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Patients’ perspective on Genome Editing

TIF Board Member and Expert Patient George Constantinou engaged in an open, multi-stakeholder discussion on crucial issues that will shape the future of advanced therapy medical products, including genome editing technologies, in the European Union. 

 

Location: European Parliament, Brussels

Date: 16 October 2018

Event:Conversations on Science, Regulation and Society – the Future of Genome Editing in European Healthcare Systems’’ Conference, co-organised by the Alliance for Regenerative Medicine and Intellia Therapeutics, and hosted by Mrs. Teresa Giménez Barbat, Member of the European Parliament, in the premises of the European Parliament 

 

Summary:

During this conference, George Constantinou gave a presentation on “Genome Editing in thalassaemia” and capitalised on the patients’ perspective seeking answers to key patient questions such as the following.

  1. Will Gene Editing lead to my complete cure? Am I going to live free of blood transfusions, iron chelation therapy, hospitalizations, pain etc.)
  2. Will the increase of HbF make me feel better than today’s care in the Western World where my Hb level is kept between 9-10.5 g/dl throughout? (I understand that in this mindset, patients in the developing world will have more to benefit from such progress).
  3. Will I inherit side effects from the curative treatment (Gene Editing) that will make me feel worse and have a poorer quality of life than before receiving the treatment?
  4. On the whole, will Gene Editing be a simpler and safer process than Gene Therapy or Haemopoietic Stem Cell Transplantation?
  5. The challenges that countries of the developing world are facing in the treatment of thalassaemia are different and perhaps patients in the developing world will benefit more from such an innovative treatment. But what are the criteria for inclusion?
  6. Are age and clinical status included in the eligibility/inclusion criteria? A big percentage of patients in the developing world have poor health, consequent to suboptimal or no treatment.
  7. Will there be ongoing research for further development of the technology to minimize or even ensure ZERO off-target effects?

Download the presentation

Patients today have a voice! It is important to ensure that it is heard!  #PatientAdvocacy

 

More information coming soon