Prediction Not checkable as stated
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.”
Assertion Supported
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.”
Insight
Tran: CRISPR is evolving into a cellular search engine platform
“Instead of just this pair of molecular scissors that everyone talks about, the biggest recent shift in the field is this push towards making CRISPR a search engine platform of the cell.”
Insight
Andy Tran: Gene therapy winners will pair platforms with disease-specific optimization
“Regardless of the type of vector that you're engineering, we believe the winners in the space are the ones that could harness a specific platform and marry it with very thoughtful optimization towards specific disease areas.”
Prediction Not checkable as stated
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.”
Opinion
Tran: Manufacturing is an overlooked existential threat to gene therapy
“So once you've designed the perfect gene therapy, perhaps one of the most overlooked, but most existential threats to the industry is challenges in manufacturing.”
Insight
Tran: Simple CRISPR knockouts often outperform complex new genome engineering tools
“Many times, the latest, most advanced, mechanistically complex, and by definition least validated system is not the best tool if a simple CRISPR knockout, for instance, can solve your problem.”
Prediction Not checkable as stated
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…”
Opinion
Tran: Gene editing is becoming a top therapeutic tool in history
“Technology is used to rewrite our genetic code is becoming one of the most powerful therapeutic arsenals of all time.”
Assertion Supported
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.”
Assertion Supported
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.”
Assertion Supported
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.”
Assertion Supported
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.”
Assertion Open · timeframe Oct 2024
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.”
Assertion Supported
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.”
Assertion Supported
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.”
Assertion Contradicted
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.”
Assertion Supported
Tran: Current genome editing tools face immunogenicity and off-target issues
“Once it is inside the cell, many of our current protein-based genome editing nucleases are still plagued by off-target effects, low efficiency, and side effects from the immunogenicity of the protein itself.”
Assertion Not checkable as stated
Tran: Big biotech giants seek acquisitions in viral vector processing
“A lot of these big biotech product development giants are looking to acquire, you know, smaller companies that have deep expertise in viral vector processing.”
