Leber Congenital Amaurosis News and Research

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Auxilium Pharmaceuticals plans to merge with QLT

Auxilium Pharmaceuticals, Inc., a fully integrated specialty biopharmaceutical company, and QLT Inc., a Canadian-based biotechnology company focused on developing innovative orphan ophthalmology products, today announced that they have entered into a definitive agreement under which Auxilium plans to merge with QLT.

26 Jun 2014

Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene expression

Successful gene therapy is based on the effective delivery and maintained expression of healthy copies of a gene into tissues of individuals with a disease-associated genetic mutation.

16 Nov 2013

Investigators report complete catalog of genes expressed in retina

Investigators at Massachusetts Eye and Ear and Harvard Medical School have published the most thorough description of gene expression in the human retina reported to date. In a study published today in the journal BMC Genomics, Drs. Michael Farkas, Eric Pierce and colleagues in the Ocular Genomics Institute at Mass. Eye and Ear reported a complete catalog of the genes expressed in the retina.

20 Jul 2013

Gene therapy does not slow or halt progression of cell loss in Leber congenital amaurosis

Independent clinical trials, including one conducted at the Scheie Eye Institute at the Perelman School of Medicine, have reported safety and efficacy for Leber congenital amaurosis (LCA), a congenital form of blindness caused by mutations in a gene (RPE65) required for recycling vitamin A in the retina. Inherited retinal degenerative diseases were previously considered untreatable and incurable.

23 Jan 2013

Gene therapy improves vision but does not halt progression of cell loss

22 Jan 2013

Gene discovery made for infant-onset blindness

Researchers have discovered that mutations in the NMNAT1 gene encoding nicotinamide mononucleotide adenylyltransferase 1 are present in patients with Leber congenital amaurosis.

1 Aug 2012

Scientists identify new gene responsible for genetic form of blindness in newborns

One of the mysteries of blindness has been solved. A team of international scientists in collaboration with the Research Institute of the McGill University Health Centre (RI MUHC) identified a new gene responsible for Leber Congenital Amaurosis (LCA), a devastating genetic form of blindness in newborns.

30 Jul 2012

Researchers isolate elusive human gene that causes NMNAT1-related Leber congenital amaurosis

Researchers from the Massachusetts Eye and Ear Infirmary, The Children's Hospital of Philadelphia, Loyola University Chicago Health Sciences Division and their collaborators have isolated an elusive human gene that causes a common form of Leber congenital amaurosis (LCA), a relatively rare but devastating form of early-onset blindness. The new LCA gene is called NMNAT1.

30 Jul 2012

Mass. Eye and Ear announces recipients of 2012 Curing Kids Fund grants

The Mass. Eye and Ear Curing Kids fund has awarded grants for research projects that aim to help the brain locate sound, identify genes that cause congenital blindness, cure deafness through hair cell regeneration, and improve treatment for childhood eye cancer.

16 May 2012

Gene therapy succeeds in the second blind eye for three patients

Three people have now have reported a dramatic improvement in vision after having gene therapy in both eyes after having lost their eyesight as children.

9 Feb 2012

Readministration of gene therapy improves vision in three patients with LCA

Gene therapy for congenital blindness has taken another step forward, as researchers further improved vision in three adult patients previously treated in one eye.

9 Feb 2012

Global gene therapy market to reach $300 million by 2015

The global gene therapy market is expected to grow to over $300 million by 2015, according to a new report available on companiesandmarkets.com. The gene therapy market will grow further driven by the human genome project, the increasing incidence of cancer and other critical diseases.

6 Jun 2011

QLT fourth quarter revenues decrease 7.3% to $10.0 million

QLT Inc. today reported financial results for the fourth quarter ended December 31, 2010 and full year 2010 as well as issued its guidance for 2011. Unless specified otherwise, all amounts are in U.S. dollars and in accordance with U.S. GAAP.

1 Mar 2011

EMA grants QLT positive opinions for Orphan Drug Designation of QLT091001 to treat inherited blindness

QLT Inc. today announced that QLT091001, an oral synthetic retinoid, has received positive opinions for two distinct Orphan Drug Designations by the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) for the treatment of the inherited retinal degenerative diseases, Leber Congenital Amaurosis (LCA) and Retinitis Pigmentosa (RP).

10 Feb 2011

QLT's oral synthetic retinoid granted orphan drug designation for treatment of Leber Congenital Amaurosis

QLT Inc. today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for QLT091001, its oral synthetic retinoid, for the treatment of Leber Congenital Amaurosis (LCA). LCA is an inherited progressive retinal degenerative disease that leads to retinal dysfunction and visual impairment beginning at birth. QLT091001 is an orally administered synthetic retinoid replacement for 11-cis-retinal, which is a key biochemical component of the visual retinoid cycle.

8 Dec 2010

FDA grants QLT's QLT091001 orphan drug designation for treatment of Retinitis Pigmentosa

QLT Inc. today announced that QLT091001, the Company's oral synthetic retinoid, has been granted orphan drug designation for the treatment of Retinitis Pigmentosa (RP) from the U.S. Food and Drug Administration (FDA). This follows the FDA grant of orphan drug designation for QLT091001 for the treatment of Leber Congenital Amaurosis (LCA) also on December 7, 2010.

8 Dec 2010

Children's Hospital pediatric immunologist collaborates with European gene therapy researchers to study WAS syndrome

A pediatric immunologist at The Children's Hospital of Philadelphia collaborated with European gene therapy researchers who achieved marked clinical improvements in two young children with Wiskott-Aldrich syndrome, a very rare but often severe immunodeficiency disorder.

16 Nov 2010

QLT third quarter sales decreases 12.9% to $20.5 million

QLT Inc. today reported financial results for the third quarter ended September 30, 2010. Unless specified otherwise, all amounts are in U.S. dollars and in accordance with U.S. GAAP.

3 Nov 2010

QLT expands QLT091001 Phase 1b LCA study to include patients with Retinitis Pigmentosa

QLT Inc. today announced expansion of the Phase 1b clinical proof-of-concept study of QLT091001 in the treatment of Leber Congenital Amaurosis (LCA) to include patients with Retinitis Pigmentosa (RP), a set of hereditary retinal diseases demonstrating clinical features similar to LCA.

28 Oct 2010

AGTC receives grant to evaluate Phase II Human Clinical Trial for LCA retinal disease

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). The grant will fund a Phase II Human Clinical Trial evaluating the safety and efficacy of a treatment for Leber congenital amaurosis (LCA), a genetic retinal disease known to cause blindness at an early age.

14 Sep 2010