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Cures of Duchenne (List of All Researches)

Research

Dystrophin Gene Therapy

Mechanism
Company
Drug
Status
Micro-dystrophin Gene Replacement
Sarepta Therapeutics
Elevidys/SRP-9001
research status green Cures of Duchenne (List of All Researches)
Approved
Micro-dystrophin Gene Replacement
Pfizer
PF-06939926
research status red Cures of Duchenne (List of All Researches)
Discontinued
Micro-dystrophin Gene Replacement
Genethon (Sarepta)
GNT-0004
research status orange Cures of Duchenne (List of All Researches)
Clinical
Micro-dystrophin Gene Replacement
RegenxBio
RGX-202
research status orange Cures of Duchenne (List of All Researches)
Clinical
Micro-dystrophin Gene Replacement
Solid Biosciences
SGT-001
research status orange Cures of Duchenne (List of All Researches)
Clinical
Micro-dystrophin Gene Replacement
Solid Biosciences
SGT-003
research status orange Cures of Duchenne (List of All Researches)
Clinical
Micro-dystrophin Gene Replacement
Ultragenyx
UX810
research status blue Cures of Duchenne (List of All Researches)
Preclinical
Micro-dystrophin Gene Replacement
Insmed
INS1201
research status blue Cures of Duchenne (List of All Researches)
Preclinical

Dystrophin Exon Skipping

Mechanism
Company
Drug
Status
PMO
Sarepta Therapeutics
Exondys 51 (Exon 51)
research status green Cures of Duchenne (List of All Researches)
Accelerated Approval
PMO
Sarepta Therapeutics
Vyondys 53 (Exon 53)
research status green Cures of Duchenne (List of All Researches)
Accelerated Approval
PMO
Sarepta Therapeutics
Amondys 45 (Exon 45)
research status green Cures of Duchenne (List of All Researches)
Accelerated Approval
PPMO
Sarepta Therapeutics
SRP-5051 (Exon 51)
research status red Cures of Duchenne (List of All Researches)
Discontinued
PPMO
Sarepta Therapeutics
Exon 44, 45, 50, 52, 53
research status blue Cures of Duchenne (List of All Researches)
Preclinical
PMO
NS Pharma
Viltepso (Exon 53)
research status green Cures of Duchenne (List of All Researches)
Accelerated Approval
PMO
NS Pharma
NS-089/NCNP-02 (Exon 44)
research status orange Cures of Duchenne (List of All Researches)
Clinical
PMO
NS Pharma
NS‐050/NCNP‐03 (Exon 50)
research status orange Cures of Duchenne (List of All Researches)
Clinical
PMO
NS Pharma
Exon 51, 55, 45
research status blue Cures of Duchenne (List of All Researches)
Preclinical
AAV-U7 snRNA
Nationwide Children’s
scAAV9.U7.ACCA (Exon 2)
research status orange Cures of Duchenne (List of All Researches)
Clinical
Stereopure-PN-ASO
Wave Life Sciences
WVE-N531 (Exon 53)
research status orange Cures of Duchenne (List of All Researches)
Clinical
Stereopure-PN-ASO
Wave Life Sciences
Exon 44, 45, 51, 52
research status blue Cures of Duchenne (List of All Researches)
Preclinical
Antibody-PMO
Avidity Biosciences
AOC 1044 (Exon 44)
research status orange Cures of Duchenne (List of All Researches)
Clinical
Antibody-PMO
Avidity Biosciences
Exon 45, 51
research status blue Cures of Duchenne (List of All Researches)
Preclinical
Antibody-PMO
Dyne Therapeutics
DYNE-251 (Exon 51)
research status orange Cures of Duchenne (List of All Researches)
Clinical
Antibody-PMO
Dyne Therapeutics
Exon 44, 45, 53
research status blue Cures of Duchenne (List of All Researches)
Preclinical
PPMO (EDO-CPP)
PepGen
PGN-EDO51 (Exon 51)
research status orange Cures of Duchenne (List of All Researches)
Clinical
PPMO (EDO-CPP)
PepGen
Exon 44, 45, 53
research status blue Cures of Duchenne (List of All Researches)
Preclinical
PPMO (Endosomal Escape)
Entrada Therapeutics
ENTR-601-44 (Exon 44)
research status orange Cures of Duchenne (List of All Researches)
Clinical
PPMO (Endosomal Escape)
Entrada Therapeutics
Exon 45, 50, 51
research status blue Cures of Duchenne (List of All Researches)
Preclinical
PS-ASO
BioMarin Pharmaceutical
BMN 351 (Exon 51)
research status orange Cures of Duchenne (List of All Researches)
Clinical

Dystrophin Gene Editing

Mechanism
Company
Drug
Status
Genome Editing
Vertex Pharmaceuticals
CRISPR/Cas9
research status blue Cures of Duchenne (List of All Researches)
Preclinical
Genome Editing (Exon 45-55)
MyoGene Bio
CRISPR/Cas9
research status blue Cures of Duchenne (List of All Researches)
Preclinical
Genome Editing (Exon 45-55)
Precision BioSciences
ARCUS-Mediated Excision
research status blue Cures of Duchenne (List of All Researches)
Preclinical
Genome Editing (Exon 50)
GenAssist
GEN6050X
research status orange Cures of Duchenne (List of All Researches)
Clinical

Dystrophin Stop Codon Read-Through

Mechanism
Company
Drug
Status
Stop Codon Read-through
PTC Therapeutics
Ataluren
research status green Cures of Duchenne (List of All Researches)
Conditional Approval in EU
Stop Codon Read-through
hC Bioscience
tRNA-based approach
research status blue Cures of Duchenne (List of All Researches)
Preclinical
Stop Codon Read-through
Tevard Biosciences
tRNA-based approach
research status blue Cures of Duchenne (List of All Researches)
Preclinical

Anti-Inflammatory

Mechanism
Company
Drug
Status
Corticosteroid
PTC Therapeutics
Emflaza
research status green Cures of Duchenne (List of All Researches)
Approved
Corticosteroid
Catalyst/Santhera/ReveraGen
Vamorolone/AGAMREE
research status green Cures of Duchenne (List of All Researches)
Approved

Other Treatments

Mechanism
Company
Drug
Status
Oral Molecule Drug
Satellos
SAT-3247
research status orange Cures of Duchenne (List of All Researches)
Clinical
HDAC Inhibitor
Italfarmaco
Givinostat/Duvyzat
research status green Cures of Duchenne (List of All Researches)
Approved
Cardiac-derived Exosomes
Capricor Therapeutics
CAP-1002
research status orange Cures of Duchenne (List of All Researches)
Clinical
Muscle Stabilizer
Edgewise Therapeutics
EDG-5506
research status orange Cures of Duchenne (List of All Researches)
Clinical
CD49d Expression Inhibitor
Antisense Therapeutics
ATL1102
research status red Cures of Duchenne (List of All Researches)
Discontinued
Anti-IL1beta Antibody
Children’s Research Institute
Ilaris
research status orange Cures of Duchenne (List of All Researches)
Clinical
PGD synthase Inhibitor
Taiho Pharmaceutical
TAS-205
research status orange Cures of Duchenne (List of All Researches)
Clinical
Thromboxane Receptor Inhibitor
Cumberland Pharmaceuticals
Ifetroban
research status orange Cures of Duchenne (List of All Researches)
Clinical
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