Irish Gene Therapy for Blindness is Acquired by US Leader in the Field

spark therapeutics genable gene therapy blindness pigmentosa rhonova

Spark Therapeutics (US) has paid over €13.5M for Genable Technologies (Ireland) and its gene therapy for a degenerative eye disease which progresses to blindness.

spark_therapeutics_genable_technologies_gene_thearpy_inherited_retinal_dystrophies_rhonova_suppression_replacementSpark Therapeutics is one of the players in the re-emerging field of Gene Therapy (which is already establishing records like the most expensive drug). It focuses on debilitating genetic diseases, like inherited retinal dystrophies (IRDs), neurodegenerative and blood disorders.

It will now add a new candidate to its IRD pipeline, RhoNova, which was in preclinical development with the collaboration with Genable Technologies since 2014.

Spark has now decided to go all the way and buy the Irish Biopharma for $6M cash and $9.15M worth of Spark’s stocks (265,000 shares), amounting to about €13.76M.

Genable has an innovative approach to the treatment of autosomal dominant diseases. For these types of genetic diseases, having only one copy of a mutated gene can lead to developing symptoms (even if the other copy is healthy).

Comparison of patterns of inheritance for autosomal genetic diseases. In recessive diseases, carriers of one defective gene are not sick. (Source: Genetics Home Reference, NIH)

Genable created a ‘suppressions and replacement’ (S&R) strategy to deal with autosomal dominant diseases – the basis for RhoNova, which is a gene therapy for rhodopsin-linked autosomal dominant retinitis pigmentosa (RHO-adRP). This condition is caused by mutations of the RHO gene, and leads to clinical blindness in most cases.

RhoNova uses RNA interference (RNAi) to suppress all RHO mRNA copies, by targeting a part of the genetic sequence which is constant (i.e. is unaffected by any mutations). This suppresses translation of all versions of the RHO gene, and works for all of the 200 different mutations that can cause the retinitis pigmentosa.

Mechanism of RNA interference (CC 2.5: Richard Robinson)

An engineered RHO gene is then introduced as a replacement in the patient’s system (with Spark’s adeno-associated virus vector), so that it codes for a normal, healthy protein – and can reverse symptoms.

RhoNova has been already granted Orphan Drug Designation in Europe and the US. But Spark is looking into the potential of Genable’s strategy for many other indications as well.

This acquisition is another success for Spark, that is also preparing to file their lead candidate (SPK-RPE65) after positive phase III results in patients with RPE65-mediated inherited retinal dystrophies.

This R&S strategy from Genable could be an important asset for Spark, as the Gene Therapy field is coming of age and more as applications for it get approved.

This is how Genable goes about the development of RhoNova…

Genable – RhoNova from Ian on Vimeo.

Feature image credit: Eye layers – © Sciencepics (BigStock ID81245891)

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