Leber Congenital Amaurosis News and Research

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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

Scientists replace human gene to preserve photoreceptor function in retinal degeneration

The easy accessibility of the eye and the established link between specific genetic defects and ocular disorders offer hope for using gene therapy to provide long-term therapeutic benefit. Two reports in the current issue of Human Gene Therapy, a peer-reviewed journal published by Mary Ann Liebert, Inc., describe the effective replacement of a human gene to preserve photoreceptor function in a mouse model of severe retinal degeneration. The articles are available free online.

16 Aug 2010

New study holds promise for patients with LCA

A new study led by Dr. Robert Koenekoop at the Montreal Children's Hospital at the McGill University Health Centre holds promise for certain patients with Leber congenital amaurosis, an inherited retinal degenerative disease that causes significant vision loss from birth. The study, sponsored by Canadian company QLT Inc., is designed to treat patients with LCA due to genetic mutations in either retinal pigment epithelium protein 65 or lecithin:retinol acyltransferase.

27 May 2010

Interim results from QLT's Phase 1b study of QLT091001 for Leber congenital amaurosis

QLT Inc. today announced interim results from the first 3 subjects enrolled in a Phase 1b clinical proof-of-concept study of QLT091001 in the treatment of Leber congenital amaurosis (LCA), an inherited progressive retinal degenerative disease that leads to retinal dysfunction and significant visual impairment beginning at birth. QLT091001 is an orally administered synthetic retinoid replacement for 11-cis-retinal, which is a key biochemical component of visual function.

20 Apr 2010

LCA: Few possess genetic mutation necessary for positive outcome from gene therapy

Leber congenital amaurosis (LCA) is a congenital retinal dystrophy present in approximately 1 of 80,000 births. It is estimated that about 3,000 people in the United States are living with LCA and will likely become blind in their lifetimes. Recently, there has been progress in gene therapy for this condition.

7 Apr 2010

QLT commences enrollment in QLT091001 Phase Ib trial of LCA pediatric patients

QLT Inc. today announced that it has initiated enrollment in a Phase Ib trial of QLT091001 in pediatric patients with Leber Congenital Amaurosis (LCA), 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.

15 Dec 2009

Study shows gene therapy can restore vision to children with Leber congenital amaurosis

Results from a breakthrough study, conducted by researchers at The Children’s Hospital of Philadelphia (CHOP) and the University of Pennsylvania School of Medicine, show that gene therapy has restored significant vision in five children and seven adults who were previously blind.

27 Oct 2009

Remarkable vision improvements after gene therapy for inherited blindness

One year after a trio of young adults received gene therapy for an inherited form of blindness, researchers have documented that the patients are still experiencing the same level of remarkable vision improvements previously measured within weeks.

13 Aug 2009