Therapeutic/Technology: Gene Therapy, cell free
Development of MyoDys45-55, a gene editing therapy for Duchenne muscular dystrophy
Translational Candidate A gene editing therapy for Duchenne muscular dystrophy that permanently removes a hotspot region of patient mutations to restore dystrophin. Area of Impact Duchenne muscular dystrophy (DMD), a fatal muscle wasting disease with no cure. Mechanism of Action Our therapy uses CRISPR/Cas9 gene editing to permanently remove a hotspot region of DMD patient […]
A Phase I/IIa Study to Evaluate the Efficacy of Toca 511/Toca FC with Standard of Care Therapy in Newly Diagnosed High Grade Glioma
Therapeutic Candidate or Device DB107 (Toca 511), a retroviral replicating vector expressing yeast cytosine deaminase, which converts an antifungal prodrug to an anticancer drug Indication Newly diagnosed high-grade glioma Therapeutic Mechanism Retroviral replicating vector DB107 (Toca 511) spreads through tumors and delivers yeast cytosine deaminase (CD), which converts the antifungal prodrug 5-fluorocytosine (5-FC) to the […]
A Phase 2 Study Evaluating the Efficacy and Safety of IV Administered rAAV9 Vector Containing RP-A501 in Male Patients with Danon Disease
Therapeutic Candidate or Device RP-A501, a recombinant Adeno-Associated Virus Serotype 9 containing the LAMP2B transgene Indication Danon Disease Therapeutic Mechanism The proposed product, RP-A501, is an in vivo gene therapy consisting of a recombinant AAV9 vector engineered with genetic elements designed to ensure consistent delivery and expression of the LAMP2B transgene. The therapy is administered […]
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 in the SCN2A gene with both GOF and LOF effects when expressed in cells. nL-SCN2A-002 is designed to specifically bind the pathogenic allele and lower […]
A Phase 1b, Randomized, Blinded, Placebo-Controlled Dose-Ranging Study of GNSC-001 Evaluating Safety, Pharmacodynamics, and Biomarkers in Knee OA
Therapeutic Candidate or Device GNSC-001 is an adeno-associated vector (AAV) expressing an optimized form of IL-1Ra, a naturally occurring protein that blocks IL-1 signaling. Indication Osteoarthritis of the knee Therapeutic Mechanism GNSC-001 is an adeno-associated virus (AAV) gene therapy vector that expresses IL-1Ra and confers long-term IL-1 inhibition following a single, local injection into the […]
Phase 1 Clinical research program for functional cure of HIV with EBT-101, in-vivo gene therapy
Therapeutic Candidate or Device EBT-101 is a novel genome editing therapeutic targeting integrated HIV-1 genome to achieve sustained virologic reduction to enable functional cure. Indication EBT-101 is intended to deplete the reservoir of integrated HIV-1 proviral DNA in immune reconstituted virally suppressed HIV-1 positive individuals. Therapeutic Mechanism EBT-101 is an in vivo gene therapy product […]
A Phase 1b Safety Study for MRI guided delivery of AAV2-GDNF for the treatment of Parkinson’s disease
Therapeutic Candidate or Device AAV2-GDNF is a gene therapy product encoding Glial cell line-Derived Neurotrophic Factor (GDNF) Indication Parkinson's disease Therapeutic Mechanism AAV2-GDNF will be delivered into the putamen. GDNF is a growth factor expected to act by stimulating regeneration of the terminals of dopamine producing neurons that are progressively lost in PD. This is […]
Novel Gene Therapy Targeting Multiple Pathological Drivers of Desmoplakin Associated Arrhythmogenic Cardiomyopathy
Therapeutic Candidate or Device RJB-0402 is a liver-targeting adeno-associated virus vector-based gene therapy that drives over expression of FGF21. Indication Desmoplakin-related arrhythmogenic cardiomyopathy (DSP ACM) at high risk of life-threatening ventricular arrhythmias and sudden cardiac death. Therapeutic Mechanism RJB-0402 targets hepatocytes to drive liver specific expression of FGF21 to restore cardiomyocyte function in patients with […]
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 Need CMT4J is an ultra-rare disorder that presently lacks any available treatment options and represents an underserved orphan population. Project Objective Successful Filing of an […]
Manufacturing of AS-202, an Antisense oligonucleotides for a Phase 1/2 Clinical Trial for Amyotrophic Lateral Sclerosis
Therapeutic Candidate or Device AS-202, an antisense oligonucleotide Indication Amyotrophic Lateral Sclerosis Therapeutic Mechanism AS-202 targets PIKFYVE, a lipid kinase. By inhibiting PIKFYVE activity, it induces exosomal secretion, which robustly clears misfolded proteins including C9ORF72 dipeptide repeat proteins (DPRs), TDP-43 and tau from neurons. Unmet Medical Need To date, therapeutic options for ALS have been […]