As part of WuXi AppTec’s ongoing efforts to collaboratively foster new thinking and actionable approaches in advancing breakthroughs for patients, we have launched a new interview series in 2022 – “Delivering on the Promise of New Modalities” – so leading voices of R&D can share how their approaches are addressing the barriers standing in the way of breakthroughs.

The next installment of our interview series features Elvire Gouze, CEO & Founder of Innoskel SAS, France, a bioscience platform company developing transformative gene therapies for the unmet needs of individuals with rare bone disorders. When Innoskel launched at the end of 2020, they achieved Series A Financing to the tune of €20 million to advance the company’s lead program clinical stage operations. Innoskel is developing treatment options for a group of rare skeletal disorders collectively known as type 2 collagenopathies that affect the structure of the body’s connective tissues (collagen). Earlier this year, the company announced the enrollment of the first patient in a study, which will evaluate the course of type II collagen disorders in children with short stature.

Thank you for taking the time to join us today, Elvire! We were excited to see early progress announced for your clinical trials earlier this year. For rare bone disorders, what are challenges in current therapeutic interventions, or current new modality solutions?

Elvire: Innoskel is developing innovative treatments for rare bone disorders. These diseases affect the mechanisms of bone growth, mainly endochondral bone growth, and the challenge is to effectively target the growth plates of the bones of affected children while limiting off target distribution.

There is no current modality for the diseases requiring gene therapy that have been shown to be effective at restoring bone growth. Indeed, common modalities, such as AAV or lipid nano particles, are known to be ineffective in targeting growth plates. Our approach is unique and a combination of the gene therapy modality, route of administration and proprietary promoter sequences allows us to have safety advantages combined with a directed biodistribution to the target tissues.

How is your gene therapy approach helping to address this challenge? Is your technology differentiated from existing approaches?

Elvire: Innoskel develops a lentiviral vector because of the ability of lentivirus to transduce non-dividing cells brings a significant advantage allowing genetic modification of the targeted proliferating cells and transmission to the daughter cells after cellular division. We have to pave the way with regulatory agencies and payers to address these diseases with a lentiviral vector administered systemically.

The durability of the effect on bone growth is expected to be permanent – nevertheless, it is anticipated that due to bone growth and chondrocyte turnover, the duration of the LVV activity may be limited to 2-3 years requiring repeat administration. Innoskel is considering an immunomodulation regimen that should allow tempering down the immune reaction to the lentiviral vector and would allow for later redosing.

In your opinion, what’s the biggest barrier to realizing the full potential of your LVV based gene therapies?

Elvire: Manufacturing is a critical aspect of therapeutic intervention, and one must anticipate the technical challenges to accelerate treatment access for the patients. Indeed, current lentiviral vector production standards need to be optimized to reach a yield and product quality compatible with in vivo systemic administration.

To solve these challenges and bring the therapies to global patients, how important is global collaboration to your company?

Elvire: Global collaboration is critical for Innoskel. Indeed, when developing an innovative treatment for rare pediatric disorders, collaboration between companies, big pharmas and patient organizations is the only way to develop treatments and achieve patient access successfully and rapidly. Global collaboration is especially important in the rare disease area, where the best experts of these diseases are scarce and where the deep understanding of these diseases and their impacts on the life of patients is limited. It is essential to bring experts and patient representatives together to accelerate the development of therapies that are truly life-transforming and meaningful to patients. Worldwide databases and international collaboration are also critical to identify the few patients that could benefit from the treatment as quickly as possible, as we know that the therapeutic window before irreversible damages are done is tight in most cases. Looking for the best experts, wherever they are, allows us to be agile, creative, relevant and efficient. For Innoskel, the global collaboration approach is the best and only way to truly commit to patients to do the best we can to answer their high unmet needs.

Thanks Elvire, really enjoy the discussion. If we were to gather here again in 10 or 15 years’ time, what do you think we’re going to be talking about in terms of what we have already achieved in the industry? What do you think are going to be some of the solutions going forward?

Elvire: The real challenge for the next 10-15 years is to keep alive the interest of the pharmaceutical sector for rare diseases. Altogether rare diseases affect more than 400 million people worldwide. However, 95% of rare diseases have no approved treatment. The disinterest from the rare disease sector would be catastrophic and as a company, we need to be innovative to build a significant market case by regrouping diseases when possible. There are some solutions going forward such as the implementation of basket trials, allowing for the investigation of multiple rare diseases simultaneously with a single treatment intervention. Adaptive trial design is another solution that can be envisioned to accelerate the development of drug candidates for rare disease with high unmet needs.

 

---

Elvire Gouze, PhD

CEO & Founder, Innoskel SAS, France

Elvire Gouze is CEO and Founder of Innoskel. She is a distinguished researcher whose career represents a rare combination of scientific excellence, drug developmentand support for the patient community. Dr. Gouze has a proven track record of progressing the development of an innovative therapeutic pipeline, as seen with her first venture TherAchon, a biotech company focusing on achondroplasia, a rare bone disease, which was acquired at Phase 1 stage for $810M by Pfizer in May 2019. She holds a PhD in molecular pharmacology. She performed her post-doctoral training at Harvard Medical School, where she later became an Instructor in Orthopedic surgery in the Center for Molecular Orthopedics. Elvire Gouze was later appointed Assistant Professor at the University of Florida and in 2009 became Team Leader at Inserm Nice, France. Dr Gouze won twice the prestigious EY Entrepreneur of the year in the Startup category, PACA area, France.