Retinitis Pigmentosa News and Research RSS Feed - Retinitis Pigmentosa News and Research

Retinitis pigmentosa (RP) is the name given to a group of inherited eye diseases that affect the retina (the light-sensitive part of the eye). RP causes the breakdown of photoreceptor cells (cells in the retina that detect light). Photoreceptor cells capture and process light helping us to see. As these cells breakdown and die, patients experience progressive vision loss. The most common feature of all forms of RP is a gradual breakdown of rods (retinal cells that detect dim light) and cones (retinal cells that detect light and color). Most forms of RP first cause the breakdown of rod cells. These forms of RP, sometimes called rod-cone dystrophy, usually begin with night blindness. Night blindness is somewhat like the experience normally sighted individuals encounter when entering a dark movie theatre on a bright, sunny day. However, patients with RP cannot adjust well to dark and dimly lit environments.
CUMC, Iowa scientists use CRISPR to repair genetic mutation responsible for retinitis pigmentosa

CUMC, Iowa scientists use CRISPR to repair genetic mutation responsible for retinitis pigmentosa

Columbia University Medical Center and University of Iowa scientists have used a new gene-editing technology called CRISPR, to repair a genetic mutation responsible for retinitis pigmentosa (RP), an inherited condition that causes the retina to degrade and leads to blindness in at least 1.5 million cases worldwide. [More]
Shire announces resubmission of lifitegrast NDA to FDA for treatment of dry eye disease in adults

Shire announces resubmission of lifitegrast NDA to FDA for treatment of dry eye disease in adults

Shire plc today announced it has resubmitted the New Drug Application (NDA) to the U.S. Food and Drug Administration for its investigational candidate, lifitegrast, for the treatment of signs and symptoms of dry eye disease in adults. Shire resubmitted the NDA in response to the complete response letter (CRL) the company received from the FDA on October 16, 2015. [More]
Incidence of childhood myopia has more than doubled over last 50 years among American children

Incidence of childhood myopia has more than doubled over last 50 years among American children

The largest study of childhood eye diseases ever undertaken in the U.S. confirms that the incidence of childhood myopia among American children has more than doubled over the last 50 years. The findings echo a troubling trend among adults and children in Asia, where 90 percent or more of the population have been diagnosed with myopia, up from 10 to 20 percent 60 years ago. [More]
New gene editing technique could hinder retinal degeneration in rats with inherited blindness

New gene editing technique could hinder retinal degeneration in rats with inherited blindness

A new technique that has the potential to treat inherited diseases by removing genetic defects has been shown for the first time to hinder retinal degeneration in rats with a type of inherited blindness, according to a Cedars-Sinai study. [More]
Dompé’s investigational biotech molecule receives orphan drug designation for treatment of neurotrophic keratitis

Dompé’s investigational biotech molecule receives orphan drug designation for treatment of neurotrophic keratitis

The Dompé biopharmaceutical company announced today that the Committee for Orphan Medicinal Products of the European Medicines Agency (EMA) has officially designated recombinant human Nerve Growth Factor (rhNGF) - the investigational biotech molecule developed by Dompé based on research by Nobel Laureate Rita Levi Montalcini - as an orphan drug for the treatment of neurotrophic keratitis. [More]
Nightstar announces $35 million Series B financing round

Nightstar announces $35 million Series B financing round

NightstaRx Ltd, a biopharmaceutical company specialising in developing gene therapies for inherited retinal dystrophies, announces a $35 million Series B financing round. The round was led by New Enterprise Associates, one of the world's leading venture capital firms. [More]
Saint Louis University ophthalmologist offers tips to manage night vision issues

Saint Louis University ophthalmologist offers tips to manage night vision issues

Owls and cats are at an advantage as the days get darker, but humans may notice their vision takes a hit during their evening commute home as daylight hours shrink. [More]
Gene therapy preserves vision in canine model of late-stage retinitis pigmentosa

Gene therapy preserves vision in canine model of late-stage retinitis pigmentosa

Gene therapy preserved vision in a study involving dogs with naturally occurring, late-stage retinitis pigmentosa, according to research funded by the National Eye Institute (NEI), part of the National Institutes of Health. The findings contribute to the groundwork needed to move gene therapy forward into clinical trials for people with the blinding eye disorder, for which there is currently no cure. [More]
Penn researchers show that canine X-linked retinitis pigmentosa can be cured over the long term

Penn researchers show that canine X-linked retinitis pigmentosa can be cured over the long term

Three years ago, a team from the University of Pennsylvania announced that they had cured X-linked retinitis pigmentosa, a blinding retinal disease, in dogs. Now they've shown that they can cure the canine disease over the long term, even when the treatment is given after half or more of the affected photoreceptor cells have been destroyed. [More]
Lonza, Benitec partner to develop scalable manufacturing process for AAV-based viral gene therapies

