Therapeutic/Technology: Gene Therapy, cell free
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 day this is minimally effective at best. Mechanism of Action The proposed clinical candidate is a virus that has been altered to carry the gene […]
Optogenetic Therapy for Treatment of Geographic Atrophy
Translational Candidate Optogenetic gene therapy for patients with geographic atrophy age related macular degeneration. Area of Impact Blindness from geographic atrophy age related macular degeneration Mechanism of Action Gene therapy to deliver optogenetic protein to the targeted cells of the retina to restore vision. Unmet Medical Need Geographic atrophy (GA) age related macular degeneration is […]
Development of an AAV gene therapy immunotherapy for the treatment of glioblastoma
Translational Candidate SRN-101, an experimental AAV gene therapy for treating glioblastoma Area of Impact Cancer – solid tumors Mechanism of Action Following delivery with an AAV, engineered cytokines are expressed from within the tumor to kill the tumor from the inside out, they are then further secreted to stimulate local immune cells to kill remaining […]
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 that alleviate cell stress which, in turn, control inflammation and fibrosis in diseases tissue. Unmet Medical Need Systemic sclerosis is an incurable disease with no […]
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 minimally effective at best. Mechanism of Action The proposed clinical candidate is a virus that has been altered to carry the gene for & produce […]
AAV Gene Therapy for Treating Congenital Hereditary Endothelial Dystrophy associated with Biallelic SLC4A11 Mutations
Translational Candidate Therapeutic candidate rAAV8-EF1α-hSLC4A11 is a recombinant AAV vector with single-stranded cDNA encoding the wild-type human SLC4A11 protein. Area of Impact The candidate is for treatment of congenital hereditary endothelial dystrophy, an orphan disease associated with congenital corneal opacification. Mechanism of Action Therapeutic candidate rAAV8-EF1α-hSLC4A11 introduces normal copies of human SLC4A11 gene into the […]
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 conditions caused or exacerbated by short telomeres. Mechanism of Action TERT mRNA LNPs transiently extend the telomeres of the alveolar epithelial cells of the lung, […]
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, FD-86 disrupts viral IE feedback circuitry and breaks homeostatic control of cytotoxic IE proteins inducing apoptosis only in infected cells. Unmet Medical Need Treatment-resistant CMV […]
Development of a Gene Therapy for the Treatment of Pitt Hopkins Syndrome (PHS) – Translating from Animal Proof of Concept to Support Pre-IND Meeting
Translational Candidate MZ-1866 is a recombinant AAV9 based gene therapy containing the transgene encoding Transcription Factor 4 (TCF4) Area of Impact Pitt Hopkins Syndrome is a rare genetic neurological disease which causes profound disability and severe health impact Mechanism of Action Patients with Pitt Hopkins Syndrome have heterozygous mutations in the Transcription Factor 4 (TCF4) […]
Optogenetic therapy for treating retinitis pigmentosa and other inherited retinal diseases
Translational Candidate Ray-001 is an AAV gene therapy delivering a light sensitive gene to treat patients with advanced Retinitis Pigmentosa. Area of Impact Retinitis pigmentosa (RP) is a genetic disease that causes retinal degeneration leading to near or complete blindness for most patients. Mechanism of Action Ray Therapeutics' therapy (Ray-001) delivers a potent transgene with […]