Vivet will be attending and presenting during the 6th annual Gene Therapy for Rare Disorders in Boston.

2022 has undoubtedly been a landmark year for the space with much-needed approvals in the summer marking the first time we have seen multiple approvals in a calendar year from the FDA. Gene Therapy for Rare Disorders 2023 will benchmark these breakthroughs, leverage strategic insights from recent success stories, and bring in-depth insights from experts

During the “Defining Value of Rare Gene Therapies” session, Vivet Therapeutic’s CEO & Co-Founder, Jean-Philippe Combal, will take part in the following panel discussion: “Navigating a Competitive Market & Establishing a Differentiated Product” (March 21, 2pm EST).

Topics to be discussed in this panel discussion will include:

👉 Exploring precompetitive collaboration to support progress to approval

👉 Navigating landscapes where multiple gene therapies are being developed for a specific disease

👉 Understanding value demonstration where standard of care exists and use of RWD to better understand disease severity

Vivet will be attending and presenting at the EPA World Congress in Amsterdam – March 21-23, 2023.


Thomas Daniel-Robin, Vivet Therapeutic’s BD Director, will take part in the HTA session and give the following presentation: “Healthcare databases versus current practice – gaps in information for patients, payers and physicians in Wilson Disease” (March 21, 4.25-5.30pm CET).

Topics to be discussed on the potential of national healthcare databases will include:

👉 Generating Real World Data for Rare Diseases: large cohorts and long-term follow-up

👉 Identifying data gaps between Real World Data and current practice: what can and cannot be captured by physicians and patients

👉 Finding the unexpected and understanding the value of enriched studies
From November 14th to 17th, Vivet will be attending and sharing our gene therapy expertise at the World Orphan Drug Congress 2022 in Sitjes (Spain).

Anne Douar, Vivet Therapeutics Chief Development Officer, will take part in the panel «Developing gene therapies for rare disease patients » on November 15 at 2 pm. She will also be a panel speaker on “Key considerations on cell & gene therapy manufacturing for commercialization” on November 17 at 10:10 am

Jean-Philippe Combal, Vivet Therapeutics Co-Founder and CEO, will participate in the panel “Designing first in human gene therapy clinical trials”, discussing gene therapy platform approaches for potential re-dosing strategy on November 15 at 3:10 pm
On November 12th and 13th, the Spanish Wilson disease association of patients and patients’ families is holding its annual conference in Valencia, Spain.

On that occasion, Dr. Gloria González-Aseguinolaza, Vivet Therapeutics CSO and FIMA Deputy Director will give an update on the development of gene therapies for Wilson disease. Don’t miss her talk on Saturday November 12th, from 10:45-11:15 am.

Thomas Daniel-Robin, BD Director, will be attending ISPOR Europe 2022 held in Vienna from November 6-9 to present a poster on recently published data on Wilson disease.

This leading European conference for Health Economics and Outcomes Research is the ideal place to present the results of Vivet’s study of Wilson disease patients data in the French national SNDS database / Health Data Hub (HDH):

Close to 2,000 patients have been identified in this database and more than 1,500 patients analyzed for up to 11 years. A great basis for better understanding and disease awareness.
👉 12% received disability compensation during the study period (2009-2019)
👉 27.2% took over six months’ sick leave and 75% were hospitalized during the year post diagnosis
👉 And 34-42% were hospitalized each year during the 10-year follow-up period

These results further highlight the high unmet medical needs in Wilson disease.

👉 56% of them did not receive standard of care medication
👉 67% showed at least one clinical manifestation post diagnosis and still 56% have clinical manifestations 11 years post diagnosis
👉 Unexpected significant higher premature mortality: 13.8% of WD patients died over the study period. Average age at death of 58

Vivet is doing further analysis of these data and exploring other healthcare databases in the USA and Japan.
Vivet will be attending and presenting during 6th annual Gene Therapy for Rare Disorders Europe in London.
“Gain European Regulatory Clarity to Progress Efficient & Robust Gene Therapies for Rare Disease” is this year’s theme.

Vivet Therapeutic’s Chief Development Officer, Anne Douar, will participate to 2 sessions:
👉 Anne will talk about “Developing a Strategy for Re-Administration of AAV Gene Therapies Exploring dosing strategies” during the Clinical Trials & Tribulations section (Wednesday Oct. 26 – 12pm)
👉 She will also participate in the following panel: “The Challenges Associated With Scaling-Up to Commercial Manufacturing” during the CMC Challenges section (Wednesday Oct. 26 - 11am)
Vivet will be attending and presenting during 3rd annual Gene Therapy Immunogenicity Conference in Boston.

