Twice a year, the Alliance for Regenerative Medicine (ARM) surveys the cell and gene therapy landscape and ARM’s initiatives to advance the sector and provide an update to their members, their stakeholders, and the public at large. While external conditions ebb and flow, the sector’s progress — in clinical milestones, regulatory developments, and patient impact — steadily advances. It has already been a record year for the approval of new gene therapies to treat rare diseases. Two Chimeric Antigen Receptor T-cell (CAR-T) therapies for blood cancers have now been approved by the US Food and Drug Administration (FDA) as earlier-line treatments. The approval of new CAR-Ts this year in the US and Europe now means that a total of six CAR-Ts are available to patients in both regions. Ten years after the discovery of CRISPR, we are less than a year away from possible FDA approval of the first CRISPR therapy — for sickle cell disease, a once overlooked disorder that is now the single most targeted rare disease in ongoing regenerative medicine clinical trials. In June, a new version of gene editing, base editing, entered the clinic for the first time to treat familial hypercholesterolemia, a common cause of heart disease.

Read more…

Alliance for Regenerative Medicine

When you partner with Avance Biosciences™, you gain a CRO partner that is creative, collaborative, and dedicated to sound science with a focus on your specific regulatory requirements Contact our technical staff to discuss how we can support you in your project!