Cell and Gene Therapy

Since Elevidys’ accelerated approval in 2023, experts have been clamoring for more data, particularly in older and non-ambulatory children. New results, presented Friday, show mobility improvements in 8- to 9-year-old patients after one year of follow-up.

Lilly will use Rznomics’ proprietary ribozyme technology to develop RNA editing therapies for congenital hearing loss.

After warnings that the dragged-out process was putting the cell therapy company at risk of bankruptcy, bluebird bio now has a new deal to offer shareholders.

It’s another wild twist in the story of Galapagos, a company that has been around for more than 25 years but has yet to get a therapy approved.

The cell engineering company, co-founded by oncologist and writer Siddhartha Mukherjee, does not see a path forward for its pipeline of early-stage cell therapies for two different types of cancer.

CRISPR Therapeutics’ partner Vertex reported that more than 65 treatment centers have been activated for the gene therapy Casgevy. While Vertex handles the market, CRISPR has been focused on its clinical program.

A new executive order aims to smooth the path for getting U.S. manufacturing facilities up and running; HHS says it will require placebo-controlled trials for all vaccine approvals; tariff threats hit BioNTech; Novo Nordisk’s FDA application for an oral version of Wegovy is accepted; and more.

The cell and gene therapy space in recent months has hit several speedbumps, including layoffs, dropped drugs and discontinued partnerships.

As Q1 2025 earnings season continues, tariffs remain top of mind for pharma CEOs and investors. Meanwhile, the American Association for Cancer Research’s annual event kicks off this year’s oncology conference season. Plus, will the FDA become politicized under HHS Secretary RFK Jr.?

The condition, recessive dystrophic epidermolysis bullosa, causes chronic wounds and has an 84% mortality rate by age 40.