According to the company, achromatopsia
results from mutations in one of several genes.
The report reviews pipeline therapeutics for Achromatopsia
by companies and universities/research institutes based on information derived from company and industry-specific sources
She believes that years down the line, research into gene therapy funded by Sight Savers may help achromatopsia
The funding will allow AGTC to continue development of its Phase 2 program in Alpha-1 Antitrypsin Deficiency (Alpha-1) and initiate full development of potential treatments for two orphan ophthalmology indications, Achromatopsia
(ACHM) and X-Linked Rentinoschisis (XLRS).
M2 PHARMA-October 20, 2016-AGTC files IND with US FDA for gene therapy product for treating achromatopsia
People who are totally colour blind, a condition called achromatopsia
, can only see things as black and white or in shades of gray.
In conjunction, AGTC has a been granted orphan drug designation from the EC and the US Food and Drug Administration (FDA) for its gene therapy product candidates for the treatment of X-linked retinoschisis and for the treatment of achromatopsia
caused by mutations in the CNGA3 and CNGB3 genes.
is a hereditary disorder of colour vision in which there is an inability to perceive colour and, consequently, to achieve satisfactory vision at high light levels.
The youngster from Kinmel Bay suffers from achromatopsia
- a rare eye condition which means she can't see in colour and is blinded by the sun.
Twins Nerys Parry and Nesta Jones and their older sibling, Rhian Thomas - all have sons with genetically inherited achromatopsia
Researchers have now pinpointed a mutation that results in achromatopsia
in these people in a gene called CNGB3.
The collaboration will focus on development of treatments for two of the more common genetic retinal diseases that cause blindness at a very early age: X-Linked Retinoschisis (XLRS) and Achromatopsia