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Novartis secures licenses to Mesoblast’s Cell Therapy for treatment of COVID-19

Novartis has recently secured a global license to Mesoblast’s cell therapy remestemcel-L in the treatment of COVID-19.

Under the terms of the agreement, Mesoblast is to receive a $50 million upfront and the support of a partner that could help address critical quality attribute concerns raised by the Food and Drug Administration (FDA).

Evidence of the prospects of remestemcel-L, a mesenchymal stem cell therapy, in COVID-19-related ARDS are expected when the 300-patient phase 3 trial wraps up early next year.

Novartis plans to commence a phase 3 trial in non-COVID-19 ARDS patients, setting it up to address the broader unmet medical need. ARDS causes 40% mortality in the around 200,000 patients who develop the condition in the United States annually.

Novartis also has an option to distribute remestemcel-L outside of Japan in GvHD and to co-fund its development and commercialization in other non-respiratory indications.

(Source: Fierce Biotech, 2020)

UCL’s Novel CAR-T cell therapy shows promising early results in children with neuroblastoma

A novel CAR-T cell therapy developed by researchers at UCL and designed to target cancerous tumors has shown promising early results in children with neuroblastoma.

Neuroblastoma is a rare type of cancer that mostly affects babies and young children and develops from specialized nerve cells (neuroblasts) left behind from a baby’s development in the womb.

Researchers from UCL Great Ormond Street Institute for Child Health (GOS ICH) and the UCL Cancer Institute modified the patient’s T-cells, equipping them to recognize and kill neuroblastoma tumor cells.

They found using an ample amount of dose of the modified CAR T-cells was able to induce a rapid reduction in tumor size in some of the patients treated. Also, the CAR T-cells did not cause any harmful side effects in healthy tissues that express the GD2 molecule.

The research team continues to prepare for their next clinical study in collaboration with Autolus, a clinical-stage biopharmaceutical company developing next-generation, programmed T-cell therapies for the treatment of cancer. This study will evaluate AUTO6NG, which builds on this approach utilizing the same GD2 CAR alongside additional programming modules designed to improve efficacy and persistence.

(Source: News Medical, 2020)

Scientists from Trinity College Dublin develops new gene therapy for eye disease

Scientists from Trinity College Dublin have developed a new gene therapy approach that could potentially treat an eye disease, Dominant optic atrophy (DOA) that leads to a progressive loss of vision. 

The results of their recent study were published in “Frontiers in Neuroscience.”

Dominant optic atrophy (DOA) includes symptoms such as moderate vision loss and some color vision defects, but severity varies, symptoms can worsen over time and some people may become blind. There is currently a cure for this eye disease. 

A gene, OPA1, provides instructions for making a protein that is found in cells and tissues throughout the body, and which is important for maintaining proper function in mitochondria, which are the energy producers in cells. Without it, mitochondrial function decreases. 

Without the protein made by OPA1, mitochondrial function is sub-optimal and the mitochondrial network which in healthy cells is well interconnected is highly disrupted.

Through their studies, the scientists have found that the gene therapy was able to protect the visual function of mice and also found that their gene therapy improved mitochondrial performance in human cells that contained mutations in the OPA1 gene, offering hope that it may be effective in people.

(Source: Eurekalert, 2020)