Gain Clarity on New Regulations to Advance Safe, Scalable & Cost-Efficient Gene Therapies From Translation to Commercialization, Expanding Clinical Reach & Patient Access

Welcome to the 8th Gene Therapy Development Summit

Transforming the Future of Gene Therapies for Rare & Common Diseases

With 14 new gene therapy approvals in the last 18 months, billion-dollar collaborations, and the development of treatments for more common conditions, the gene therapy field is rapidly gaining momentum and broadening scope beyond rare diseases. Returning to Boston in March, the 8th Gene Therapy Development Summit, formerly known as the Gene Therapy for Rare Disorders Summit, will unite 200+ gene therapy experts to tackle the field’s biggest challenges of achieving genuine scalability and accessibility to meet growing patient demand.

For the first time, this industry-defining meeting will reflect the field’s growing focus on developing therapies for common diseases, whilst addressing existing hurdles in rare and ultra-rare disorders. This is your unparalleled opportunity to delve into:

The latest global regulatory requirements

Optimizing routes for CNS

Ocular and inner ear disorders

Innovations in capsid engineering and dosing strategies

Increasing volumetric productivity to reduce CoGs

Join your peers to drive gene therapy innovation across translational, clinical, regulatory, and manufacturing tracks. This event is designed to help attendees navigate the entire gene therapy pipeline.

By refining gene delivery platforms, optimizing manufacturing processes, and navigating regulatory pathways, we can revolutionize the future of gene therapies—bringing them to broader patient populations and significantly enhancing clinical outcomes.

World-Class Speaker Faculty Includes:

Anjulika Chawla, bluebird bio

Anjulika Chawla

Vice President, Medical Affairs, Clinical Lead

bluebird bio

Delphine Agathon, Pfizer

Delphine Agathon

Senior Director - Category Clinician

Pfizer

Jie Li, Vertex Pharmaceuticals

Jie Li

Director

Vertex Pharmaceuticals

Olivier Danos, REGENXBIO

Olivier Danos

Chief Scientific Officer

REGENXBIO

Peter Marks, FDA

Peter Marks

Director, CBER

FDA

Sitra Tauscher W, Novartis

Sitra Tauscher-Wisniewski

Vice President - Clinical Research

Novartis

Uditha DeAlwis, Sarepta

Uditha deAlwis

Vice President - Analytical Development & Quality Control

Sarepta

Previously Attending Companies Include:

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Our Partners:

azzur bio
vinta bio
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Worldwide Clinical Trials
Cryoport

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