Overcome Clinical, Manufacturing, Regulatory & Commercial Challenges from Clinical Trial Initiation Through to Approval and Turn the Significant Potential of Gene Therapies into a Commercial Reality

Welcome to the 7th Gene Therapy for Rare Disorders Summit!

This past year has seen more gene therapies approved by the FDA than the previous five years combined, and almost triple the number of clinical trials in gene therapy have been initiated in H1 of 2023 compared to the same time frame in the previous year. 

To maximize the value of your late-stage gene therapy assets, the 7th Gene Therapy for Rare Disorders Summit is returning to tackle head-on the significant clinical, manufacturing, regulatory, and commercial challenges facing gene therapy programs today. 

Bringing together key figures from industry, regulatory bodies, patient non-profits, payers, and key service providers, this is the field’s foremost meeting for collectively progressing rare gene therapy programs into and through the clinic.  

Join us to leverage essential insights from over 250 peers from leading biopharma companies with direct experience in driving gene therapies through the clinic and into the market. Keep your finger on the pulse, form connections with top decision-makers, and address your specific clinical, manufacturing, regulatory, and commercial bottlenecks to safeguard against costly delays and progress your gene therapies through the pipeline quickly and safely.

3

DEEP DIVE

TRACKS

9

ADDITIONAL WORKSHOPS &

FOCUS DAYS

70+

EXPERT SPEAKERS

SHARING INSIGHTS

150+

COMPANIES

REPRESENTED

250+

ATTENDEES TO

NETWORK WITH

The Expert Speaker Faculty Includes:

Liz Fowler

Director, CMS Innovation Center

Centers for Medicare & Medicaid Services

Michael Sherman

Venture Partner

RA Capital Management

Monica Hayhurst Bennett

Head of Gene Therapy Unit, Neuroscience

Novartis

Pat Furlong

Founding President & Chief Executive Officer

Parent Project Muscular Dystrophy

Peter Marks

Director

FDA

Richard Colvin

Chief Medical Officer

bluebird bio

Sheila Mikhail

Co-Founder & Advisor

AskBio

Tim Hunt

Chief Executive Officer

Alliance for Regenerative Medicine

GTx Rare 2
GTx 5
GTx Rare 1

Lead Partner:

precision for medicine

Expertise Partner:

azzur_group

Program Partners:

AGC Biologics
advancedmedicinepartners_logo
Matica Logo
Vinta Logo

Hosting Partner:

Worldwide Clinical Trials logo

Exhibition Partners:

Forge Biologics Logo
cryoport systems - logo
polysciences
paysign logo
sgs

Program Partners:

AGC Biologics
Matica Logo
Vinta Logo
advancedmedicinepartners_logo

Expertise Partner:

Hosting Partner:

azzur_group
Worldwide Clinical Trials logo

Exhibition Partners:

Forge Biologics Logo
cryoport systems - logo
polysciences
paysign logo
sgs

Event Partners:

bostonbiodevelopment_logo
compliance architects

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