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Find Drugs for Rare Diseases and Disorders in Preclinical Development
NM3086 (an anti-properdin antibody) blocks the alternative pathway without blocking the classical pathway and as a result a) blocks hemolysis of PNH-like erythrocytes, b) reduces lactate dehydrogenase, and c) reduces free hemoglobin (Hb) in an animal model of PNH.
Avilar develops ATACs (ASGPR Targeting Chimeras), a new class of protein degraders that shuttle disease-causing proteins from circulation to the endolysosome where the unwanted proteins are degraded.
Rapid and robust in vivo protein degradation using ATACs that employ Avilar’s proprietary high-affinity, small molecule ASGPR ligands in a monovalent format.
Toregem BioPharma will have access to WuXi Biologics’ integrated CMC services in cell line development, cell culture development, biologics GMP manufacturing and related services. WuXi Biologics will support Toregem BioPharma on the TRG035 project for its IND application.
The collaboration will support the activities as development of new technologies to improve ongoing research activities, enhance expression and production of proteins within plants and for development of new approaches and plant lines to increase plant-derived product yields.
NPI-001 is a differentiated, purified, prescription-quality, GMP-grade N-acetylcysteine amide (NACA) being developed to treat a broad set of diseases and conditions associated with oxidative stress and reduced glutathione levels.
The focus will be on therapies derived from biologics — cells, blood, enzymes, tissues, genes, or genetically engineered cells — for use in medicines. In addition, the collaboration seeks to advance biologic discoveries toward early stage clinical trials.
Proceeds of financing will be used to validate advances of the Company's proprietary epigenetic editing in non-human primates, expand expertise and capabilities, and sponsor early-stage clinical investigations.
STRM.BIO has developed a proprietary, large capacity EV-based therapy for in vivo nucleic acid and protein delivery that specifically targets hematopoietic stem cells (HSCs) in bone marrow, is amenable to large scale commercial manufacture and presents with low immunogenicity.
Proceeds from the financing will be used to fund the diversification of ReCode’s pipeline into central nervous system, liver, and oncology indications, while continuing to advance lead mRNA programs for primary ciliary dyskinesia and cystic fibrosis into the clinic.
Lead Product(s):
SORT LNP-based DNAI1 mRNA Therapeutic
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