This clinical trial is an open-label one-time injection dose study in which scAAV1.tMCK.NTF3 will be administered by intramuscular injections into muscles in both legs in CMT1A subjects with PMP22 gene duplication. Three subjects ages 18 to 35 years receiving (8.87e11 vg/kg) will be enrolled.

Start Date01 Apr 2025

Sponsor / Collaborator

Nationwide Children's Hospital

100 Clinical Results associated with scAAV1.tMCK.NTF3(Nationwide Children's Hospital)

100 Translational Medicine associated with scAAV1.tMCK.NTF3(Nationwide Children's Hospital)

100 Patents (Medical) associated with scAAV1.tMCK.NTF3(Nationwide Children's Hospital)

Literatures (Medical) associated with scAAV1.tMCK.NTF3(Nationwide Children's Hospital)

01 Apr 2022·Gene TherapyQ3 · MEDICINE

AAV1.NT-3 gene therapy for X-linked Charcot–Marie–Tooth neuropathy type 1

Q3 · MEDICINE

Article

Author: Sahenk, Zarife ; Moss, Kyle ; Freidin, Mona M ; Chen, Lei ; Abrams, Charles K ; Mckinney, Jennifer ; Myers, Morgan ; Ridgley, Alicia ; Ozes, Burcak ; Bai, Shasha ; Mendell, Jerry R

AbstractX-linked Charcot-Marie-Tooth neuropathy (CMTX) is caused by mutations in the gene encoding Gap Junction Protein Beta-1 (GJB1)/Connexin32 (Cx32) in Schwann cells. Neurotrophin-3 (NT-3) is an important autocrine factor supporting Schwann cell survival and differentiation and stimulating axon regeneration and myelination. Improvements in these parameters have been shown previously in a CMT1 model, TremblerJ mouse, with NT-3 gene transfer therapy. For this study, scAAV1.tMCK.NT-3 was delivered to the gastrocnemius muscle of 3-month-old Cx32 knockout (KO) mice. Measurable levels of NT-3 were found in the serum at 6-month post gene delivery. The outcome measures included functional, electrophysiological and histological assessments. At 9-months of age, NT-3 treated mice showed no functional decline with normalized compound muscle action potential amplitudes. Myelin thickness and nerve conduction velocity significantly improved compared with untreated cohort. A normalization toward age-matched wildtype histopathological parameters included increased number of Schmidt-Lanterman incisures, and muscle fiber diameter. Collectively, these findings suggest a translational application to CMTX1.

01 Nov 2021·Journal of Biomedical Materials Research Part AQ3 · ENGINEERING & TECHNOLOGY

Surface modification of neurotrophin‐3 loaded PCL/chitosan nanofiber/net by alginate hydrogel microlayer for enhanced biocompatibility in neural tissue engineering

Q3 · ENGINEERING & TECHNOLOGY

Article

Author: Mohammadi, Parvin ; Andalib, Sina ; Forouzideh, Narges ; Habibizadeh, Mina ; Fattahi, Ali ; Nadri, Samad ; Rostamizadeh, Kobra ; Hamidi, Mehrdad

AbstractThis study prepared a novel three‐dimensional nanocomposite scaffold by the surface modification of PCL/chitosan nanofiber/net with alginate hydrogel microlayer, hoping to have the privilege of both nanofibers and hydrogels simultaneously. Bead free randomly oriented nanofiber/net (NFN) structure composed of chitosan and polycaprolactone (PCL) was fabricated by electrospinning method. The low surface roughness, good hydrophilicity, and high porosity were obtained from the NFN structure. Then, the PCL/chitosan nanofiber/net was coated with a microlayer of alginate containing neurotrophin‐3 (NT‐3) and conjunctiva mesenchymal stem cells (CJMSCs) as a new stem cell source. According to the cross‐sectional FESEM, the scaffold shows a two‐layer structure with interconnected pores in the range of 20 μm diameter. The finding revealed that the surface modification of nanofiber/net by alginate hydrogel microlayer caused lower inflammatory response and higher proliferation of CJMSCs than the unmodified scaffold. The initial burst release of NT‐3 was 69% in 3 days which followed by a sustained release up to 21 days. The RT‐PCR analysis showed that the expression of Nestin, MAP‐2, and β‐tubulin III genes were increased 6, 5.4, and 8.8‐fold, respectively. The results revealed that the surface‐modified biomimetic scaffold possesses enhanced biocompatibility and could successfully differentiate CJMSCs to the neuron‐like cells.

01 Jan 2020·Brain ResearchQ4 · MEDICINE

Gene therapy to promote regeneration in Charcot-Marie-Tooth disease

Q4 · MEDICINE

Review

Author: Sahenk, Zarife ; Ozes, Burcak

The molecular pathogenesis underlying Charcot-Marie-Tooth (CMT) neuropathy subtypes is becoming increasingly variable and identification of common approaches for treatment, independently of the disease causing gene defect, is therefore much desirable. Gene therapy approach from the clinical translational view point is particularly challenging for the most common "demyelinating" CMT1 subtypes, caused by primary Schwann cell genetic defects. Studies have shown that impaired regenerative capacity of distal axons is major contributing factor to distal axonal loss in primary Schwann cell genetic defects and neurotrophin 3 (NT-3) improves impaired regeneration in CMT1 mouse models. This review surveys the evidence supporting the rationale for AAV1.NT-3 surrogate gene therapy to improve nerve regeneration in CMT1A. The translational process, from proof of principal studies to the design of the phase I/IIa trial evaluating scAAV1.tMCK.NTF3 gene therapy for treatment of CMT1A is summarized.

100 Deals associated with scAAV1.tMCK.NTF3(Nationwide Children's Hospital)

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Indication	Highest Phase	Country/Location	Organization	Date
Charcot-Marie-Tooth Disease	Phase 2	-	Nationwide Children's Hospital	-
Charcot-Marie-Tooth Disease, Type Ia	Phase 1	US	Nationwide Children's Hospital	01 Apr 2025

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