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.
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.
Former Biogen R&D head Al Sandrock defines the agility of a small company not just as speed, but as the ability to make decisions by informally gathering key people in the hallway, bypassing the need to schedule formal meetings. This contrasts with large organizations where many more people and committees are necessarily involved.
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.
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.
Voyager CEO Al Sandrock supports the FDA's use of accelerated approval for severe diseases but argues it must be coupled with industry accountability. He praises Amelix for voluntarily pulling its ALS drug after a failed confirmatory trial, framing such responsible actions as essential for maintaining the FDA's willingness to be flexible with approvals based on surrogate endpoints.