Development

  • Development and approval of a first in class monoclonal antibody

  • Recombinant protein: review of programme to characterise the drug substance and final product

  • Establishment of the key preclinical investigations for a recombinant protein

  • Determination of key elements and review of comparability programmes: recombinant protein used in development program vs. commercial product


Regulatory strategy

  • Analysis of the regulatory pathway and regulatory strategy for biotech products with the EU as the target market

  • Target product profile and SmPC / product information development for monoclonal antibodies

  • Switch of a nationally registered medicinal product to a medical device allowing enhanced commercialization

  • Orphan drug and PIP strategies for monoclonal antibodies

  • Design and conduct of a regulatory roadshow in preparation of a centralized procedure

ATMPs

  • Genome edited cell-based product using CRISPR/Cas: Set-up of integrated development and regulatory strategy including implementation (1.5 years from lead candidate selection to submission for clinical trials!)

  • T-cell receptor (TCR) modified T cells: Set-up of integrated regulatory strategy, preparation of regulatory interactions; input to selection of viral vector for modification (clinical trial was initiated without in vivo animal studies)

  • Allogenic, genome edited CAR-Ts: input to development program, execution of international regulatory strategy

  • Due diligence for a product portfolio intended for in vivo genome editing using AAV based technologies

  • Due diligence for ex vivo genome edited product portfolio 

  • 3-D printed cell-based product: Set up of EU based manufacturing and clinical development strategy for US based client 

  • CMO selection for genetically modified tissue engineered product and oncolytic virus portfolio

  • Advisory meetings for oncolytic virus portfolio 

  • Gap analysis for mesenchymal stem cell product

  • Tissue engineered product using pluripotent stem cells: set up of development program, regulatory strategy, agency meetings

  • Integration of acquired cell/gene therapy company into an established mid-size pharma company incl. product portfolio strategy, integrated development and regulatory strategy

  • Submissions for clinical trials, both EU and US 

  • Multiple regulatory interactions in EU and US related to cell, gene and tissue engineered products as well as oncolytic viruses

  • Set-up of CMC strategy for several ATMP projects

  • Set-up of comparability / bridging strategy for genetically modified cells

  • Development of risk mitigation strategies for various ATMPs about to progress to first in human clinical trials 

  • Development of dosing rationale for various ATMPs

  • Multiple ERA (environmental risk assessment) and GMO (genetically modified organism) submissions for a broad range of gene therapy products incl. AAV based products

  • Invited expert to EMA genome editing workshop 

  • Invited by Stem Cell Network to promote translation of stem cell products (expert panel at German Ministry of Commerce) 

  • Presenter at various cell- and gene therapy conferences, multiple ATMP-related publications

Centralised procedure

  • Preparation and successful conduct of a centralized procedure for a monoclonal antibody

  • Review of the clinical Module 2 CTD section for monoclonal antibody submissions of TOP 20 pharma

  • Review of the Module 2 for recombinant protein submissions of TOP 20 pharma

  • Preparation, conduct and debrief of pre-submission meetings with EMA

  • Liaison with the EMA for preparation of centralised submissions for monoclonal antibodies

  • Interpretation of the CHMP list of questions and preparation of response documents for various recombinant proteins

  • Support in appeal processes at CHMP level

  • Preparation, rehearsal and debrief of oral hearings incl. SAG meetings for recombinant proteins and monoclonal antibodies

  • Adaptation of a US CTD for EU-submission of a Marketing Authorisation Application for two recombinant proteins


Agency interactions

  • Briefing packages for Scientific Advice at national EU authorities

  • Strategic outline and review of the briefing package for a pre-IND meeting at the FDA including presentation of specific topics at the meeting on behalf of the client

  • Drafting questions and company's positions for Scientific Advice meeting with the German authority, PEI, including review of briefing book

  • Liaison with the EMA for preparation of numerous scientific advice submissions incl. pre-submission activities

  • Communication with the German agency PEI for national scientific advice


Rescue projects

  • Turn negative CHMP opinions into positive opinions for a monoclonal antibodies

  • Turn a negative CHMP opinion into a conditional approval for a monoclonal antibody

  • CMC trouble-shooting regarding changes introduced in the manufacturing of a marketed biotech product

  • Use of invalid equipment caused by supplier error in purification of recombinant proteins – evaluation, presentation, decision-making on use of the material, interaction with agencies


Due diligence for

  • Tissue-engineered products

  • Portfolio of monoclonal antibodies

  • Merger / acquisition of a biopharmaceutical company


Clinical trial applications

  • Gap analysis of the preclinical programme to support the first-in-human clinical trial of a monoclonal antibody

  • Review of the protocol for a first-in-human trial with regard to regulatory requirements


Paediatric investigational plan (PIP)

  • Paediatric investigational plan for monoclonal antibodies: review of application and analysis of PDCO feedback


Board and advisory board activities

  • Member of the scientific and regulatory evaluation boards of three TOP-10 Pharma and several mid-size biotech companies

  • Preparation and conduct of scientific board meetings of TOP20 pharma and start-up biotech companies