Pipeline & Programs:Pipeline

Solid has a diversified pipeline across neuromuscular and cardiac diseases with indications we believe are characterized by high unmet need, clear mechanistic rationale and significant market opportunities

Program
Research/
Discovery
Preclinical
IND Submission
Phase I/II
Phase III
 
Neuromuscular
 
 
 
 
 
 
 

Preclinical

SGT-003 is a next generation AAV gene therapy candidate that utilizes a novel, muscle-tropic AAV capsid, called AAV-SLB101, to deliver Solid’s proprietary and differentiated nNOS microdystrophin for the treatment of Duchenne.

AAV-SLB101 is a rationally designed AAV capsid developed by Solid to enhance muscle delivery following systemic administration. In in vivo studies evaluating AAV-SLB101 compared to AAV9 using a reporter construct in wild type mice and non-human primates, AAV-SLB101 demonstrated enhanced muscle biodistribution and transgene expression, as well as reduced liver tropism.

Solid’s nNOS microdystrophin encodes for a functional dystrophin protein surrogate that stabilizes essential associated proteins at the muscle membrane, including nNOS. Data from Solid’s clinical trial evaluating the first-generation candidate SGT-001, which uses the AAV9 capsid, showed that expression of the nNOS microdystrophin transgene may have the potential to slow or stop the progression of Duchenne. In studies of SGT-003 performed in the mdx mouse model of Duchenne and in vitro in human Duchenne cell lines, multiple fold increases in microdystrophin expression have been observed in comparison to AAV9, suggesting that therapeutic dose levels using AAV-SLB101 can be lower than first generation candidates.

 

Preclinical

AVB-202 is a novel gene transfer product candidate being developed for the treatment of Friedrich’s ataxia. Friedrich’s ataxia is a rare inherited genetic disease caused by loss of frataxin with both neurological and cardiac manifestations affecting muscle control and coordination, with possible loss of vision and hearing and slurred speech. AVB-202, like other potential gene replacement treatments for Friedrich’s ataxia, is intended to replace the frataxin gene across relevant tissues, with the goal of preventing progression or reversing the course of the disease. AVB-202 is in preclinical development and utilizes a dual route of administration (via both intrathecal and intravenous routes) to more rigorously target disease pathology. Preclinical data from three animal models, including mouse and nonhuman primate, supported preclinical proof of concept. Early findings in preclinical studies demonstrated improved survival and cardiac function, as well as mitochondrial function in mice.

Cardiac
 
 
 
 
 
 
 

Preclinical

AVB-401 is a novel gene transfer product candidate being developed for the treatment of BAG3 mediated dilated cardiomyopathy (BAG3). BAG3 is a rare cardiac disease and is characterized by mutations in the BAG3 gene. Sufficient levels of functional BAG3 are required for healthy cardiac function.

 

Preclinical

Solid is currently researching two undisclosed cardiac programs, for undisclosed cardiac indications.

As we move forward, expect to see a diversified pipeline, including emerging technologies that will be evaluated for their potential to target underlying diseases and bring groundbreaking genetic therapies to more patients