Gene therapy remains attractive when data show new technologies can advance the field, but gene regulation, editing and RNA therapies are grabbing investors’ attention, panelists said at ARM’s Cell and Gene Meeting on the Mesa.
The genetic mutation that causes sickle cell disease was first discovered in 1956, but only recently have any drugs have been approved to treat the condition. None, however, show significant impacts on organ disease, and there are great unmet needs for prophylaxis as well as severe disease in acute care settings. On October 12, biopharma companies came together to discuss this at the Cell & Gene Meeting on the Mesa, hosted by the Alliance for Regenerative Medicine.
The emerging field of cardiovascular cell and gene therapies shows great promise to significantly improve clinical outcomes, but it still faces key challenges, according to an October 13 plenary session at the Cell & Gene Meeting on the Mesa.
One big problem that cell and gene therapies face is that reimbursement systems across the world, particularly in Europe and the US, have been built around providing access to chronic disease treatments administered over a long period of time, said Kristin Wolff, bluebird bio’s vice-president, global policy strategy and US government payer. She was speaking at a panel on strategies for managing access and reimbursement challenges at this week’s Cell and Gene Meeting on the Mesa, organized by the Alliance for Regenerative Medicine.
There are more opportunities in the cell and gene therapy sector than ever. But to profit from them, investors must be more discerning than in the past, according to a panel of investors at the Cell & Gene Meeting on the Mesa on October 14.