SUMMARY A Sindbis virus-based self-amplifying RNA vector has been engineered for long-lasting, safe gene expression in skin cells, aiming to treat genetic skin disorders like recessive X-linked ichthyosis by restoring missing genes with improved durability and reduced side effects. The Unmet… Read More
SUMMARY First-in-class small molecules to change how the PRNP gene is spliced, reducing harmful prion levels and offering a new treatment approach for prion diseases The Unmet Need: Safe, effective, and deliverable therapies to reduce prion protein levels in the central… Read More
SUMMARY Phosphorodiamidate morpholino oligomer (PMO)-based RNA-degrading chimeras reduce α-synuclein protein by degrading its messenger RNA preventing protein aggregation and propagation associated with neurodegenerative diseases. The Unmet Need: More potent and selective therapeutic strategies for neurodegenerative α-synucleinopathies Neurodegenerative diseases such… Read More
SUMMARY This technology regulates the expression of the CACNA1A gene, which encodes a neuronal calcium channel subunit, by redirecting alternative splicing to treat related neurological diseases. This approach can potentially correct gene expression variations linked to disorders including episodic ataxia, migraines, epilepsy,… Read More
SUMMARY Antibodies that block growth hormone signaling by binding the receptor or hormone to stop receptor pairing, based on high-affinity, defined sequences for therapeutic and research use. The Unmet Need: Effective therapeutic strategies that can precisely and sustainably inhibit growth hormone receptor-mediated… Read More
SUMMARY Synthetic monoclonal antibodies using phage display to bind and block the human growth hormone receptor, potentially offering a more potent and specific treatment for conditions like acromegaly by inhibiting receptor activation and downstream signaling. The Unmet Need: Innovative approaches to develop… Read More
SUMMARY Many human diseases arise from insufficient protein levels, often due to genetic mutations that result in haploinsufficiency or loss of protein function. Effectively treating these conditions necessitates therapeutic strategies capable of increasing the production of specific, vital proteins. There is a significant unmet need for programmable methods to… Read More
SUMMARY Spinocerebellar ataxia type 6 (SCA6) is a rare, dominantly inherited disease that causes Purkinje cell degradation and results in: loss of coordination, tremors, and uncoordinated muscle movement. Current therapies only mitigate symptoms and do not address the underlying pathophysiology responsible for disease progression. It is known that individuals with… Read More