Alpha 1 Antitrypsin Deficiency News and Research

RSS

Nature publishes Sangamo's gene correction strategy for A1AT deficiency

Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced the publication of a preclinical study demonstrating highly specific, functional correction of the alpha 1-antitrypsin (A1AT) gene defect in patient-derived induced pluripotent stem cells (iPSCs) using zinc finger nucleases.

14 Oct 2011

Isis designates ISIS-AATRx candidate in collaboration with GSK

Isis Pharmaceuticals, Inc. announced today that it has designated the second development candidate, ISIS-AATRx, in its collaboration with GlaxoSmithKline

12 Oct 2011

AGTC receives FFB grant to evaluate gene therapy treatment for X-linked Retinoschisis

Applied Genetic Technologies Corporation, a privately-held, clinical stage biotechnology company developing novel systems to deliver human therapeutics, announces that it has received a grant of $1.5 million from the Foundation Fighting Blindness to fund a pre-clinical trial evaluating the safety and efficacy of a gene therapy treatment for X-linked Retinoschisis (XLRS), a genetic eye disease affecting over 35,000 patients in the US and Europe.

22 Sep 2011

PPTA applauds introduction of 'Preserving Access to Orphan Drugs Act'

The Plasma Protein Therapeutics Association (PPTA) applauds the introduction of bipartisan legislation, the Preserving Access to Orphan Drugs Act (H.R. 2672) and (S. 1423), which will safeguard the development of drugs and therapies that treat patients with rare diseases by eliminating barriers to innovation.

30 Jul 2011

Intrexon, Halozyme partner to develop subcutaneous alpha 1-antitrypsin for A1AT deficiency

Halozyme Therapeutics, Inc. and Intrexon Corporation, today announced the signing of a worldwide exclusive licensing agreement for the use of rHuPH20 in the development of a subcutaneous injectable formulation of Intrexon Corporation's recombinant human alpha 1-antitrypsin (rHuA1AT).

7 Jun 2011

PPTA offers new DVD on plasma donation

Anyone curious about what to expect as a plasma donor and how plasma contributes to lifesaving medicines can now learn about the process thanks to a new DVD from the Plasma Protein Therapeutics Association.

29 Jan 2011

Breakthrough in understanding mechanisms behind hereditary alpha-1-antitrypsin deficiency

Researchers from the Royal College of Surgeons in Ireland (RCSI) and Beaumont Hospital have made a breakthrough in understanding the mechanisms behind the most severe form of hereditary emphysema and how protein treatments can improve the condition. The findings of this study may also lead to new treatments for patients with smoker's emphysema.

24 Jan 2011

Neurology professor receives MFRF's 'See the Light' award for Tay-Sachs research

Miguel Sena-Esteves, PhD, associate professor of neurology at the University of Massachusetts Medical School, was presented with the annual "See the Light" award from the Mathew Forbes Romer Foundation in recognition of his work for leading the fight against children's genetic diseases of the brain.

12 Jan 2011

Investigators receive two Alpha-1 Foundation grants for research on lung disorder

The Alpha-1 Foundation announced today that it has awarded two research grants to investigators whose research will contribute to our understanding of the causes and mechanisms that give rise to lung disease.

6 Jan 2011

Science and precedent do not support biosimilar approach for plasma protein therapies

The current science and experience does not support the safety and efficaciousness of a biosimilar pathway for plasma protein therapies. At a public hearing last week, the Plasma Protein Therapeutics Association (PPTA) urged the United States Food and Drug Administration (FDA) to make patient safety its top priority by adopting a global approach in its evaluation of plasma protein therapies for the biosimilars process.

15 Nov 2010

Talecris announces publication of A1PI studies for AAT deficiency in Respiratory Research journal

Talecris Biotherapeutics announced today the publication of combined data from two studies demonstrating that augmentation therapy with Alpha(1)-Proteinase Inhibitor (Human) (A1PI) significantly reduces lung tissue loss in patients with emphysema related to Alpha(1)-antitrypsin (AAT) deficiency.

