpha-1 Antitrypsin Drugs estimated at US$1.1 Billion in the year 2020, is projected to reach a revised size of US$2 Billion by 2027, growing at a CAGR of 9.6% over the period 2020-2027.\nThe U.S. Market is Estimated at $319.3 Million, While China is Forecast to Grow at 9% CAGR\nThe Alpha-1 Antitrypsin Drugs market in the U.S. is estimated at US$319.3 Million in the year 2020.

Key Points: 
  • b'The "Alpha-1 Antitrypsin Drugs - Global Market Trajectory & Analytics" report has been added to ResearchAndMarkets.com\'s offering.\nAmid the COVID-19 crisis, the global market for Alpha-1 Antitrypsin Drugs estimated at US$1.1 Billion in the year 2020, is projected to reach a revised size of US$2 Billion by 2027, growing at a CAGR of 9.6% over the period 2020-2027.\nThe U.S. Market is Estimated at $319.3 Million, While China is Forecast to Grow at 9% CAGR\nThe Alpha-1 Antitrypsin Drugs market in the U.S. is estimated at US$319.3 Million in the year 2020.
  • China, the world\'s second largest economy, is forecast to reach a projected market size of US$354.1 Million by the year 2027 trailing a CAGR of 9% over the analysis period 2020 to 2027.\nAmong the other noteworthy geographic markets are Japan and Canada, each forecast to grow at 8.9% and 7.8% respectively over the 2020-2027 period.
  • Within Europe, Germany is forecast to grow at approximately 7.8% CAGR.\n'

Global Alpha 1 Antitrypsin Deficiency Treatment Market Report 2020 with COVID-19 Impact Insights

Retrieved on: 
Tuesday, December 22, 2020
Medical specialties, Anatomy, Acute-phase proteins, Alpha-1 antitrypsin deficiency, Hepatology, Serpinopathies, Organ systems, Alpha 1, Alpha-1 antitrypsin

The global alpha 1 antitrypsin deficiency treatment market grew at a CAGR of around 9% during 2014-2019.

Key Points: 
  • The global alpha 1 antitrypsin deficiency treatment market grew at a CAGR of around 9% during 2014-2019.
  • Looking forward, the global alpha 1 antitrypsin deficiency treatment market to continue its strong growth during the next five years.
  • What has been the impact of COVID-19 on the global alpha 1 antitrypsin deficiency treatment market?
  • What is the structure of the global alpha 1 antitrypsin deficiency treatment market and who are the key players?

Alpha 1 Antitrypsin Deficiency Treatment Market - Global Industry Trends, Share, Size, Growth, Opportunity and Forecast 2020-2025 - ResearchAndMarkets.com

Retrieved on: 
Wednesday, December 16, 2020
Health, Pharmaceutical, Anatomy, Medical specialties, Acute-phase proteins, Peptidase, Alpha-1 antitrypsin deficiency, Hepatology, Serpinopathies, Alpha 1, Alpha-1 antitrypsin, Organ systems, Trypsin

The "Alpha 1 Antitrypsin Deficiency Treatment Market: Global Industry Trends, Share, Size, Growth, Opportunity and Forecast 2020-2025" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Alpha 1 Antitrypsin Deficiency Treatment Market: Global Industry Trends, Share, Size, Growth, Opportunity and Forecast 2020-2025" report has been added to ResearchAndMarkets.com's offering.
  • The global alpha 1 antitrypsin deficiency treatment market grew at a CAGR of around 9% during 2014-2019.
  • Looking forward, the global alpha 1 antitrypsin deficiency treatment market to continue its strong growth during the next five years.
  • What is the structure of the global alpha 1 antitrypsin deficiency treatment market and who are the key players?

Intellia Therapeutics Achieves Normal Human Alpha-1 Antitrypsin Protein Levels in Non-Human Primates Through Targeted Gene Insertion for the Treatment of AAT Deficiency

Retrieved on: 
Saturday, December 12, 2020
Branches of biology, Biology, Medical specialties, Alpha-1 antitrypsin deficiency, Hepatology, Serpinopathies, Genome editing, Biotechnology, Genetic engineering, Alpha-1 antitrypsin, Serpin, CRISPR

In parallel with advancing to the clinic treatments for other severe diseases, we will continue preclinical studies that further validate our wholly owned, CRISPR-based AAT deficiency treatment strategies for achieving normal AAT protein levels.

