Late February, France Biotech annual report “Panorama Health Tech 2023” was published. Here is a brief biotech focused summary we would like to share with you:

In 2023, the French HealthTech sector established itself as a key innovation ecosystem, representing more than 2,600 companies and 60,000 jobs, a 33% increase in turnover and a 19% increase in employment. It is rapidly expanding in the digital health field and is the European leader in rare disease treatment. In a tense economic context, the sector attracted 1.8 billion euros in funding but faces financing challenges, especially for emerging startups. Future efforts will focus on financing issues, societal, and environmental challenges.
Regarding biotechnology companies, in 2023 there were 820 biotech firms, each developing an average of 3 products (25% of the products developed in biotech were related to oncology, followed by infectious diseases and central nervous system). 59% of the products were in early development phases, 31% in clinical phases, and 10% were marketed. The average amount invested per company was 5.1 million euros in 2022 (a 16% increase compared to 2021). Finally, in 2023, 50% of biotech companies filed patent applications.

Biotherapy Partners is a consulting firm providing support for the drug development of Biologicals like mAbs or gene and cell therapies.

Biotherapy Partners experts attended the FDA webinar on considerations for the development of chimeric antigen receptor (CAR)-T cell products. Here are our key take-away messages.

Product design
Product design should be evaluated in early development based on key elements: CAR construct, vector type, cellular starting material, drug product formulation;

Chemistry, Manufacturing and Control (CMC)
– For IND-enabling studies during early development stage of autologous CAR-Ts, the use of starting material from healthy donors was considered as likely appropriate for manufacturing process demonstration batches; but applicability to cells from patients should be discussed; discussing carefully potential risks of non-representativeness.
– The importance of the uniformity of collection and processing of cells for autologous CAR-Ts was stressed; if intermediate(s) are stored, stability must be demonstrated. Maintaining a robust chain of custody and identity chain is also important for intermediate storage;
– Vector titer through transgene expression is likely to be sufficient for phase I (transgene functional potency test at release needed for later stage);
– Manage carefully manufacturing and analytical changes by assessing comparability during the CAR-T cell product lifecycle (process evolution, multi-site manufacturing, method changes & evolutions). Refer to “Manufacturing changes and comparability for human cellular and gene therapy products: draft guidance for industry”

Non-clinical
– Due to diversity of programs and modalities (autologous/allogenic, vector types used to deliver genetic material encoding the CAR to T cells (e.g. lentiviral, transposons, mRNA …) a case-by-case assessment should be performed using a combination of in vitro, in silico & in vivo studies;
– Specificity to antigen and off–target effect to be carefully documented
– Assessment of the potential for CAR-T cells to undergo stimulation-independent growth changed from “cytokine-independent growth” to “uncontrolled proliferation” to include both cytokine- and antigen-independent growth;

Clinical
– The selection of the study population should consider the anticipated risks and the potential benefits for the study subjects, to ensure that the overall benefits outweigh the risks.
– Treatment plan should be detailed extensively, as expected: starting dose, dose (de)escalation rules, management of manufacturing delays or failures, bridging therapy, definition of dose limiting toxicity (DLT), stopping rules;
– A staggered enrollment is favored for new CAR-Ts and repeat dosing is not preferred as CAR-T cell expansion on redosing is not equivalent;

For more information, please refer to “Considerations for the Development of Chimeric Antigen Receptor T Cell Products” and watch the records of the webinar recently shared by FDA
hashtag#FDA hashtag#guidance hashtag#CAR-T hashtag#drugdevelopment hashtag#nonclinical hashtag#CMC

Biotherapy Partners is a consulting firm providing support for the drug development of Biologicals like mAbs or gene and cell therapies

Olivier Negre, co-founder of Biotherapy Partners, will attend SFTCG’s workshop (link).

To schedule a meeting, contact us at info@biotherapypartners.com.

Biotherapy Partners is a consulting firm providing support for the drug development of Biologicals like mAbs or gene and cell therapies

Key Pathway Resources | EuroGCT

Biotherapy Partners is serving international biotechs, VCs & TTOs to address their scientific, technical & operating challenges in drug development, i.e. Non-Clinical/Translation, CMC/AD, Clinical, Bioanalysis, Lab operations, Regulatory and QA & Project Management. Biotherapy Partners is tackling specifically the challenges of Biologicals, including mAb/ADC, gene and cell therapies.

Our purpose is to translate research ideas into therapies, for the benefit of patients.

During a full day (program), meet the experts from academia, clinical and industry in Cell and Gene Therapies.

With scientific talks given by high level speakers, an exhibition hall allowing companies to showcase their last innovations and B2B meetings to connect and create partnership.

Meet our Experts at the conference: Alexis COLLETTE, President & cofounder, and Olivier NEGRE, Partner & cofounder.

To schedule a meeting, contact us at info@biotherapypartners.com.

Biotherapy Partners is a consulting firm providing support for the development of Advanced Therapy Medicinal Products, especially gene and cell therapies

Alexis COLLETTE, co-founder and President of Biotherapy Partners, will attend France BioProduction Congress 2024, where French drug manufacturing is discussed.

To schedule a meeting, contact us at info@biotherapypartners.com.

Biotherapy Partners is a consulting firm providing support for the development of Advanced Therapy Medicinal Products, especially gene and cell therapies

Alexis COLLETTE, CEO & co-founder of Biotherapy Partners is attending Biospring Europe 2024 in Barcelona, Spain, from March 18th to 20th, 2024 to meet clients, partners and providers for biotherapy product development.

To schedule a meeting, contact us at info@biotherapypartners.com.

Biotherapy Partners is a consulting firm providing support for the development of Advanced Therapy Medicinal Products, especially gene and cell therapies

Wrap up of 2023 for Biotherapy Partners

Olivier Negre, co-founder of Biotherapy Partners and Chief Scientific Officer of Smart Immune will give a presentation on CAR ProTcell platform developed by Smart Immune: CAR ProTcell, towards well-tolerated and persistent off-the-shelf allogeneic CAR-T cells.

To schedule a meeting, contact us at info@biotherapypartners.com.

Biotherapy Partners is a consulting firm providing support for the development of Advanced Therapy Medicinal Products, especially gene and cell therapies