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Stand-Out Points from an Insightful Week of Discussions at Advanced Therapies Week

Anna Osborne
2 February 2022
For this round-up, Phacilitate’s Anna Osborne summarises key highlights from the range of insightful talks that took place at Advanced Therapies Week.

1- 2021 in Review

We are witnessing a “cell and gene therapy revolution” – Emma Cheesman, Director of Development Consulting and Scientific Affairs, Biopharma Excellence.

The first full day started with a consideration of the cell and gene therapies landscape – where are we now and where are we heading? Noting considerable successes of the last year, Janet Lynch Lambert, Chief Executive Officer for the Alliance for Regenerative Medicine, referred to reasons for celebration within the industry. It was a record breaking year for investment, headed up by venture capital. $23.1 billion total investment was raised in 2021, a 16% increase on the previous year with gene therapy gaining most financial interest. 2021 also saw many new regenerative medicine models gain approval; it was the best year on record for cell based immuno-oncology products, which make up over 50% of the current 2261 ongoing CGT clinical trials. The FDA’s RMAT designation also had an impactful presence in 2021, granting designation for seven therapies. Fate Theraputics’ FT516 – targeting relapsed/refractory DLBCL – was one of these therapeutics announced at the end of 2021 to be supported and accelerated by RMAT designation.

2- A Long Way to Go

“[The industry] share a common mission to get the clinical properties of these therapies realised” Jason C. Foster, Chief Executive Officer, Ori Biotech.

From present day successes to future areas for improvement, Jason Foster of Ori Biotech presented an interesting perspective acknowledging the limited applications of cell and gene therapies. He highlighted the issue of patient access to CGT treatments. For example only 1 in 9 children can access this CAR-T infusion therapy as a treatment option for the devastating disease ALL. “We can do better,” says Jason.

In order to improve patient access, it is essential to find ways to make AT more affordable. Ori Biotech works to provide pioneering biologic industrialisation, providing flexibility and scalability in one CGT platform to reduce costs and enable mass production of personalised therapies in a safe and considerate way. Integrating a digital platform of data analysis for more efficient manufacturing accelerates a therapies journey to commercialisation and reduces drug cost. The more affordable the industry aims to make these novel therapies, the wider areas they will reach, ultimately becoming more accessible and common as first round treatments. Jason discussed this envision for the future, alongside Ori Biotech Chief Data Officer Kevin Gordon, in this recent interview with Phacilitate Editor.


3- Achieving Commercial Success

“Commercial success is decided long before launch” – Holly May, Chief Commercial Officer of AVROBIO.

A third of all drugs launched to market fail. To warrant a successful commercial product, applications for commercialisation need to be considered from the initial stages of development. Addressing the un-met needs of patients is at the heart of CGT, considering from the early pre-clinical stages how the therapeutic will successfully reach the patient, and what the patient requires from the therapy, is crucial. It is key to keep the main stakeholders in mind during the product development journey, these include: patients, caregivers, healthcare providers, advocacy groups, investors and regulators, and act upon their insights and feedback when making strategic choices in clinical stages. Beginning the process with a commercialisation mind-set is paramount for therapeutic success.

4- Tackling the Skills Shortage

“Our people create the competitive advantage to bring these therapeutics to market” – Jeff Boyd, Partner and Life Sciences Tools & Services Sector Co-Leader, Heidrick & Struggles.

It was important to turn our attention to a major challenge faced by the CGT industry in light of its recent and exponential growth; the skills shortage. Dr Angela Justice, Chief People Officer at TCR2 Therapeutics began this particular session outlining that the demand for a skilled CGT workforce is set to more than double from 7,000–15,000 by 2026, with the main area of concern in manufacturing roles. There are many ideas and programs underway in order to increase the talent pool including: ATAC apprenticeships, ATSTN training centres, headhunting industry outsiders with transferable skills, and working with schools and universities. Ultimately a varied blending of talent is required to offer a sustainable solution.

Lynn Fischer, Chief Executive Officer of Title 21 Health Solutions, explained that the world of employment is changing. Due to Covid-19 we have seen a “digital transformation,” from which CGT has benefited. Technological advancement and the digitalisation of clinical and lab systems has increased efficiency – the industry has seen a 44% increase in employee productivity and an 83% decrease in data input errors. Not only does this improve productivity, data traceability, compliance and quality, it also can increase job flexibility and satisfaction.


5- Therapeutics 4.0

“The next level of digital foundation is supporting the next level of advancement” – Sean Werner, President of Sexton Biotechnologies.

As the demand for CGT therapeutics on a commercial scale becomes a reality the need to produce high volumes in a short time scale presents challenges to current small scale working processes. New automated solutions for the design and delivery of these therapeutics is seeing the industry entering into a new era of development. Sean informs that implementing fully automated systems for the modelling, optimisation and implementation of commercial strategy, will see the real-time monitoring and comparability of these products as they scale into GMP production, for a more efficient and controlled scalable process.

      • Ryan Cawood the Chief Scientific Officer at WuXi Advanced Therapeutics and OXGENE shared the company’s new TESSA technology – a novel AAV vector designed to deliver high quality and mass produced AAV- as a solution to the need to produce AAV on a larger scale for systemic diseases. Watch Ryan’s recent TECH DEMO on our website to find out more.


