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Media & Perspectives

The work behind the breakthrough.

Behind every milestone are families, scientists, clinicians, and development teams working to move genetic medicines from possibility toward patients. Explore the research, partnerships, clinical milestones, and stories shaping our work across rare and ultra-rare diseases.

Featured Story

Clinical Milestone

The First FOXG1 Patient has Received FRF-001

After years of determination by FOXG1 families, scientists, and the FOXG1 Research Foundation, the first child received FRF-001 in the Foundation's first-in-human gene therapy trial. Aurelix founder Dr. Brandon Henry serves as the Foundation's Chief Medical Officer. For the FOXG1 community, this milestone represents a long-awaited step from the laboratory toward the possibility of treatment.

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Source

FOXG1 Research Foundation

Published

September 2026

The Newsroom

Stories from the work.

Clinical milestones, development news, scientific research, and perspectives from across the rare-disease community.

Leadership News

September 2026

Raiden Science Foundation Introduces Dr. Brandon Henry as Chief Drug Development Officer

The Raiden Science Foundation announced Dr. Brandon Henry as Chief Drug Development Officer as it advances its UBA5 gene therapy program toward clinical investigation. Dr. Henry also leads Aurelix Bio, which provides integrated development expertise and program leadership in support of Raiden's mission to create a treatment opportunity for children affected by UBA5 deficiency.

Raiden Science Foundation / LinkedIn

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Partner & Development News

September 2026

Raiden Science Foundation Introduces Aurelix Bio as a Development Partner

The Raiden Science Foundation introduced Aurelix Bio as a key development partner in its effort to bring an investigational UBA5 gene therapy toward the clinic. Aurelix is helping lead the scientific, regulatory, clinical, and operational development work needed to turn a promising therapeutic program into a clinical opportunity for children and families affected by this ultra-rare disease.

Raiden Science Foundation / LinkedIn

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Clinical Milestone

September 2026

The First FOXG1 Patient has Received FRF-001

After years of determination by FOXG1 families, scientists, and the FOXG1 Research Foundation, the first child received FRF-001 in the Foundation's first-in-human gene therapy trial. Aurelix founder Dr. Brandon Henry serves as the Foundation's Chief Medical Officer. For the FOXG1 community, this milestone represents a long-awaited step from the laboratory toward the possibility of treatment.

FOXG1 Research Foundation

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Regulatory News

August 2026

Galibra Neuroscience Receives FDA Orphan Drug and Rare Pediatric Disease Designations for SSADH Deficiency Gene Therapy

Galibra Neuroscience received FDA Orphan Drug and Rare Pediatric Disease Designations for its investigational gene therapy for SSADH deficiency. Aurelix Bio serves as a development partner and regulatory strategy team for the program, supporting the work required to translate promising science into a viable clinical development pathway for this rare neurological disease.

Galibra Neuroscience / PR Newswire

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Partner & Development News

August 2026

Genezen and the Raiden Science Foundation Announce Partnership for AAV9 Gene Replacement Therapy for UBA5 Clinical Trial

The Raiden Science Foundation and Genezen announced a manufacturing partnership to advance Raiden's investigational AAV9 gene replacement therapy for UBA5 deficiency. The collaboration marks an important step in preparing the program for clinical investigation. Aurelix Bio works alongside Raiden as a development partner, helping connect the scientific, manufacturing, regulatory, and clinical activities needed to move the program forward.

Genezen / Raiden Science Foundation / PR Newswire

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In the Media

June 2026

Brandon Michael Henry: Interpreting Complement Activation After AAV Gene Therapy

Hemostasis Today highlights Dr. Brandon Henry's perspective on one of the important safety challenges in AAV gene therapy: distinguishing laboratory signals of complement activation from patterns that may indicate clinically meaningful toxicity. The coverage reflects the scientific and clinical judgment required to develop genetic medicines responsibly.

Hemostasis Today

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Research & Publications

June 2026

Laboratory Monitoring of Complement Activation in Gene Therapy: Analytical Pitfalls and Clinical Interpretation

Safe clinical development of AAV gene therapies requires more than measuring laboratory abnormalities—it requires understanding what those changes mean for patients. In this peer-reviewed work, Dr. Brandon Henry and coauthors examine the interpretation of complement biomarkers following gene therapy, including the analytical and clinical challenges involved in recognizing potentially significant toxicity.

Seminars in Thrombosis and Hemostasis

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Clinical Milestone

June 2026

Riaan Receives First Gene Therapy for Cockayne Syndrome

After nearly five years of extraordinary work by Riaan's family and the Riaan Research Initiative, Riaan became the first patient to receive an investigational gene therapy for Cockayne syndrome. Dr. Brandon Henry serves as a key advisor to the Foundation, providing clinical, scientific, and regulatory guidance with support from the Aurelix Bio team, including work supporting the program's IND and path to first treatment.

Riaan Research Initiative

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Leadership News

February 2026

FOXG1 Research Foundation Names Dr. Brandon M. Henry Chief Medical Officer

The FOXG1 Research Foundation named Aurelix founder Dr. Brandon Henry as Chief Medical Officer as the Foundation prepared to advance FRF-001 into first-in-human clinical investigation. The appointment brought together the Foundation's parent-led mission and clinical development leadership focused on building a potential treatment pathway for children and families living with FOXG1 syndrome.

FOXG1 Research Foundation

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Research & Publications

February 2026

When Mechanism Becomes Evidence: The FDA's Plausible Mechanism Pathway and the Future of Rare Disease Development

For many ultra-rare diseases, conventional evidence-generation models present substantial challenges. In this article, published in IFAPP Today: the Global Pharmaceutical Medicine Journal, Dr. Brandon Henry examines the FDA's plausible-mechanism approach and its implications for creating scientifically rigorous, more feasible development pathways for therapies intended for very small patient populations.

IFAPP Today

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The Work Continues

Every breakthrough begins with people who refuse to give up.

From families pursuing a treatment to scientists advancing a discovery, meaningful progress is built together. If you're working to change what's possible for people living with a rare disease, we'd welcome the conversation.

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