Disease Focus: Pediatrics

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) […]

CRISPR/Cas9-mediated gene editing of Hematopoietic stem and progenitor cells for Friedreich’s ataxia

Translational Candidate Autologous human CD34+ HSPC of patients with Friedreich’s ataxia, modified ex vivo using CRISPR/Cas9 to remove the GAA expansion mutation in frataxin Area of Impact Friedreich’s ataxia (FRDA) for which there is no effective treatment available Mechanism of Action The proposed therapy intervention is intended to impact the target indication of Friedreich's ataxia […]

Human Neural Stem Cells (hNSCs) for neuroprotection in perinatal hypoxic-ischemic brain injury (HII)-Pre-IND-enabling Studies

Translational Candidate An established stable human neural stem cell line unmanipulated genetically & propagated under defined conditions Area of Impact Perinatal asphyxia (also called hypoxic-ischemic injury), a major untreatable cause of cerebral palsy & cognitive disability Mechanism of Action hNSCs rescue the penumbra, the part of the brain lesion following perinatal asphyxia that still has […]

ASCENT- Advanced Stem Cell Enteric Neuropathy Therapy

Translational Candidate ASCENT – Advanced Superdonor Cellular Enteric Neuropathy Therapy, is a donor progenitor cell population that replaces the enteric nervous system. Area of Impact ASCENT would treat enteric neuropathies including Hirschsprung disease and total intestinal aganglionosis which currently have no direct therapy Mechanism of Action Our goal is to develop an allogeneic “off 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 […]

Efficacy and safety of cryopreserved autologous CD34+ HSC transduced with EFS lentiviral vector encoding for human ADA gene in ADA-SCID subjects

Therapeutic Candidate or Device Autologous CD34+ hematopoietic stem cells (HSCs) transduced with a lentiviral vector encoding the human ADA gene (or "OTL-101") Indication Adenosine Deaminase – Severe Combined Immunodeficiency (or ADA-SCID) Therapeutic Mechanism This project will lead to a License Application for OTL-101 as a treatment for ADA-SCID. The patient’s own stem cells ("autologous") are […]

Efficacy and safety of cryopreserved autologous CD34+ HSC transduced with EFS lentiviral vector encoding for human ADA gene in ADA-SCID subjects

A Phase I/II, Non Randomized, Multicenter, Open-Label Study of G1XCGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease

Therapeutic Candidate or Device The therapeutic product candidate is autologous CD34+ hematopoietic stem/progenitor cells (HSPC) transduced with the G1XCGD lentiviral vector. Indication The target indication is for the transplantation of patients with severe X-linked Chronic Granulomatous Disease (XCGD) lacking matched donors. Therapeutic Mechanism Transplantation and engraftment of gene-corrected autologous HSPC after reduced intensity conditioning for […]

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 […]

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