Voyager CEO Al Sandrock views partnerships as more than just revenue. He emphasizes that strong scientific collaborations are invaluable because direct interaction between partner scientists accelerates learning and overall progress for both organizations. This intellectual cross-pollination is a key, often overlooked, benefit of partnering out platform technology.

Instead of diversifying across diseases, Kenai is building deep expertise in Parkinson's. Its pipeline addresses different patient needs: replacing lost cells (lead program), repairing existing damaged cells (002), and targeting inherited forms (003), creating a comprehensive disease franchise.

Founder Sean Ainsworth intentionally started his pioneering AAV gene therapy in an ocular setting before any Western approvals existed. Because an intravitreal injection uses a very small vector amount, it provided a significant safety advantage and a manageable way to prove the technology before attempting systemic delivery.

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 field of ophthalmology is particularly well-suited for a hub-and-spoke model because it utilizes a wide range of treatment modalities (small molecules, biologics, devices, gene therapy). This allows a central hub to leverage shared expertise in areas like ocular delivery and regulatory pathways across multiple, diverse spokes.

Voyager CEO Al Sandrock suggests the 30% average efficacy of new Alzheimer's drugs isn't uniform. Instead, some patients may see a complete halt in progression while others see no benefit. He argues the next critical step is predicting these responders, which will determine whether future therapies like anti-tau agents should be added on or used as a replacement.

The success of Praxis's small molecule for a genetic epilepsy presents a strategic alternative to cell and gene therapies. In an era where complex modalities face funding, safety, and commercial hurdles, advanced small molecules offer a viable and potentially more practical path for treating genetic disorders.

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.

Voyager's ALPL-based blood-brain barrier shuttle offers a different pharmacokinetic profile than transferrin receptor shuttles. While delivering a lower initial peak concentration (Cmax), it provides a much longer half-life. This steady, sustained exposure is optimal for therapies that require constant pathway blockage, highlighting a key strategic trade-off in delivery system design.