Duchenne muscular dystrophy (DMD) is a disorder characterized by progressive degeneration of muscles, resulting in muscle loss, mobility limitations and lately, mortal risk. DMD is caused by mutations in the dystrophin gene (DMD) and about 80% of these are suitable for exon skipping, obtaining a functional dystrophin protein.
Denali Therapeutics Inc.’s extensive update on clinical programs in central nervous system diseases at the start of this year included plans for lead asset DNL-310 in mucopolysaccharidosis II (MPS II), also known as Hunter syndrome – a space where other notable players include such names as Regenxbio Inc. and Takeda Pharmaceutical Co. Ltd.
Denali Therapeutics Inc.’s extensive update on clinical programs in central nervous system diseases at the start of this year included plans for lead asset DNL-310 in mucopolysaccharidosis II (MPS II), also known as Hunter syndrome – a space where other notable players include such names as Regenxbio Inc. and Takeda Pharmaceutical Co. Ltd.
Abbvie Inc. and Regenxbio Inc. have announced a partnership to develop and commercialize RGX-314, a potential one-time gene therapy for the treatment of wet age-related macular degeneration (wet AMD), diabetic retinopathy and other chronic retinal diseases. Under the terms of the agreement, Abbvie will pay Regenxbio $370 million up front, plus up to $1.38 billion in additional development, regulatory and commercial milestones. The deal gives '314 – already the most advanced gene therapy in wet AMD – another potential edge against its nearest competitor, Adverum Biotechnologies Inc.’s ADVM-022.
The 2017 FDA approval of Luxturna (voretigene neparvovec-rzyl, Roche Holding AG) spurred a race to create the next gene therapy for the eye. The organ is very amenable to gene therapy given that it's a confined space with post-mitotic cells that has immune privilege and requires substantially smaller amounts of viral vector compared to systemic treatments.
Earlier this month, an update on phase I/IIa data rolled out from Rockville, Md.-based Regenxbio Inc. with RGX-314 for age-related wet macular degeneration (AMD). The company has a pivotal program in subretinal delivery of the compound set to start by the end of this year, and questions about routes of administration – always an issue in AMD – continue to simmer.
Following FDA approval of Novartis AG's VEGF-A inhibitor, Beovu, in wet age-related macular degeneration earlier this month, speculation immediately started on how much market share that new therapeutic will capture at the expense of Regeneron Pharmaceutical Inc.'s Eylea.
