Dr. Dwi Kemaladewi and her team at the University of Pittsburgh have been diligently working on the development of a novel gene therapy approach to reverse severe muscle wasting in LAMA2-CMD patients. This research group recently secured new funding by UPMC Enterprises to propel this research to the next level. UPMC Enterprises is an investment group committed to advancing groundbreaking therapeutics toward FDA approval.
LAMA2-CMD is caused by various mutations in the LAMA2 gene, resulting in a nonfunctional form of the laminin alpha 2 protein, which typically plays a crucial role in maintaining muscle and nerve structure. Dr. Kemaladewi’s innovative gene therapy approach aims to restore muscle function by increasing the expression of another analogous protein in the body: laminin alpha 1. This protein can compensate for the nonfunctional laminin alpha 2 in LAMA2-CMD, helping to restore muscle function, alleviate symptoms, and reverse disease progression.
As each LAMA2-CMD patient may possess a unique set of genetic mutations, developping a gene therapy that can reverse mutations in all patients is particularly challenging. Dr. Kemaladewi’s approach offers a key advantage by being mutation-independent. Instead of targeting specific mutations in the LAMA2 gene, the approach focuses on elevating levels of laminin alpha 1 which could potentially make it effective for a diverse range of patients with varying mutations.
In collaboration with Dr. Rob Nichols, the team has also engineered the gene therapy to be not only safer but also more cost-effective to produce, two crucial features for any therapy aiming to eventually reach clinical trials. Thanks to the new funding from UPMC Enterprises, Dr. Kemaladewi and her team will continue to advance their research to confirm the safety and effectiveness of this promising gene therapy approach.
Dr. Dwi Kemaladewi has collaborated closely with LAMA2 Europe since 2019, and we will ensure that you stay well-informed about the latest developments in her pioneering research.