Voyager CEO Al Sandrock outlines a focused strategy: remain specialists in neurology, but broaden the therapeutic modalities (gene therapy, proteins, oligonucleotides). This allows them to pursue well-validated CNS targets that are considered "undruggable" by traditional small molecules, which have historically been the only option for crossing the blood-brain barrier.

To overcome on-target, off-tumor toxicity, LabGenius designs antibodies that act like biological computers. These molecules "sample" the density of target receptors on a cell's surface and are engineered to activate and kill only when a specific threshold is met, distinguishing high-expression cancer cells from low-expression healthy cells.

The GSK3 inhibitor was developed for CNS diseases, requiring high specificity and the ability to cross the blood-brain barrier. These same pharmaceutical characteristics—potency and lipophilicity—proved highly advantageous for treating cancer, demonstrating an unexpected but effective therapeutic pivot from neuroscience to oncology.

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.

Actuate’s drug was designed to be highly lipophilic (fat-soluble) to cross the blood-brain barrier for CNS treatment. This same property proved crucial for its success in oncology, as it allows the drug to easily penetrate cancer cell membranes and reach the nucleus.

After several tau-targeting antibodies failed, including J&J's pazdenimab, confidence in blocking extracellular tau is waning. The field's new hope is Biogen’s Biv80, an antisense drug that prevents tau protein production at the mRNA level, a mechanism that has shown potential to reverse pathology in early data.

Many innovative drug designs fail because they are difficult to manufacture. LabGenius's ML platform avoids this by simultaneously optimizing for both biological function (e.g., potency) and "developability." This allows them to explore unconventional molecular designs without hitting a production wall later.

Voyager CEO Al Sandrock explains their AAV capsids are engineered to be so potent at crossing the blood-brain barrier that doses can be an order of magnitude lower than standard. Crucially, the capsids are also designed to *avoid* the liver, directly addressing the toxicity issues that have plagued the field.

Ipsen avoids the high-risk, capital-intensive phase of basic research. Instead, its R&D strategy focuses on licensing promising drug candidates from universities and biotechs. The company then leverages its expertise in later-stage development, including toxicology, manufacturing scale-up (CMC), and clinical trials, to bring these de-risked assets to market.