Lonza, Benitec partner to develop scalable manufacturing process for AAV-based viral gene therapies

Lonza Houston, Inc., a global leader in biological, cell and viral gene therapy manufacturing, and Benitec Biopharma, a clinical stage biotechnology company commercializing a patented gene-silencing technology, DNA-directed RNA interference (ddRNAi), today announced that they have entered into a Manufacturing Services Agreement to develop a scalable manufacturing process for Benitec's ddRNAi-based, Adeno-Associated Virus (AAV)-delivered products intended for therapeutic use in humans. [More]
Benitec Biopharma initiates new site for ongoing Phase 1/2a TT-034 trial for hepatitis C

Benitec Biopharma initiates new site for ongoing Phase 1/2a TT-034 trial for hepatitis C

Benitec Biopharma Limited, a clinical-stage biotechnology company developing innovative therapeutics based on its gene-silencing technology, DNA-directed RNA interference (ddRNAi), is pleased to announce it has initiated a new site for its ongoing Phase 1/2a TT-034 trial at the Methodist Health System Clinical Research Institute in Dallas, Texas. [More]
Benitec Biopharma announces closing of U.S. initial public offering of ADSs

Benitec Biopharma announces closing of U.S. initial public offering of ADSs

Benitec Biopharma Limited, a clinical-stage biotechnology company, is pleased to announce the closing of its U.S. initial public offering of 1,500,000 American Depositary Shares (ADSs), representing 30,000,000 fully paid ordinary shares of Benitec, together with warrants to purchase 500,000 ADSs, representing 10,000,000 fully paid ordinary shares. [More]
Benitec prices initial public offering of 1,500,000 American Depositary Shares

Benitec prices initial public offering of 1,500,000 American Depositary Shares

Benitec Biopharma Limited, a clinical-stage biotechnology company, is pleased to announce the pricing of its U.S. initial public offering of 1,500,000 American Depositary Shares (ADSs), representing 30,000,000 fully paid ordinary shares of Benitec and warrants to purchase 500,000 ADSs, representing 10,000,000 fully paid ordinary shares, at a price of US$9.21 per ADS and US$0.01 per warrant. [More]
First clinical trial to test use of retinal progenitor cells for treating retinitis pigmentosa begins

First clinical trial to test use of retinal progenitor cells for treating retinitis pigmentosa begins

Participants are being enrolled in the first clinical trial that tests the use of retinal progenitor cells to treat retinitis pigmentosa, reported project director Dr. Henry Klassen of UCI's Gavin Herbert Eye Institute and Sue & Bill Gross Stem Cell Research Center. The product of stem cell research at UCI, these retinal progenitors are similar to stem cells in terms of potential regenerative properties, but they're specific to the retina. [More]
Sigma 1 receptor appears to play vital role in supporting the retina

Sigma 1 receptor appears to play vital role in supporting the retina

A receptor that is already a target for treating neurodegenerative disease also appears to play a key role in supporting the retina, scientists report. [More]
Shire acquires Foresight Biotherapeutics for $300 million

Shire acquires Foresight Biotherapeutics for $300 million

Shire plc announced today that it has acquired New York-based, privately held Foresight Biotherapeutics Inc. for $300 million. [More]
Gene therapy provides life-long protection to photoreceptor cells in animal model of retinitis pigmentosa

Gene therapy provides life-long protection to photoreceptor cells in animal model of retinitis pigmentosa

A collaboration between scientists in the UK and the USA has shown that gene therapy can give life-long protection to the light-sensitive photoreceptor cells responsible for colour vision in a mouse model of the most common inherited eye disorder. [More]
Researchers compare effectiveness of two stem cell types in treating retinal degeneration

Researchers compare effectiveness of two stem cell types in treating retinal degeneration

By growing two types of stem cells in a "3-D culture" and measuring their ability to produce retinal cells, a team lead by St. Jude Children's Research Hospital researchers has found one cell type to be better at producing retinal cells. [More]
New NEI study shows that microglia can accelerate damage wrought by blinding eye disorders

New NEI study shows that microglia can accelerate damage wrought by blinding eye disorders

Spider-like cells inside the brain, spinal cord and eye hunt for invaders, capturing and then devouring them. These cells, called microglia, often play a beneficial role by helping to clear trash and protect the central nervous system against infection. But a new study by researchers at the National Eye Institute (NEI) shows that they also accelerate damage wrought by blinding eye disorders, such as retinitis pigmentosa. [More]
Canada's first human gene therapy trial for choroideremia now underway at Royal Alexandra Hospital

Canada's first human gene therapy trial for choroideremia now underway at Royal Alexandra Hospital

Canada's first human gene therapy trial for eyes -- the replacement of a faulty gene with a healthy one -- is now underway at the Royal Alexandra Hospital to preserve and potentially restore vision for people with a genetic disorder that leaves them blind by middle age. [More]
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