Gene therapy
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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