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Webinar: Uncharted paths: successfully validating your LV gene therapy product process to gain FDA and EMA Approval

53 min watch


Process validation in cell and gene therapy development is complex and full of uncertainty. In this relatively untested space, identifying and employing the best practices to advance a therapy from concept to market can feel impossible – but it doesn’t have to be!

Lenmedly™, a hematopoietic stem cell (HSC) gene therapy from Orchard Therapeutics, is the first gene therapy to achieve FDA and EMA approval for treatment of metachromatic leukodystrophy (MLD). 

AGC Biologics’ experienced Cell and Gene CDMO team in Milan provided end-to-end support for the drug product through development, clinical stages, CMC strategy, and commercialization in the context of regulatory approval. 

We’ll walk through the process our leading team uses to produce lentiviral vector-based products that can scale at any stage and meet rigorous regulatory demands.



  • Investing to empower your Stage 1 activities  
  • Strategic planning of Stage 2 and 3 timelines
  • Leveraging the knowledge and data available for the platform process approach  
  • Continuous support from analytics: platform methods and in-house testing are preferred   
  • Creating a strong scientific and statistical justification of the specifications set  
  • Understanding final considerations for regulatory review and approval stages 

Francesca Rossetti

Analytical Method Development Director, AGC Biologics

Francesca started at AGC Biologics in 2004 and later became the Head of Analytical Development in 2017. Her work focuses around the design of analytical methods for characterization and release of retroviral, lentiviral, adeno-associated vectors and of cells in compliance to GMP rules and ICH guidelines. In 2021 she became a qualified person for the AGC Biologics’ Milan site.

She has expertise from product development and pre-clinical projects all the way to market, actively contributing to drawing up and revising regulatory documentation, such as: IMPD, IND, CTD, BLA submissions, defining product validation and CMC characterization strategies. She holds 20 years of experience working in an international project management team working with development and qualified analytical methods ready for ATMP’s release, assuring product quality.

Francesca holds a degree in Biological Sciences from the University of Pavia and a second level master in “Expert in analytical chemistry for pharmaceutical industry”.

Francesca Rossetti headshot

Monika Pema

Senior Manager MSAT, AGC Biologics

Monika started at AGC Biologics in 2015 as a technical Project Manager after 10 years in academic scientific research. During these years she guided and supported the collaboration between AGC Biologics and multiple international clients running different CGT projects, covering early development through clinical phases.

In 2021 she joined the MSAT-process validation team where her goal was to build a new unit and drive the preparation of four different PPQ campaigns for the validation of lentivirus manufacturing processes, for both EMA and FDA applications. She actively contributed to the definition of the validation strategies by constantly working with clients. Her expertise was further expanded following the post-approval management of commercial products, including lentivirus and retrovirus manufacturing processes and HSC Drug Product manufacturing. She contributed to the revision of regulatory documents on behalf of clients and during the assessment phase by the regulatory agencies she supported the clients by providing technical inputs information requests. She was also involved in quality and regulatory inspections providing key support as an SME for process validation topics.

Monika holds a degree in Biological Sciences from the University of Milano-Bicocca and a PhD in Molecular Medicine from the San Raffaele University of Milan. She has an executive master in “Leadership, Empowerment & Change Management” from 24ore Business School.

Monika Pema

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