The Ledger
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Tran predicts 10 to 20 gene therapy approvals annually by 2030
“We're expecting at least 10 to 20 gene therapy approvals per year by the end of the decade.”
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: Metagenomic mining will uncover genome editing systems beyond CRISPR
“As we continue to mine new metagenomic data of new species, it's not crazy to think that perhaps even CRISPR itself is by no means the final frontier of genome engineering.”
Tran: Genome Engineering Will Eventually Treat Common Chronic Diseases
“As the technology, infrastructure, and economics of these drugs continue to progress, it is not too far-fetched to think of a future in which eventually many of our most devastating diseases, even more common chronic ones, can be targeted and treatable with th…”
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: Single-base genome editing enables treatment of previously untreatable SNP diseases
“This limit of precision actually opens the door for addressing an entire field of devastating SNP-based genetic diseases that were previously untreatable before.”
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.”
Andy Tran: AAVs are the only clinically approved gene therapy vectors
“To jump in, probably the natural place to start is on the AV side, as these adeno-associated viruses are the only clinically approved vector so far for gene therapy.”