Gene therapy
Kelonia Therapeutics’ in vivo gene placement system is being tapped to help Astellas Pharma expand its portfolio of in vivo CAR-T cell therapies for cancer.
Citing the need for more time to review additional Chemistry, Manufacturing and Controls information, the FDA has extended its target action date for Rocket Pharmaceuticals’ investigational gene therapy by three months.
With last year’s approval of Vertex and CRISPR’s Casgevy, it’s the start of a new era of gene editing. But there are still challenges we must face.
Shares of preclinical genetic medicines company Metagenomi tanked more than 30% on Friday afternoon in a disappointing debut for its initial public offering, bucking the trend of positive IPOs so far this year.
Under the FDA’s compassionate use program, an eyedrop formulation of Krystal Biotech’s Vyjuvek restored the vision of a teenager with the rare genetic disease dystrophic epidermolysis bullosa.
Metagenomi could potentially raise over $100 million if the underwriters exercise their option to purchase additional shares in full, assuming an initial public offering price of $16 per share.
Topline results from a mid-stage study show that 4D Molecular Therapeutics’ investigational gene therapy cut annual rates of Eylea injections by 85% and 89% for the low and high doses, respectively.
This week, Lori, Greg and Tyler discuss the first surge of IPO activity this year plus gene therapy pricing,
CRISPR gene-editing has had its first ever approval in the UK. Will the FDA follow suit? What can patients expect the price tag to be?
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