Updates on Gene Therapy Research for Rare Diseases During the COVID-19 Pandemic

Research During COVID

Many medical research projects have faced hurdles since the pandemic has started. Regulators have changed development timelines, and many have had to delay their clinical trials due to safety concerns.

But nonetheless, researchers have persevered, doing all they can to continue their research pipeline.

Below, is some of the notable progress that’s been made. Some of this progress includes an increased understanding of what doesn’t work, and some is major headway in understanding what does.

Research Progress

  • Hemophilia – Valoctocogene roxaparvovec was not approved by FDA due to its inability to produce sustained results for more than one year. Now, researchers are working on ways to allow the therapy to be dosed multiple times.
  • Hemophilia A and phenylketonuria (PKU) – New approach that avoids an autoimmune response (delivered by a lipid nanoparticle) has been found.
  • Neurodegenerative diseases- Three new gene therapies are in the works by the creators of the spinal muscular atrophy (SMA) therapy called Zolgensma.
  • GM2 Gangliosidosis- New therapy is in the works which delivers two different genes simultaneously
  • Dravet Syndrome– Gene therapy has been uncovered that incorporates DNA in order to help increase the production of the protein delivered.
  • GM1 Gangliosidosis– Development of a therapy which increases the expression of the protein in both the cells and the cerebral spinal fluid.
  • Hunter syndrome and Fabry disease– New tags attached to the proteins have been developed that help them reach the lysosomes of the targeted cells.
  • Methylmalonic acidemia– New safety monitoring concerns have been addressed and a human trial is moving forward.

You can read more about this research progress and what to look forward to in the realm of gene therapy for rare diseases here.

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