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The collaboration will explore the development of gene therapy product CTx-TFEB, targeting the transcription factor EB (TFEB) for the treatment of alpha-synucleinopathies, such as Multiple System Atrophy (MSA) and idiopathic Parkinson’s disease (PD).
APL-1030, a potential first-in-class, brain-active C3 inhibitor for neurodegenerative and other complement-driven diseases, and APL-2006, an ophthalmological candidate in development to treat both wet age-related macular degeneration (AMD) and geographic atrophy.
Under the two-stage collaboration both companies will initially combine their complementary expertise to co-develop manufacturing technologies for AAV-based gene therapy products including CTx-GBA1.
Under the license and collaboration agreement, REGENXBIO received equity in Corlieve and is eligible to receive milestone payments and royalties on net sales of AMT-260.
U.S. FDA has granted orphan drug designation to BrainEver's product BREN-02, the recombinant human homeoprotein Engrailed 1, for the treatment of amyotrophic lateral sclerosis.
Lead Product(s):
Recombinant human homeoprotein Engrailed 1
The patent complements the portfolio of patents covering Ezéprogind and its therapeutic uses already granted in Europe, and in 11 other countries including Japan and China.