15 Nov 2010

New iPSC cell lines could help in treating patients with lung diseases

A team of researchers from Boston University's Center for Regenerative Medicine and the Pulmonary Center have generated 100 new lines of human induced pluripotent stem cells (iPSC) from individuals with lung diseases, including cystic fibrosis and emphysema. The new stem cell lines could possibly lead to new treatments for these debilitating diseases.

29 Oct 2010

Baxter announces launch of GLASSIA in the U.S.

Baxter International Inc. today announced the commercial launch of GLASSIATM [Alpha1-Proteinase Inhibitor (Human)] in the United States. GLASSIATM is the first available ready-to-use liquid alpha1-proteinase inhibitor (Alpha1-PI) and is indicated as a chronic augmentation and maintenance therapy in adults with emphysema due to congenital deficiency of alpha-1 antitrypsin (AAT), an under-diagnosed hereditary condition characterized by a low level of alpha-1 protein in the blood.

From Baxter International Inc. 26 Oct 2010

BioRx receives limited distribution rights from Baxter for AATD drug GLASSIA

BioRx, a specialty pharmacy company, has received limited distribution rights from Baxter International Inc. for a new drug, GLASSIA™ [Alpha 1-Proteinase Inhibitor (Human)], the first and only liquid, ready-to-use treatment for alpha-1 antitrypsin deficiency (AATD) in the United States. AATD is a genetic disorder that causes low levels of alpha 1-antitrypsin, resulting in emphysema. AATD is also referred to as "genetic" or "congenital" emphysema.

From Baxter International Inc. 13 Oct 2010

Talecris Biotherapeutics announces 2010 eALTA award recipients

Talecris Biotherapeutics GmbH announced yesterday the recipients of the 2010 European alpha1-antitrypsin Laurell's Training Awards (eALTA). The annual awards, sponsored by Talecris, provide two fellowships of 50,000 euros to young investigators whose research aims to enhance the understanding and treatment of alpha1-antitrypsin deficiency (AAT deficiency).

21 Sep 2010

FDA grants AGTC $1M for Phase II human clinical trial in alpha-1 antitrypsin deficiency

Applied Genetic Technologies Corporation, a privately-held, clinical stage biotechnology company developing novel systems to deliver human therapeutics, announces that it has received a grant of $1 million from the Food and Drug Administration (FDA Orphan Drugs Program) to fund a Phase II Human Clinical Trial evaluating the safety and efficacy of a treatment for Alpha 1 Antitrypsin Deficiency (AAT), a genetic disease known to result in serious lung and/or liver disease.

17 Sep 2010

PPTA supports CMS' proposal for hospital outpatient reimbursement for plasma protein therapies

The Plasma Protein Therapeutics Association (PPTA) recommends that the Centers for Medicare and Medicaid Services (CMS) finalize its Proposed Rule regarding reimbursement to better ensure patient access to vital medicines in the hospital outpatient setting.

8 Sep 2010

Californians recognized for contributing to plasma protein lifesaving therapies

This month, Californians are recognized for their outstanding contributions to lifesaving therapies that treat critically ill individuals and for their donations of the blood component, plasma, that makes the creation of these unique therapies possible.

3 Sep 2010

Kamada, Baxter enter distribution and manufacturing agreement for Glassia Alpha-1 Antitrypsin Product

Kamada, Ltd., a biopharmaceutical company engaged in development, production and marketing of specialty life-saving therapeutics and Baxter International Inc., a global, diversified healthcare company announced today that they have entered into an exclusive distribution and manufacturing agreement for Kamada's liquid, ready to use, intravenous Alpha-1 Antitrypsin Product for the treatment of hereditary Alpha-1 Antitrypsin Deficiency approved by the USFDA on July 1st, 2010 for marketing under the trade name "Glassia™".

From Baxter International Inc. 25 Aug 2010

Baxter announces definitive agreement with Kamada for GLASSIA alpha1-proteinase inhibitor

Baxter International Inc. today announced a definitive agreement with Kamada Ltd. for exclusive commercial rights to GLASSIA™ [Alpha 1-Proteinase Inhibitor (Human)], the first and only liquid alpha1-proteinase inhibitor, in the United States, Australia, New Zealand and Canada.

From Baxter International Inc. 24 Aug 2010