Conference Day One

Hanson wade have taken the decision to cancel this meeting. Please do accept our apologies for any inconvenience or disappointment this will cause. Please register your interest if you would like updates on the meeting or topic.

8:30 am | Pre-Conference & Networking

9:00 am Reviewing the Past 12 Months of IEM Therapeutic Development


  • Comprehensively assess the current therapeutic landscape for a variety of inborn metabolic diseases
  • Highlight the challenges the field must still address to advance treatments for these diseases and meet the currently unmet clinical needs

Working Alongside Clinicians to Identify & Diagnose Patients with Inborn Errors of Metabolism

9:30 am Establishing a Powerful Disease Monitoring Program for Your Rare Disease

  • Antonio Nin Vice President - Clinical Development, Ultragenyx Pharmaceutical Inc


  • Discussing the key attributes of a strong disease monitoring program
  • Explaining the value this disease monitoring platform provides to all stakeholders

10:00 am | Speed Networking & Morning Coffee Break

Clinical Endpoints & Biomarkers: Monitoring Disease and Therapeutic Outcomes

11:00 am Selecting a Clinical Endpoint & Building Rational Behind it

  • Asif Paker Vice President - Clinical Development, SwanBio Therapeutics


  • Identification of endpoints that convey an objective and accurate representation of the patient experience, and which can be measured in a clinical trial setting
  • Selection of endpoints that are meaningful to a broader range of stakeholders, including patients and providers

Clinical Trial Design, Patient Stratification & Ethics

11:30 am Unique Aspects & Challenges of Running Rare Disease Clinical Trials in the EU

  • Tom Pulles Vice President & Head of Medical Affairs & Patient Advocacy, EMEA, Ultragenyx Pharmaceutical Inc


  • Challenges of running rare disease clinical trials in the EU
  • Understanding different EU country rules & regulations
  • Considerations for enrolling CT patients across Europe

12:00 pm | Lunch Break

Optimizing Enzyme Replacement & mRNA Therapy

1:00 pm Advancing Cell Therapies as Living Medicines for Metabolic Diseases

  • Deya Corzo Executive Vice President & Chief Medical Officer, Sigilon Inc.


  • Explore the potential of cell therapy in rare in born metabolic diseases
  • Discuss the advances Sigilon have made in advancing this modality

1:30 pm Evading Immune Responses to AAV Capsids for Gene Therapy Therapeutics


  • Discuss how altering the AAV molecule directly is extending the length of time treatments will work for
  • Explain how patient data is best used to predict the level of immune activation and how this can contribute to ensuring patient safety

14:30 pm | Afternoon Coffee Break

3:30 pm Challenges in Cell Therapy: Mitochondrial Augmentation Therapy for Primary Mitochondrial Disease

  • Noa Sher Chief Scientist, Minovia Therapeutics


  • Drug development challenges given paucity in preclinical models
  • Challenges of developing potency assays for ‘living medicines’
  • Bridging the gap: Maintaining control from preclinical scale to robust manufacturing processes

4:00 pm Panel Discussion: The Future of Gene Therapy Modalities

  • Chris Mason Founder & Chief Scientific Officer, AVROBIO
  • Noa Sher Chief Scientist, Minovia Therapeutics
  • Deya Corzo Executive Vice President & Chief Medical Officer, Sigilon Inc.


  • In this panel session we will discuss how we can translate promising science, current investment and clear opportunity into safe and effective gene therapy for metabolic disease

4:40 pm | End of Day One