Insights into issues and developments from Advanced Therapies Week

Dr. Jörg Schneider | Director – Principal Consultant at BioPharma Excellence

Dr. Jörg Schneider, Director – Principal Consultant at BioPharma Excellence, shares key takeaways from Advanced Therapies Week 2023 and the latest developments in cell and gene therapy.

Much is happening in the world of cell and gene therapy, with new innovations, new opportunities as well as challenges and more diverse stakeholders involved in the field. The most recent Advanced Therapies Week 2023 in Miami brought all of this together in a deeply informative – and at times deeply moving – format.

During the plenary session, Susan Nichols, Chief Business Officer at ViroCell Biologics, summarized the Top Ten Events of 2022 from a developer’s perspective. It should be noted that by “events” Nichols meant not only innovation breakthroughs but also setbacks in the area of cell and gene therapies.

Third from the top of her list was the first regulatory approval of an allogeneic cell therapy, Ebvallo, for a rare blood cancer, from Atara Biotherapeutics in December 22, and second from the top was approval of CAR T as second line large B cell lymphoma treatment (Yescarta). The most notable event was, unfortunately, the withdrawal of 7 of 24 approved products in the EU due to reimbursement challenges, underscoring the issue of market access and the importance of addressing this more proactively.

Funding and talent barriers

In his presentation, David Barrett, CEO of American Society for Gene and Cell Therapies discussed the vast research landscape in the field, noting that more than 3,700 therapies are in development, ranging from preclinical through pre-registration. However, while research is increasing, funding is lagging with startup financing for gene, cell, and RNA therapy companies down significantly in volume and value. This is a trend that is predicted to continue during 2023.

The next panel presenter, Cynthia Pussinen, Former Chief Technology Officer at Spark Therapeutics, shared her thoughts on cell and gene therapy research, noting it is poised for more significant advances, with more than 50 CGT products expected to be approved by the US Food and Drug Administration (FDA) by 2024.

However, she raised several pressing challenges, such as regulatory expectations and lack of clarity, potency assays, scalability and availability of production space, as well as cost. In particular, she highlighted the challenge the industry is having in attracting talent, particularly people with the right leadership profile and technical expertise.

One of the impediments to attracting talent is competing demands for the same expertise. In Washington, DC, for example, the large number of biotech companies compete with the FDA for experts. This issue will require creative solutions, for example, remote working where possible, although this is not feasible for people working at sites, such as in manufacturing.

Barrett also offered thoughts on addressing the talent issue with the next generation by empowering young thinkers through visits to schools and colleges and outreach programs. As an example, Jeff Galvin, the CEO of American Gene Technologies, a pioneering gene therapy company, gives his time to speak with school and college students about careers in the industry and opportunities to play a role in finding cures for HIV, cancer and other diseases.

The final plenary speaker, Dominic Clarke, Chief Technical Officer at Cell and Gene Therapy, Discovery Life Sciences focused his talk on possible strategies to successfully industrialize cell and gene therapies and overcome CMC hurdles as a key requirement for patient access.

Patient associations as drug developers

Given the nature of cell and gene therapies as possible cures for rare and ultra-rare diseases, it’s not surprising that patient associations have entered the fray. This is particularly notable with ultra-rare diseases that are not commercially attractive to biopharma companies because there is a very small eligible population. Most of these diseases impact children, which has led many parents to invest their own time and energy into not only lobbying but learning how to carry out drug development.

FAST´s Chief Scientific Officer Allyson Berent-Weisse , shared her own experience and the role of FAST in finding a cure for Angelman syndrome. Her powerful, rich presentation underscored the challenges parents and carers of children with ultra-rare diseases face and the commitment to finding treatments and cures. It also highlighted the pressure of time running out for patients and how this will drive further questions from sponsors about regulatory paradigms. This was highlighted by Wilson Bryan, FDA, in his presentation: Gene therapy for rare diseases – Can we go faster and still get it right?

This commitment and battle to save children with rare diseases came across compellingly in the full-length documentary shared at the meeting, Of Medicines and Miracles from the Emily Whitehead Foundation. Emily was diagnosed with acute lymphoblastic leukemia (ALL) in May 2010 when she was five years old and became resistant to more traditional treatment. Determined to fight for their daughter, her parents enrolled her in a Phase I clinical trial led by Dr. Carl June for what was then an experimental CAR T-cell therapy in 2012 and 11 years later she is still cancer free.

While Emily’s story and the commitment of organizations such as FAST are hugely inspiring, unfortunately access to life-changing cell and gene therapies is far from equitable. Jennifer Chow, CEO of Chimeric Therapeutics, pointed to the racial inequality when it comes to access to CAR T treatment for cancer patients. Only 20% of eligible patients have access – 79% of them White, 4% Asian and 4% Black, despite the fact that incidence is as high for Black patients as it is for White patients.
Clearly, while there is much to celebrate with advances in cell and gene therapy, there is a long way to go both in terms of breakthroughs as well as patient access.

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