Development of AS-241, an UNC13A Targeting Antisense Oligonucleotide (ASO) Treatment for ALS, for IND-enabling Studies
Translational Candidate AS-241, an antisense oligonucleotide Area of Impact Amyotrophic Lateral Sclerosis Mechanism of Action AS-241 targets the cryptic exon (CE) of UNC13A and suppresses CE inclusion during RNA splicing,…
Development of a Gene Therapy for Treatment of Guanidinoacetate Methyltransferase Deficiency-Translating In Vivo Proof of Concept to Support a Pre-IND
Translational Candidate Adeno-associated viral vector serotyped for tropism to express guanidinoacetate methyltransferase in hepatocytes and brain cells. Area of Impact Developing a new therapy for Guanidinoacetate Methyltransferase Deficiency, where present…
Noncoding RNA drug TY1 as a therapeutic candidate for scleroderma and systemic sclerosis
Translational Candidate Modified synthetic noncoding RNA molecule Area of Impact Systemic Sclerosis Mechanism of Action The mechanism of action of TY1 is alleviating cell stress and damage through enhancing genes…
Development of a Gene Therapy for the Treatment of Arginase Deficiency – Translating from Proof of Concept to Support Pre-IND Meeting
Translational Candidate Adeno-associated viral vector serotyped for hepatic tropism to express Arginase 1 in hepatocytes. Area of Impact Developing a new therapy for Arginase Deficiency, where present day this is…
Telomerase mRNA for short telomere related pulmonary fibrosis
Translational Candidate Nucleoside-modified mRNA encoding telomerase reverse transcriptase (TERT) encapsulated in a lung-targeting lipid nanoparticle delivered intravenously. Area of Impact 1) Idiopathic pulmonary fibrosis (IPF) and 2) Other diseases and…
Escape-Resistant Oligonucleotide Therapy (ONT) for Cytomegalovirus (CMV) Disease in Hematopoietic Stem-Cell and Solid-Organ Transplant Patients
Translational Candidate Cytomegalovirus antiviral FD-86: DNA Oligonucleotide Therapy Area of Impact HSCT transplant rejection and childhood cognitive and hearing impairment caused by Cytomegalovirus Mechanism of Action The candidate oligonucleotide therapy,…
Personalized antisense oligonucleotide therapy for rare pediatric genetic disease: SCN2A
Therapeutic Candidate or Device Investigational personalized antisense oligonucleotide drug (nL-SCN2A-002) Indication SCN2a-associated genetic disorder Therapeutic Mechanism The study participant has a pathogenic de novo p.R853Q gain-of-function amino acid substitution mutation…
Pre-Clinical To Clinical Gene Therapy Development For CMT4J
Therapeutic Candidate or Device AAV9 Gene Therapy For An Ultra-Rare Disease Called CMT4J Indication CMT4J or the FIG4 Gene Therapeutic Mechanism Gene Therapy through an Intrathecal One-Time Injection Unmet Medical…