Overcoming Translational Hurdles, Improving Immunology Assays & Optimizing Clinical Strategies for Mitigating Adverse Immune Responses are gene therapy’s biggest challenges today.

Vivet Therapeutic’s Co-Founder & Chief Executive Officer, Jean-Philippe Combal, will address some of these challenges as part of the panel "EXAMINING DOSE SIZE & DOSING STRATEGIES IN THE CONTEXT OF IMMUNE RESPONSE" (Wed. Oct. 19 9.30-10.30 am) and answer key questions related to the administration of high doses of AAV in the context of benefit/risk:

👉 Exploring dosing strategies: “One and Done” or “Lower & Slower”?
👉 What impact would a cumulative vector genome delivery approach have on safety and efficacy?
👉 Moving away from one and done: what does best & future practice look like for dosing strategies?
Vivet will be attending and presenting some new and exciting results from its different programs and platforms this week at the upcoming ESGCT congress in Edinburgh.

2️⃣ presentations planned:

🩸Key data about seroprevalence of anti-adeno-associated virus 3B neutralizing antibodies in an international cohort of 200 healthy donors. These results will be presented by Blanche Tamarit (P515; Oct. 12, 7.30-9pm)

🐭 Nick Weber will present new preclinical data about our product VTX-803 for PFIC3 demonstrating the importance of testing in multiple systems prior to selecting final AAV candidates (P114; Oct. 13, 5.30-7.15pm).

🧬 Vivet’s CSO, Gloria Gonzalez-Aseguinolaza, will co-chair the “in vivo gene therapy & immune responses” session (Oct. 11 – 5-7.15pm; Lomond Suite)
For more details:
Vivet will be attending and presenting during the 1st Annual Optimizing AAV Safety Summit in Boston.

With hundreds of clinical trials using AAV currently underway, it has never before been so important to create and implement an effective AAV safety strategy. Improving prediction of AAV toxicity, accurately monitoring potential safety signals, extracting safety data from non-clinical & clinical studies to streamline AAV safety processes and safeguard patient outcomes, are all reasons that bring key leaders together.

There will be 2 sessions with Jean-Philippe Combal, CEO and Co-Founder of Vivet Therapeutics:
1️⃣ Jean-Phillipe will chair a workshop "Demonstrating Appropriate Toxicity & Safety Data to Gain Regulatory Approval of AAV Therapies" and will answer key challenges by putting forward:
👉 Real life case studies from key companies demonstrating how they progressed their own AAV programs, including best practice for IND submissions and phase I – III clinical development programs.

2️⃣ Then he will present during "The lessons learned from Clinical Trials to Enlighten Development for AAV Gene Therapies" session with Katherine High, President – Askbio and Eric David, CEO - BridgeBio
👉 Lessons learned from both successes and setbacks (such as clinical holds) to improve gene therapy development moving forward

And on the 2nd day of the conference, Gloria Gonzalez-Aseguinolaza, Chief Scientific Officer & Co-Founder of Vivet Therapeutics will also participate in 2 sessions:
1️⃣ Gloria will present "Strategies to Overcome the Action of Neutralizing Antibodies Against AAV" focused on several strategies under investigation to overcome this first barrier and unwanted side effects, and

2️⃣ Gloria will take part on the panel “Designing an Appropriate Immunosuppressive Regimen to Minimize Impact of the Immune Response to Administered AAV Therapies". Key questions will be answered such as:
👉 The best methods to circumvent the activation of both innate and adaptive immune response to the protein capsid, vector genome and transgene product to ensure no SAEs and reduce risk of clinical holds.
Vivet will be attending the 2nd Gene Patient Engagement congress with Sonia VALERO, Head of Clinical Operations and Patient Advocacy.

On the agenda, very interesting sessions and roundtables are planned, notably:

• Unique Characteristics of Gene Therapy & Their Ethical Implications For Clinical Trial Participation & Post-Approval Access
• Setting & Managing Expectations of Gene Therapy Within The Patient Community
• Building a Patient-Driven Informed Consent Process for a Gene Therapy
• Building a Patient-Centered Clinical Trial Protocol
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