It is expected to slow down or stop the progress of DMD!

Recent popular reports from Yimaike ★NoticeShanghai Minhang Biopharmaceutical Industry Development Conference and 2021 Life and Health Industry Summit Forum is about to open in Shanghai ★September·ShanghaiOncolytic Virus Drug Development Forum is about to open Click on the picture and sign up for the conference September 25, 2021 EMedClub News/--Recently, the life science company Solid Biosciences released the latest phase I/II clinical trial results of the gene therapy SGT-001 for Duchenne muscular dystrophy (DMD).
The research data is in the World Muscle Association The oral session of the virtual conference in 2021 was announced
.

Duchenne muscular dystrophy (DMD) is a fatal genetic disease.
It is caused by mutations in the dystrophin gene on the X chromosome.
The key structural function can strengthen muscle fibers to protect them from daily wear and tear.
If the protein function is missing, the body's muscles will be excessively damaged by daily activities and cannot be regenerated, leading to fibrosis, scarring and the formation of adipose tissue
.

The muscle weakness symptoms of DMD first occur in early childhood (2-5 years old), lose the ability to walk in adolescence (10-13 years old), and eventually lead to adolescence (25-28 years old) due to complications of heart or respiratory failure Death
.

It is estimated that about 15,000 boys in the United States suffer from DMD, and there are an estimated 300,000 patients worldwide
.

At present, most DMD clinically have no effective treatment method, and gene therapy is a potentially effective treatment plan
.

SGT-001 is a new type of adeno-associated virus (AAV) vector-mediated gene transfer therapy, designed to solve the impact of dystrophin gene mutation in DMD
.

SGT-001 delivers the loaded dystrophin gene (called microdystrophin) to the target tissues of the body through systemic administration
.

This microdystrophin encodes a functional protein substitute that stably expresses related proteins necessary for the human body in muscles, including neuronal nitric oxide synthase (nNOS)
.

According to reports, the expression of nNOS can enable muscles to receive appropriate blood flow when necessary to meet the high energy requirements of muscle response
.

▲Gene sequence expressed by SGT-001 (picture source: Solid) IGNITE DMD 1.
5 years data On September 23, Solid reported the 1.
5 years of IGNITE DMD Phase I/II clinical trials of 6 patients in the high-dose group (No.
4-6) Functional data and outcome indicators reported by patients.
All patients in this cohort received SGT-001 at a dose of 2E14 vg/kg.
Analysis of long-term biopsy data collected from patients 4-6 1.
5 years after administration showed evidence of micromuscular nourishment.
Long-lasting and extensive expression of protein
.

For more details, scan the QR code for a complete reading.
"The data provided today demonstrate the persistent expression and function of microdystrophin in biopsy samples collected 12 to 24 months after the administration of SGT-001," IGNITE DMD Clinical Investigator Dr.
Vamshi Rao said, “In addition, compared with natural history data, these data provide encouraging evidence of functional benefit 1.
5 years after treatment and show a significant improvement in patient-reported results
.

From others Patient data should enhance our understanding of the role SGT-001 may play in improving the prognosis of Duchenne patients
.

” said Ilan Ganot, CEO, president and co-founder of Solid Biosciences: “We still believe that SGT-001 may be Duchenne.
Patients provide differentiated benefits, and it is expected that more patients will be dosed in IGNITE DMD
.

"‍‍▲Solid Biosciences CEO, president and co-founder Ilan Ganot has had a bumpy journey.
DMD gene therapy will eventually turn cocoons into butterflies.
Prior to this, SGT-001 clinical trials were suspended twice because some patients had appeared.
Serious adverse reactions
.

However, Solid revised the clinical protocol and manufacturing process later and provided more data
.

As the patient's remission was satisfactory and the adverse reactions were controllable, the FDA lifted the clinical suspension.
Solid will be released in 2020.
The clinical trial was restarted, and the adverse event was finally resolved
.

On March 25, 2021, Solid announced the low-dose and high-dose drug SGT-001 for the treatment of DMD at the virtual clinical and scientific meeting of the Muscular Dystrophy Association (MDA).
In the one-year follow-up results of the dose group, overall, the efficacy indicators have improved without serious side effects
.

Recommended reading: The latest data! Can this DMD gene therapy, which has been suspended for more than a year, effectively improve the patient's condition Medical Mai Meng broke the news and the newly updated 1.
5-year follow-up study data of the high-dose group.
Solid proved that SGT-001 is safe regardless of gene mutation or disease stage, and can slow or prevent the progression of DMD
.

In addition to Solid, Two other giants of DMD gene therapy are also active in this field: Pfizer's Phase III clinical trial of gene therapy PF-06939926 has opened 14 test sites in 8 countries
.

Up to now, test sites in Spain and the United Kingdom Patients with DMD have begun to take drugs
.

Recommended reading: Pfizer launched a key phase 3 clinical study of AAV gene therapy, DMD ushered in another important milestoneYimai Meng revealed that Sarepta is also pursuing a genetic solution to DMD, including the most advanced procedure It should be the gene therapy SRP-9001 agreed with Roche
.

In May 2021, muscle biopsy of 11 boys with Duchenne muscular dystrophy (DMD) in a phased clinical trial of SRP-9001 showed early signs of biological efficacy
.

Although DMD is a rare disease, there is still an urgent need for a treatment plan with curative potential for the affected family.
It is hoped that gene therapy can obtain regulatory approval as soon as possible and be applied to the treatment of rare gene diseases
.

Reference materials: 1.
https:// -ii-clinical-trial-of-sgt-001/2.
https:// /