A Global Alliance to Save Lives: How Two Parents and PackGene Are Redefining What's Possible in Gene Therapy with Help from Weill Cornell Medicine

HOUSTON, Jan. 8, 2025 /PRNewswire/ -- Two Australian parents, faced with the unimaginable prospect of losing their 14-month-old daughter to the ultra-rare neurological disease Hereditary Spastic Paraplegia Type 56 (SPG56), sold their home and everything they owned to save their daughter. Their act of courage led to the founding of Australian non-profit Genetic Cures for Kids (GC4K) and sparked a global movement. Today GC4k announced a collaboration with cutting-edge biotech leader PackGene Biotech Inc. and the Belfer Gene Therapy Core Facility at Weill Cornell Medicine to potentially deliver a new kind of hope: an accessible, custom-tailored gene therapy solution.

A Small Team Taking on a Vast Medical Challenge
SPG56 is a progressive, degenerative disorder that typically begins in early childhood. Children like Tallulah—now five years old—experience a loss of motor and cognitive abilities as the disease advances. Diagnosing SPG56 is often an uphill battle, compounded by limited genetic testing and the ultra-rare nature of the condition. Fewer than one in a million people worldwide are diagnosed, leaving most families in the dark and without treatment options.

But this initiative goes beyond a single disease. Genetic Cures for Kids, born from one family's desperate determination, aims to create a replicable framework for tackling over 7,000 other genetic conditions that currently have no cure. In doing so, this effort transforms a personal fight into a global blueprint, ensuring that treatments will not remain locked behind paywalls or profit margins.

Uniting World-Class Expertise with Grassroots Resolve

• Weill Cornell Medicine's Belfer Gene Therapy Core Facility: The facility has tailored its AAV (adeno-associated virus) manufacturing capabilities specifically to serve small, patient-driven projects.
  
  
• PackGene Biotech Inc.: A world-leading CRO and CDMO specializes in providing innovative manufacturing solutions to accelerate and make gene therapy affordable. It offers comprehensive services, including plasmid DNA, viral vector, and mRNA production, supporting programs from research to commercialization. After meeting Tallulah and witnessing the human impact of their work, PackGene pledged to subsidize costs and fast-track the development of a clinical-grade gene therapy.

"Our work at the Belfer Gene Therapy Core Facility is driven by a commitment to making gene therapy accessible to all patients, especially those with rare diseases like SPG56," said Stephen M. Kaminsky, Ph.D., Professor of Research in Genetic Medicine at Weill Cornell Medicine. "Together with my colleague Professor Dolan Sondhi, our long-established track record in supporting therapeutic development programs, combined with our expertise in AAV vector manufacturing, is being harnessed to potentially deliver life-changing treatments for Tallulah. This effort also serves as an example for addressing thousands of other rare genetic conditions."

"Our collaboration with Genetic Cures for Kids and Weill Cornell Medicine's Belfer Gene Therapy Core Facility is a testament to the power of compassion paired with scientific excellence," said Dr. Xin Swanson, Chief Strategy Officer of PackGene. "We are leveraging our expertise not just to create plasmid DNA and AAV vectors, but to reshape the future landscape of gene therapy—making it affordable, accessible, and deeply human."

Why This Matters: A Call to Arms for the Global Community

Pharmaceutical giants often prioritize large patient populations and high-return therapies. This project proves that even ultra-rare diseases can draw international attention, funding, and breakthrough innovations. The collaboration harnesses top-tier science to deliver tailored solutions for a handful of children—an approach that can be replicated for thousands of other conditions currently deemed "too rare" to warrant significant investment.

"As the mother of a child living with SPG56, I've come to understand that families like ours are not just fighting diseases—we're fighting for the attention, resources, and urgency that our children's lives deserve," said Golden Whitrod, founder of Genetic Cures for Kids and mother to Tallulah Moon. "Until governments seriously invest in the credible and worthwhile market of personalized medicine, family foundations like ours will continue to lead the charge. But we cannot do it alone. The generosity and philanthropy of companies like PackGene and institutions like Weill Cornell Medicine remind us that compassion and innovation can change the world, one life at a time."

This effort also highlights the transformative potential of gene therapy. By focusing on SPG56, the alliance is setting a precedent for tackling thousands of other genetic conditions that currently have no cure, building a global blueprint for change.

AAV9: Turning a Virus into a Lifesaver
In a stroke of medical ingenuity, the team is using a harmless virus, AAV9, as a "good virus" to deliver the healthy gene directly into patient cells. In other words, they are fighting disease with a re-engineered virus. It's a sensational pivot: what once caused harm now paves the way to healing, giving children a real chance at regaining their futures.

Join the Mission: Every Contribution Counts
This story began with two parents who refused to accept "no cure" as an answer. It now encompasses a global network of families, researchers, clinicians, and donors, all working toward the same goal: delivering cures, not excuses.

Be part of this historic effort. Visit www.ourmoonsmission.org to learn more, contribute, and discover how a united global community can bring life-changing therapies from the lab to the children who need them most.

Images for Media: https://photos.app.goo.gl/ksKpVg3mrbjMDZmL9 (see sources in image info: Genetic Cures for Kids, PackGene, Weil-Cornell Medicine)

Contacts:

PackGene Biotech Inc. , Corporate, Irene Song, Senior Director of Marketing, [email protected], 1-732-778-8546, https://www.packgene.com/

For media, Susan Thomas, Principal Endpoint Communications, [email protected], 1-619-540-9195

Genetic Cures for Kids Media Contact: Golden Whitrod, Co-founder GC4K, [email protected], +61 449 154 411, www.ourmoonsmission.org

SOURCE Packgene