Aldevron

06/19/2026

Today is World Sickle Cell Awareness Day.

We honor the strength of those living with sickle cell disease and trailblazers like Victoria Gray, one of the first people in the U.S. to receive a groundbreaking gene-editing treatment that put her sickle cell into remission.

Her story represents hope, progress, and the power of continued research and advocacy. ❤️

06/19/2026

Breakthroughs in science ultimately come down to moments of human decision.

At last year’s Advanced Beyond, Victoria Gray shared what it meant to say yes to becoming the first patient to receive CRISPR gene editing for sickle cell disease.

Faced with uncertainty, her decision was grounded in understanding—how the therapy worked, the risks involved, and what options remained if it didn’t succeed. From the absence of graft-versus-host disease to the ability to pursue a bone marrow transplant later, each factor played a role.

Her story underscores the reality behind innovation: every advancement depends not only on science, but on the trust and courage of patients willing to take that first step.

Watch the full video: https://youtu.be/Wr9-l-cL3IA

06/19/2026

Today, we recognize Juneteenth: a day that commemorates the end of slavery in the United States and serves as a moment to reflect on history, acknowledge progress, and celebrate community.

We believe that understanding and honoring important historical milestones helps strengthen a culture of respect, awareness, and belonging.

In observance of Juneteenth, our offices will be closed on Friday, June 19th, and will reopen on Monday, June 23rd.

We take this time to reflect, learn, and recognize the importance of continued progress.

06/18/2026

Plasmid DNA remains foundational to gene editing—but not all plasmids are created equal. Conventional designs can introduce challenges that impact editing efficiency, expression durability, and overall cellular health.

In this on-demand webinar, we explore how next-generation Nanoplasmid™ vectors—featuring a minimal, antibiotic‑free backbone—are designed to support stronger, more sustained expression while reducing transfection-related toxicity and transgene silencing.

What you’ll learn
✅ How Nanoplasmid design principles align with modern gene editing needs
✅ Advantages for editing outcomes, cell viability, and expression durability
✅ Case-based insights demonstrating translational relevance
✅Strategies to help de-risk preclinical development and support clinical progression

Hear directly from Tyler Kozisek, Ph.D., and Charissa Quinlan as they share perspectives spanning R&D innovation through scalable, GMP-ready applications.

Access the webinar on demand: https://www.aldevron.com/watch-on-demand-enhancing-gene-editing-outcomes-and-safety?utm_medium=social&utm_source=linkedin&utm_campaign=9437112-gene-editing&utm_term=nanoplasmid

06/18/2026

Innovation in cell and gene therapy is about navigating uncertainty on the way to breakthrough.

At our Advanced Beyond Symposium last June, Tirtha Chakraborty offered an honest perspective on translational science: early on, even the most promising concepts don’t always reveal a clear therapeutic path.

Rather than knowing exactly what drug to build, teams often move forward with multiple candidates—conducting parallel studies to determine what will ultimately succeed. It’s a reminder that the gap between scientific insight and viable therapy is where some of the most critical work happens.

This reality underscores the rigor, adaptability, and decision-making required to turn potential into progress.

Watch the full video: https://ow.ly/2ApH50Zc3pB

06/18/2026

As cell and gene therapies move toward broader patient access, manufacturing strategies need to evolve just as quickly. From early development through commercialization, consistency, flexibility, and regulatory readiness all have to align to support real scale.

This week's blog explores what it takes to move from promising science to scalable treatment approaches, including how process design, infrastructure, and quality considerations shape long-term success.

Read more: https://www.aldevron.com/blog/reach-scalable-treatment-approaches

Dr. Kiran Musunuru challenges personalized CRISPR hype and maps the hurdles to scalable gene therapy for broader patient access.

06/17/2026

What if the key to safer, more effective therapies isn’t turning genes off—but targeting how they’re regulated?

Daniel Bauer shared a defining insight during last year’s Advanced Beyond Symposium: rather than disrupting BCL11A entirely—which plays critical roles across multiple cell types—researchers identified a way to target an erythroid-specific enhancer.

This distinction matters. By focusing on regulatory elements active only in red blood cell precursors, it becomes possible to increase fetal hemoglobin while preserving the gene’s essential functions in other systems like the immune system and brain.

It’s a compelling example of how deep biological understanding—down to chromatin accessibility and cell-type specificity—can translate into more precise and viable therapeutic approaches.

Watch the full video: https://ow.ly/9t3I50ZbX2B

06/16/2026

Some of the most impactful breakthroughs in medicine start with a single observation.
We revisit Daniel Bauer’s conversation around a pivotal discovery: the protective role of fetal hemoglobin in sickle cell disease. First identified in the 1940s, this insight revealed that even modest increases in fetal hemoglobin can significantly improve patient outcomes—including more than a decade of increased survival.

At a molecular level, fetal hemoglobin prevents the polymerization that drives sickling—making it a powerful, naturally occurring therapeutic target.

Today, this foundational understanding continues to shape modern gene editing approaches and therapeutic strategies.

Watch the full video: https://ow.ly/PK9l50ZbWoF

06/15/2026

What was once unimaginable is now redefining medicine.

We’re reflecting back on our Advanced Beyond Symposium this month. In this video, Fyodor Urnov captured a defining truth of this moment in science: the impossible becomes possible when technologies and expertise converge.

From disease-modifying treatments for sickle cell disease to rapid development of personalized therapies, these breakthroughs are the result of chemistry, engineering, biology, and manufacturing working together in sync.

That’s the power of collaboration driving cell and gene therapy forward—and what makes this era so extraordinary to be part of.

Watch the full video: https://youtu.be/DBy5Hjbh1Ok

06/12/2026

Great couple of days in Naples at the Advanced Cell and Gene Therapy Workshop.
It was a rewarding opportunity to engage with a highly motivated group of PhD students focused on advancing RNA and gene therapy.

A standout moment was Aldevron's Ilaria Chiarugi’s session with Abdelilah Mekhloufi from Integrated DNA Technologies, “In Vivo Gene Therapy: The Case of Baby KJ.” The discussion connected real-world application with core scientific principles and led to thoughtful questions and strong engagement from the audience.

Workshops like this play an important role in sharing knowledge, supporting early-career researchers, and continuing to build momentum across the RNA space.

Thank you to the organizers and everyone who participated.