6- CAR-T in Solid Tumours

“We all agree we have a lot of work ahead of us… success in liquid tumours has raised expectations” – Nina Bauer, Director, Head of Commercial, Gene Editing and Novel Modalities, MilliporeSigma.

The successes witnessed in CAR-T treatment for liquid tumours is yet to be replicated in CGT targeting of solid tumours. Unlike tumours in circulation recognised by CD19, solid tumours are bordered off with the tumour micro-environment creating a physical barrier that is not easily infiltrated by CAR-T cells. It is also difficult to find targets which are expressed solely by solid tumours and not healthy tissues. Novel systems of alternative engineered T cells are under clinical investigation to pervade solid tumours.

      • Mark Throsby, Chief Scientific Officer of clinical stage biotech, Gadeta, explains the γδ TCR tumour-specific autologous cell therapy they have developed to target solid tumours. The receptor-engineered T lymphocytes recognise tumour epitopes – immune response HLA surface proteins. TCR can therefore bind to a large range of tumour cell lines providing future applications for allogeneic therapy. Gadeta’s product line, GDT002, is currently due to enter clinical stages form multiple myeloma.
      • Jak Knowles, Chief Executive Officer at Affini-T Therapeutics details how they have developed oncogenic driver mutation targeting TCR’s. KRAS is an oncogene expressed exclusively in tumour cells. Affini-T Therapeutics lead program sees TCR’s target KRAS G12V and G12D mutated tumour cells through recognising the surface HLAs. Modifying both CD8+ and CD4+ T cells with Affini-T’s TCR and a CD8 co-receptor allows the TCRs to bind to HLA’s, causing a combined cytotoxic and inflammatory response against tumour cells.

7- Enabling in vivo Technologies

“Integrated in-vivo cell therapy approaches seek to provide solutions to the challenges in both blood and solid tumour therapeutics”- Tamer Mohamed, President & CEO, Aspect Biosystems

Despite the many success of conventional CAR-T therapies there are in vivo limitations and challenges of this therapy including lack of activity targeting and dosage control.

      • Daniel Shelly, Vice President at Prescient Therapeutics shares details of their new technology – OmniCAR, of which they have 3 different products at the pre-clinical stages. OmniCAR is used to develop new CAR-T therapies for an expanded range of indications, where in vivo behaviour can be controlled through modifying T cells to target tumour-specific antigen profiles and inducing tumour killer cells. This reduces cell exhaustion and antigen escape, whilst increasing CAR-T safety and dosage control through specific receptor targeting. OmniCAR works using both autologous and allogeneic cells.
      • Alan Kypson, Cardiac Surgeon at REX Hospital/UNC discuss the in vivo applications of Humacyte’s bioengineering platform HAV, in light of their positive pre-clinical results and extensive HAV portfolio. 80–90% of CABG surgery uses SVGs – however this concurs a 30% failure rate one year post-op. Humacyte’s bioengineered extracellular tube HAV acts like a blood vessel and eliminates the need for vessel harvesting from the patient. It provides immediate off-the-shelf access and is viably produced at a commercial level. Implantation of a 6mm HAV implanted into humans displayed long-term durability with no evidence of immunogenicity. Following these trials in vivo, imaging identified that the vessel lumen had been endothelialised, as the patient’s own cells repopulated around the mechanical lumen.

Moving forward, a focus on reducing immunogenicity of allogenic cells will enhance in vivo control of these therapeutics.

8- Challenges in Gaining Investment

“We’re backing you to help make something special happen to patients…. with strategic financial collaboration” – Craig Shepherd, Senior Managing Director, Blackstone Life Sciences

Gaining sufficient investment is paramount for company and product investigation and scale-up. There appears to be a disconnect between biotechs and investors. The marketing of a biotech to investors to gain their financial support can be challenging. Conveying scientific information and therapeutic impact in an economic environment requires careful marketing, but biotechs, particularly small start-ups, don’t always have access to this type of expertise.

Another key issue comes with presenting the risk-reward system of a therapeutic. Being a relatively novel area of pharmaceuticals, advanced therapies comes with risk as a precedent, navigating the communication of these risks in terms of potential patient implications is important to reassure investors. A start-up biotech can be deemed as a higher level of finical risk for investors, as they are yet to produce supporting analytical data for product success and financial return. Bridging the gap to allow novel companies and therapeutics to gain access to investment can be attained through expert marketing and education. As investor interest in CGT increases finding supporting financial models to fit into the unusual financial nature of AT will play a central role as the industry continues to move forward.

You can find out more about the current climate in biotech investment in Phacilitates monthly finance series. 

Impressions from the Editors

As a positive week of networking and knowledge sharing drew to a close, it was clear to see the impact the in-person event had on the attendees and industry as a whole. Providing a space to celebrate industry successes and communicate current challenges creates solutions for a successful future.

Ultimately, as more therapies reach commercialisation facilitated by enabling technologies, skilled workforces and confident investments, more patients are reached and more lives changed.

“We appreciate Phacilitate for having the fortitude for putting on a conference” – Lynn Fischer, Chief Executive Officer, Title 21 Health Solutions.

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