Key Points: 
  • In parallel with advancing to the clinic treatments for other severe diseases, we will continue preclinical studies that further validate our wholly owned, CRISPR-based AAT deficiency treatment strategies for achieving normal AAT protein levels.
  • Intellia is advancing multiple genome editing strategies that may treat both lung and liver manifestations of AAT deficiency (AATD), which occur due to mutations in the SERPINA1 gene.
  • The normal human AAT protein levels Intellia achieved following targeted insertion of the human SERPINA1 gene remained stable through 11 weeks in an ongoing NHP study.
  • One of the editing strategies Intellia is studying as a potential single-course AATD treatment is using the companys SERPINA1 gene insertion approach to restore normal human AAT protein levels.

Treatments for rare diseases at risk, due to the decline of plasma donations

Retrieved on: 
Friday, December 11, 2020
Medical specialties, Clinical medicine, Anatomy, Blood products, Transfusion medicine, Complement deficiency, Serpinopathies, Blood, Alpha-1 antitrypsin, Plasmapheresis, Angioedema, Hereditary angioedema

These therapies are often the only treatments available for people with primary immune deficiencies, bleeding disorders, Alpha-1 Antitrypsin deficiency, hereditary angioedema, and certain neurological conditions.

Key Points: 
  • These therapies are often the only treatments available for people with primary immune deficiencies, bleeding disorders, Alpha-1 Antitrypsin deficiency, hereditary angioedema, and certain neurological conditions.
  • Roundtable participants stressed the continued urgent need for plasma donations.
  • This sharp decline in plasma collections could cause more significant challenges in coming months.
  • PPTA also administers standards and programs that help ensure the quality and safety of plasma protein therapies, donors and patients.

Global Alpha-1 Antitrypsin Deficiency (A1AD) Pipeline Review 2020: Therapeutic Development & Assessment, Companies Involved, Drug Profiles, Dormant Projects

Retrieved on: 
Thursday, October 15, 2020
Anatomy, Medical specialties, Alpha-1 antitrypsin deficiency, Hepatology, RTT, Serpinopathies, Alpha 1, Alpha-1 antitrypsin, Organ systems

Alpha-1 Antitrypsin Deficiency (A1AD) - Pipeline Review, H2 2020, provides comprehensive information on the therapeutics under development for Alpha-1 Antitrypsin Deficiency (A1AD) (Other Diseases), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type.

Key Points: 
  • Alpha-1 Antitrypsin Deficiency (A1AD) - Pipeline Review, H2 2020, provides comprehensive information on the therapeutics under development for Alpha-1 Antitrypsin Deficiency (A1AD) (Other Diseases), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type.
  • The Alpha-1 Antitrypsin Deficiency (A1AD) (Other Diseases) pipeline guide also reviews the key players involved in therapeutic development for Alpha-1 Antitrypsin Deficiency (A1AD) and features dormant and discontinued projects.
  • The pipeline guide reviews pipeline therapeutics for Alpha-1 Antitrypsin Deficiency (A1AD) (Other Diseases) by companies and universities/research institutes based on information derived from company and industry-specific sources.
  • Formulate corrective measures for pipeline projects by understanding Alpha-1 Antitrypsin Deficiency (A1AD) (Other Diseases) pipeline depth and focus of Indication therapeutics.

Takeda and Arrowhead Collaborate to Co-Develop and Co-Commercialize ARO-AAT for Alpha-1 Antitrypsin-Associated Liver Disease

Retrieved on: 
Thursday, October 8, 2020
Pharmaceutical, Health, Clinical trials, Other Health, Medical specialties, RTT, Anatomy, Hepatology, Acute phase proteins, Alpha-1 antitrypsin deficiency, Serpinopathies, Organ systems, Alpha-1 antitrypsin, Liver disease, Liver, Arrowhead Pharmaceuticals, Alpha-1 Antitrypsin-Associated Liver Disease, ARO-AAT, Takeda Pharmaceutical Company Limited, Arrowhead Pharmaceuticals

We are excited to collaborate with Arrowhead to bring forward this exciting late-stage liver asset for the Alpha-1 community as part of our growing GI portfolio.

Key Points: 
  • We are excited to collaborate with Arrowhead to bring forward this exciting late-stage liver asset for the Alpha-1 community as part of our growing GI portfolio.
  • Alpha-1 Antitrypsin-Associated Deficiency (AATD) is a rare genetic disorder associated with liver disease in children and adults and pulmonary disease in adults.
  • Individuals with the homozygous PiZZ genotype have severe deficiency of functional AAT leading to pulmonary disease and liver disease.
  • However, augmentation therapy does nothing to treat liver disease, and there is no specific therapy for hepatic manifestations.