About Event

Industry’s Leading Rare Gene Therapy Conference

As the gene therapy field progresses, marked by a mounting wave of approvals and programs entering clinical development, Gene Therapy for Rare Disorders returns as industry’s leading meeting for benchmarking success stories and tackling the key challenges that face your clinical therapies today. 

Cutting-edge talks, multistakeholder panel discussions, and expert-led group discussions will leave you with the strategic insights you need to overcome the clinical, manufacturing, regulatory, and commercial challenges posing a risk of costly setbacks and delays to your late-stage programs.

Boasting a program co-created by a faculty of senior decision makers, this is your one-stop shop for rare gene therapy expertise, with content to be covered including: 

What You Missed in 2024:

Plenary Sessions

See Agenda Highlights

Leverage key insights from expert seminar speakers and panellists – including an array of multistakeholder perspectives – into:

- Accelerating rare gene therapy development

- Addressing the complexity of gene therapy investment and funding models

- Progressing the pricing, reimbursement and market access landscape

- Bridging the gap between drug sponsors and patient communities

Clinical Stream
Manufacturing Stream
Regulatory Stream
Decision Makers’ Bootcamp
Gene Editing Innovation Stream
Gene Therapy Beyond Rare Diseases Stream

What Previous Attendees Have to Say:

Untitled design (27)

"The conference offered incredible quality of speakers in all areas associated with the development of a gene therapy product"

Untitled design (26)

"An excellent opportunity to combine scientific discussions and network with the main gene therapy players in the world"

Untitled design (25)

"The quality of speakers was excellent, and the topics were current"

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"The topics of the presentations were fully aligned with my needs and very valuable. I gained insights that I can implement immediately in my work"