Through the agreement, Vertex
will get a hold of both of Semma’s preclinical products, a stem cell
manufacturing process and a novel delivery system via the device.
Patients affected by a rare inherited eye disorder now will be able to
access a new treatment option in the form of Gene therapy.
The UK’s National Institute for Health and Care Excellence (NICE) has given its recommendation for the use of Novartis’ gene therapy Luxturna (voretigene neparvovec) to treat rare inherited retinal dystrophies.
Luxturna, originally developed by Spark Therapeutics, is an adeno-associated virus vector-based gene therapy. Indicated for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy.
In the therapy, the gene is
directly injected into the eye directly to restore the vision.
At the moment, there exists no standard cure for inherited retinal dystrophy. It leads to a loss of vision to a point of total blindness. The most common retinal dystrophy, Retinitis pigmentosa (RP) has a prevalence of roughly 1:4,000.
According to DelveInsight, the Retinitis Pigmentosa therapeutic market in the 7MM is expected to grow by 2028 driven at a Compound Annual Growth Rate (CAGR) of 28.90%.
In contrast to EU5 and the USA,
Japan’s Retinitis pigmentosa therapy market, so far, has been dominated by the
use of Luxturna.
As per the assessments by
DelveInsight’s analysts, the market shares of prescription therapies were found
to be USD 133.82 million in 2017.
Despite the approval of the gene
therapy and the emerging therapies, the market size of the supportive therapies
will continue to grow due to the fact that therapies to treat all types and
subsequent subtypes have not yet been developed, and therefore a significant
number of patient population will continue to be prescribed supportive
therapies as a management practice.
Bristol-Myers Squibb has announced a strategic partnership with a drug development
accelerator BioMotiv, to advance breakthrough drug discoveries.
The partnership will augment the ability of both organizations to develop novel advanced and innovative therapies for the patient pool.
Under the terms of the agreement, Bristol-Myers Squibb (BMS) will have an option to invest funds in specific projects of interest. The companies have decided to fund and develop novel therapeutics in the field of disease that has unmet needs and poor standards of therapies. Moreover, the partnership will allow BMS to acquire the company solely upon the identification of new molecules for treating any indication.
Bristol-Myers Squibb isn’t the first one to form the partnership to advance the drug discovery with BioMotiv. Other global pharma leaders that are already in the collaboration with BioMotiv are Takeda Pharmaceuticals, Biogen, Arix Bioscience and Charles River Laboratories.
