The Aurelix Track Record
Built through experience.
Proven through execution.
Across rare and ultra-rare diseases, Aurelix Bio and its leadership bring scientific, regulatory, and clinical development experience to the work of building and advancing investigational genetic medicines.
Our 2026 milestones reflect progress across therapeutic programs, regulatory advancement, and first-in-human clinical investigation.
2026 Development Milestones
From scientific promise to clinical progress.
4
IND Clearances
To date in 2026
15+
Therapeutic Programs Supported
Across rare and ultra-rare diseases in 2026
3
First-in-Human AAV Gene Therapy Dosing Milestones
2026
1
First-in-Human Gene Editing Dosing Milestone
Pediatric neurological disease · United States · 2026
Selected 2026 development milestones across programs supported by Aurelix Bio and its leadership. Individual programs may be independently owned or sponsored by partner organizations.
Regulatory Advancement · 2026
Advancing therapies through the FDA.
Selected regulatory milestones across rare and ultra-rare disease programs supported by Aurelix Bio and its leadership.
3
Orphan Drug Designations
ODD · 2026
4
Rare Pediatric Disease Designations
RPDD · 2026
3
Fast Track Designations
FDA Fast Track · 2026
1
RDEP Designation
Rare Disease Evidence Principles · 2026
Clinical Translation
The work becomes real when a patient is treated.
Behind a first-in-human treatment are years of scientific research, therapeutic development, regulatory preparation, and extraordinary effort from families, investigators, and development teams.
Our work across rare-disease programs reflects the commitment required to move investigational genetic medicines from scientific possibility into clinical investigation.
For a child and family who have spent years working toward a treatment opportunity, reaching that first patient is an extraordinary milestone.
Built With the End in Mind
The first patient is a milestone.Not the finish line.
For rare and ultra-rare diseases, advancing an investigational therapy into its first clinical study is an extraordinary achievement.
But the work cannot end there.
From early development onward, we consider the evidence, manufacturing strategy, regulatory pathway, and resources needed to support continued development and pursue treatment opportunities for additional patients.
Our ambition is to build therapeutic programs capable of progressing beyond initial clinical investigation toward sustainable development and broader patient access.
Why the Work Continues
Every milestone matters.Every patient matters more.
The milestones are evidence of progress. The patients are the reason we keep building.