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Find Clinical Drug Development Pipelines & Deals | PipelineProspector
These mechanisms of the two licensed novel small moleculeare are distinct and potentially complementary to that of Oxbryta tablets, a novel hemoglobin S polymerization inhibitor approved in the United States for the treatment of SCD in patients ages 12 years and older.
The collaboration aims to evaluate the opportunities for peptide-based therapeutic candidates across multiple indications through the well-established phage and mRNA display platforms.
The proceeds will support the advancement of KateTx’s next generation AAV-based gene therapies for muscle and heart disease programs, including myotonic dystrophy type 1 and facioscapulohumeral muscular dystrophy, which are the leading causes of adult-onset muscular dystrophy.
The collaboration will combine the use of novel Huntington's disease drug targets from Aitia's Gemini Digital Twins with UCB's expertise in drug research and preclinical model systems in neurodegeneration to accelerate the discovery of new therapies for Huntington's disease.
Under the terms of the partnership, Nanite will design delivery vehicles for genetic medicines to Schwann cells via PNPs, an emerging technology for drug delivery to hard-to-hit targets and to enhance therapeutic efficacy of antisense oligonucleotides in CMT1A.
Neurochase is developing a proprietary drug delivery system and Spark will develop selected gene therapies for CNS disorders using the Neurochase technology, which aims to improve targeted delivery of AAV gene therapy to neural structures using the method of CED.
The funding will be used toward developing Myosana’s platform with the goal of identifying the first development candidate for Duchenne muscular dystrophy by 2025.
Gensaic will use the net proceeds to develop full CFTR gene delivery to lung tissue using its proprietary phage-derived particle (PDP) platform in CFTR-based gene Therapy.
As part of the research collaboration, the companies will leverage Pfizer's expertise and Gero’s technology platform with the aim to identify genes and pathways linked to fibrotic diseases.
Through the collaboration with Orbit, Sanegene is looking to develop a portfolio of advanced RNAi based Therapies. Orbit’s proprietary technology enables the screening of large libraries of peptides using a combination of DNA encoded libraries and bead-based presentation.
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Sanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme. 
