Next Generation Therapy:
Journey from clinical to commercial
Cell & gene therapy in ophthalmology
What happens when the mutation causing a disease is also key to unlocking its treatment? Beghou's Vishal Singal sits down with Dr. Marianna Weener, Retinal Physician Scientist at the Broad Institute of MIT and Harvard, and Lulia Kim, an individual living with retinitis pigmentosa, to explore the clinical and patient perspectives on this disease and the outlook for cell & gene therapies in ophthalmology.
What you will hear:
- How a repetitive mutation could be a “founder effect,” and how such genetic variants may form the basis for treatment
- Why gene editing, rather than gene replacement, may be the preferred treatment tool in an ophthalmologic disease state, and why some forms of editing are considered safer than others.
- The gap between the kinds of endpoints currently required for regulatory approval versus those that are more realistic for patients with slowly progressing retinal disease
- What next steps look like: safety confirmation, haplotype mapping, patient registry building, and screening of editing tools
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How to make cell & gene therapy commercialization work:
Key learnings from leaders who’ve been there
Cell and gene therapies promise breakthrough outcomes, but many stumble at launch. In this panel, leaders from Novartis, Orca Bio, BioMarin, and Beghou share real-world lessons on what drives CGT success, from cross-functional collaboration to adapting models to meet commercialization challenges.
Additionally, listen to our two-part podcast series featuring Rita (COO, ARM), which focuses on navigating CGT complexities.
Caught beneath the surface: The hidden potential of patient data
AI-powered insights from structured and unstructured data speed diagnoses, reveal patient behaviors, and enable more personalized care.
Taming the Complexity of CGT Commercialization
How five strategic levers help companies design an executable CGT commercialization model.
Initial CGT launches faltered. The next wave doesn’t have to.
Explore four key considerations for commercializing the next generation of potentially curative therapies.
“Cell & gene therapies work, but the challenge of modern medicine is to identify the right unmet need in a disease area, provide equitable access to patients, and scale the manufacturing and distribution in order to transform patient lives.”
The Beghou difference
AI that fits your world and grows with it.
We design AI strategies grounded in your therapeutic context and aligned with real business goals. Combining deep life sciences expertise with data science rigor, we deliver adaptable solutions that drive adoption, create long-term value, and scale with your organization.
Our approach centers on practical, high-impact meta use cases with measurable ROI. We work across commercial, medical, and IT teams to ensure AI becomes an integrated part of your operations. And with compliance, security, and scalability built in from the start, you get technology you can trust and outcomes you can track.
