The FDA lifted the clinical hold and a variety of innovative therapies for DMD are on the market

DMD is a rare X chromosome recessive genetic disorder that causes a series of muscle diseases

Sarepta can be described as a "star enterprise" in this disease field, and the three antisense oligonucleotide (ASO) therapies EXONDYS 51, VYONDYS 53 and AMONDYS 45 for DMD that have been approved for listing are all developed by the company, targeting exon 51, 53 and 45 jump mutations

In a Phase 2 clinical trial called MOMENTUM, patients treated with SRP-5051 at a dose of 30 mg/kg once a month produced higher levels of anti-amyotrophin

Innovative therapies for DMD are under development

World Duchenne Awareness Day is celebrated on 7 September each year, which aims to let more people know about DMD and the real life of patients with the disease, and then adopt effective strategies to improve the quality of life of

Gene therapy

SRP-9001（Sarepta）

SRP-9001 (Sarepta) is a gene therapy jointly developed by Sarepta and Roche to transmit genes encoding micromyotroprotein to muscle tissue through AAV, so that muscle cells express the corresponding protein, thereby slowing or preventing muscle degeneration

Currently, the candidate therapy is in the Phase 3 trial phase, and Sarepta plans to file a biologics licensing application (BLA)

PF-06939926（Pfizer）

PF-06939926 (Pfizer) is an intravenous AAV gene therapy developed by Pfizer that uses recombinant adeno-associated virus type 9 to deliver shortened dystrophin genes for therapeutic purposes
.

He has previously been granted FDA status for orphan drugs and rare pediatric diseases
.

In January 2021, the drug candidate completed the first patient administration in the Phase 3 trial, which is currently in
progress.

Both of the above are candidate gene therapies that are advancing faster, and it is expected to provide patients with more treatment options
as soon as possible.

In addition, Solid's AAV gene therapy SGT-001 is in Phase 1/2 clinical trials, and Généthon's AAV gene therapy GNT0004 is in Phase 1/2/3 clinical trials
.

Cell therapy

CAP-1002

CAP-1002 is an allogeneic cell therapy developed by Capricor for the treatment of advanced DMD consisting of cardiac-derived cells (CDCs), a special population of cells containing cardiac progenitor cells that improve muscle scarring or fibrosis and heart function
in DMD patients by releasing exosomes containing microRNAs, non-coding RNA, and proteins.

In addition, CAP-1002 has also shown potent immunomodulatory activity, which may promote cell regeneration
.

Data from a Phase 2 clinical trial showed that CAP-1002 can reduce the loss of upper extremity skeletal muscle function by up to 70% while being safe
.

In July 2022, Capricor announced the completion of the first patient administration of CAP-1002 in a Phase 3 trial
.

Gene editing therapy

CRD-TMH-001

In August 2022, the FDA approved an IND application for CRISPR gene editing therapy called CRD-TMH-001 for the treatment of DMD
.

The therapy is also the first CRISPR gene-editing therapy
approved to enter clinical trials for the treatment of DMD.

The candidate therapy sends the CRISPR gene-editing system directly into the body through a large number of viruses to restart the backed up anti-amyotrophin genes
in the patient's body.

However, the safety of the candidate therapy needs to be verified
by more trial data.

summary

Resources:

1.
The official website of each enterprise

——List of recent popular events——

▼ On September 14, exosome drug delivery vector research was shared

▼ On September 15, cost reduction and efficiency increase, small volume sterilization of gene therapy drugs and interpretation of regulations

▼ On September 21, the opening ceremony of Merck China Biosafety Testing And Experiment Center