The Ledger
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Andy Tran: Gene Editing Enables Curative Single-Dose Treatments
“Of course, this is certainly a big paradigm shift and very exciting as these technologies have given us the power to precisely target And eliminate the origin of disease, as well as be very curative with just a single dose of the drug.”
Tran: 24 gene therapies slated for Phase 3 trials in 2019
“There are about two dozen more gene therapies that are slated to just have phase three trials in this year alone.”
Andy Tran: Gene therapy startups drove major biotech exits
“In fact, taking a look at the past several years, some of the largest biotech exits have been from companies developing these gene therapies.”
Tran: CRISPR cut gene editing engineering timelines from months to days
“Compared to old tools, engineering was cut down from a few months to a matter of weeks or days.”
Tran: Alternative Cas nucleases offer advantages in size and precision over Cas9
“Some Cas nucleases are much smaller, come from less immunogenic strains, are more precise, or have new mechanisms altogether.”
Tran: CRISPR tools can directly target and edit RNA bases
“We can even now directly target and edit RNA bases, opening up the entire field of transcriptomic editing.”
Tran: FDA and CBER introduced accelerated approval guidance for gene therapies
“This past year we've seen FDA and CBER leaders drum up plans to make a big push on assessing new cell and gene therapies, as well as introducing new guidance on accelerated approvals for these new areas of product development.”