Gene editing pioneer David Liu is developing a platform that could treat multiple, unrelated genetic diseases with a single therapeutic. By editing tRNAs to overcome common nonsense mutations, one therapy could address a wide range of conditions, dramatically increasing scalability and reducing costs.

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Gordian Biotechnology embeds unique genetic "barcodes" into hundreds of different gene therapies. This transforms gene therapy from a treatment modality into a high-throughput screening tool, allowing them to test many potential drugs simultaneously inside a single living animal and trace which ones worked.

To overcome regulatory hurdles for "N-of-1" medicines, researchers are using an "umbrella clinical trial" strategy. This approach keeps core components like the delivery system constant while only varying the patient-specific guide RNA, potentially allowing the FDA to approve the platform itself, not just a single drug.

To normalize the ethically fraught practice of embryo gene editing, startups like Preventive are shifting the narrative from just curing disease to radical cost reduction. They claim editing embryos could cost $5,000, a fraction of the $2 million price tag for current adult gene therapies.

The tech world is fixated on trivial AI uses while monumental breakthroughs in healthcare go underappreciated. Innovations like CRISPR and GLP-1s can solve systemic problems like chronic disease and rising healthcare costs, offering far greater societal ROI and impact on longevity than current AI chatbots.

CRISPR reframes its commercial strategy away from traditional drug launches. By viewing gene editing as a 'molecular surgery,' the company adopts a go-to-market approach similar to medical devices, focusing on paradigm shifts in hospital procedures and physician training.

The commercial advantage of one-time CRISPR/Cas9 therapies is shrinking. Advancements in RNA modalities like siRNA now offer durable, long-lasting effects with a potentially safer profile. This creates a challenging risk-reward calculation for permanent gene edits in diseases where both technologies are applicable, especially as investor sentiment sours on CRISPR's long-term safety.

In a meeting with political figures, gene editing pioneer David Liu set an audacious public goal of achieving 1,000 bespoke "N-of-1" cures, similar to the famous Baby KJ case, by 2030. This marks a shift towards public accountability and sets a quantitative benchmark for the entire precision medicine field.

While complex gene editing may be challenging in vivo, Colonia's platform presents a novel opportunity: targeting different immune cell types (e.g., T-cells and NK cells) with distinct payloads in a single treatment. This could create synergistic, multi-pronged attacks on tumors, a paradigm distinct from current ex vivo methods which focus on engineering a single cell type.

"China Speed," once synonymous with rapid antibody development, now extends to RNA silencing technologies. A surge in homegrown RNAi companies and programs, with dozens unpartnered, indicates China's biotech ecosystem is rapidly diversifying into new, complex therapeutic modalities beyond its established strengths.

Beyond transient RNA, Create has developed a unique retrotransposon based on the human Line-1 element. This technology allows for stable, scarless gene delivery using only RNA, providing an option for durable expression (e.g., for CD19 CAR-T) alongside their transient approaches, creating a highly versatile platform.

David Liu's Lab Pursues a Single Gene Editor to Treat Multiple Diseases | RiffOn