Inherited retina degeneration like Retinitis Pigmentosa (RP), Leber's congenital amaurosis (LCA) and Stargardt's disease (STGD) lead to severe vision impairment or blindness. Each indication is characterized by a different age of disease onset and speed of degeneration.
A newly discovered cell death mechanism shows that photoreceptor cells degenerate and eventually die due to excessive cGMP signaling. This principal disease mechanism is caused by a large number of mutations in over 250 genes.
Retinitis Pigmentosa (RP), Leber congenital amaurosis (LCA) and Stargardt disease (STGD) are each orphan diseases with a combined patient number of about half a million in Europe, North America and Japan.
TWO main challenges for establishing therapies
The blood-retinal barrier, shielding the retina and its photoreceptors from pathogens, toxins, etc., but which also prevents access for therapeutic compounds.
The enormous genetic diversity of hereditary retinal degeneration, which would in principle require establishing a new gene therapy for each individual gene mutation.
Two major innovations introduced by mireca
Mireca applies a liposomal drug delivery technology which uses glutathione (GSH) to transfer therapeutic compounds across the blood-retinal barrier.
Mireca introduces cGMP analogues as a new class of therapeutic compounds that can interfere with excessive cGMP-signaling in photoreceptors. Importantly, excessive cGMP signaling is a phenomenon common to many, if not most, forms of hereditary retinal degeneration, thus allowing to treat many of these diseases with the same therapeutic compound.
Xu J et al.: cGMP accumulation causes photoreceptor degeneration in CNG channel deficiency: evidence of cGMP cytotoxicity independently of enhanced CNG channel function. J Neurosci. 2013 Sep 11;33(37):14939-48
PCT/EP2016/055659, Targeted liposomal delivery of cGMP analogues
PCT/EP2017/066113, PKG antagonists (Inhibitors)
PCT/EP2017/071859, PKG agonists (Activators)
ODD EU/3/15/1462, LP-DF003 for Retinitis Pigmentosa