Express promotes the main DMD gene therapy into the clinic, and the new sharp joint efforts to create a gene therapy pipeline

Solid Biosciences today announced a merger agreement with AavantiBio to acquire AavantiBio's gene therapy pipeline
.
The transaction will combine the expertise of both parties to create a precision genetic medicine company
.
The merger will focus on the development of drugs for neuromuscular and rare heart diseases, including the development of
the two companies in Duchenne muscular dystrophy (DMD), Friedreich's Ataxia (FA), BAG3-mediated dilated cardiomyopathy and other heart diseases that have not yet been revealed.

The combined company will focus on leading projects on SGT-003, AVB-202 and AVB-401, as well as the establishment of the
next-generation adeno-associated virus (AAV) capsid library.
SGT-003 is the next generation of AAV gene therapy that uses the innovative promuscularly procapsid protein (AAV-SLB101) to deliver Solid Biosciences' proprietary neuron-type nitric oxide synthase (nNOS) microdystrophin protein into the muscles to treat Duchenne muscular dystrophy
.
It is a rare genetic disorder caused by mutations in genes encoding antiamyotrobin on the X chromosome
.
Deletion or defect of antiamyotrobin leads to chronic damage to muscles during contraction, onset of inflammation, affecting muscle regeneration
.
Solid Biosciences intends to continue and prioritize the development of the SGT-003 program and to deliver new drug clinical trial (IND) applications in mid-2023, and if approved, to begin patient recruitment
by the end of 2023.

AVB-202 is AavantiBio's preclinical AAV candidate gene therapy for the treatment of Friedrich Reich's ataxia
.
The disease is caused by repeated amplification of triple-nucleotides in the FXN gene encoding frataxin, a change that results in a significant decrease
in frataxin protein levels.
The lack of frataxin can lead to iron accumulation in tissues, affecting cells with high energy requirements in tissues such as the heart and brain, which in turn can gradually cause damage to the patient's nervous system and motor difficulties
.
Three animal model studies from mice and non-human primates conceptually confirmed the effectiveness
of this therapy.
Solid Biosciences expects to file an IND application for
AVB-202 in the second half of 2024.

In addition, Solid Biosciences will continue to innovate the development of AAV capsid to enhance the selectivity of viral capsid to heart and skeletal muscle, while reducing targeting the liver
.
The first capsid screened through its skeletal muscle library was AAV-SLB101
.
This capsid was used in the SGT-003 project
.
Compared with many other preclinical studies using AAV9, the expression and biological distribution of the virus have improved
.

Mr.
Ilan Ganot, President, CEO and Co-Founder of Solid Biosciences, said: "This merger provides us with the perfect opportunity to bring our potential 'best-in-class' Duchenne dystrophy project SGT-003 to patients
.
At the same time, innovative gene therapies have expanded our pipeline, allowing us to begin to meet the significant unfinished needs
of other rare disease indications.
”

As WuXi AppTec's CTDMO focused on cell and gene therapies, WuXi Biologics is committed to accelerating and transforming the development, testing, production and commercialization
of gene and cell therapies and other high-end therapies.
WuXi Sheng is able to help customers around the world bring more innovative therapies to market early for the benefit of patients
.
If you have relevant business needs, please click on the picture below to fill in the specific information
.