Welcome to the 3rd Annual Gene Therapy for Blood Disorders

Ensuring Safety & Durability for Gene Therapy & Gene Editing Approaches for Hemophilia, Beta-thalasemia & Sickle Cell Disease

In the context of intense scrutiny over the safety and durability of blood disease-targeted gene therapies and a flurry of excitement around novel gene editing technologies, the 3rd Gene Therapy for Blood Disorders Summit will unite the leading large pharma and innovative biotechs to share essential learnings from across this rapidly evolving field, enabling you to progress your pipeline to address the significant unmet need in this field.

Focused specifically on hemophilia, beta-thallasemia and sickle cell-directed gene therapies and gene editing approaches, this event is an invaluable opportunity to learn the realities of durability challenges, understand strategies to guarantee safety at every stage of development and get a head-start in understanding the most promising technologies set to revolutionise the field.

Incorporating insights from translational, clinical and commercial industry experts, as well as leading clinicians actively involved in late-stage trials, this is your opportunity to investigate the realities of managing immunogenicity for systemically administered gene therapies, establishing less cytotoxic conditioning regimens for ex vivo approaches and prepare for the commercial reality of launching a gene therapy and ensuring there is widespread patient adoption.

Health & Safety

The health, safety and well-being of our community remains our highest priority, therefore, all attendees will be required to complete a health declaration prior to attending the event, as well as comply with the City of Boston’s B Together policy. This policy requires individuals to show you proof of vaccination against COVID-19 in order to gain admittance to the conference venue. For further information around Health and Safety onsite, please visit our FAQs page here or for more information on the B Together Policy, please see here.

* Please note, this is subject to change at any time without prior notice.

Expert Speakers Include

Adam Wufsus

Medical Director Rare Blood Disorders

Novo Nordisk

Clark Paramore

Head of Value Demonstration & HEOR

bluebird bio

Gabriella Denning

VP Research & Development

Expression Therapeutics

Ian Winburn

Global Medical Lead Haemophilia


Meagan O’Brien

Senior Medical Director


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