Krystal Biotech became the second company to secure the FDA’s platform technology designation for a genetically modified, non‑replicating herpes simplex virus type platform, which can streamline review for multiple products built on the same vector platform. The designation is intended to speed regulatory pathways for follow‑on assets using the registered platform. In parallel, an AAV‑delivered PPT1 gene therapy produced encouraging preclinical results for CLN1 disease, demonstrating potential in a devastating pediatric neurodegenerative condition. The two developments exemplify ongoing momentum for in vivo genetic medicines and the regulatory pathways being refined to handle platform‑based approvals. Platform designations and positive gene‑therapy preclinical/early clinical data both accelerate investor and developer interest, but companies noted the need for rigorous safety characterization and manufacturing scale to convert platform advantages into approved products.