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

  • Translating knowledge and experience in virology into novel therapeutic concepts involving infectious agents such as viral agents employed in oncolytic therapies and AAV or related constructs employed in various cell and gene therapy approaches

  • 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

Vaccines

  • Providing services to academic institutions, small and medium sized enterprises and globally operating vaccine manufacturers

  • Providing globally acknowledged expertise in vaccine development and licensure

  • Providing in-depth scientific contributions to all issues related to vaccine development and licensure, tailor-made for every regulatory region in the world

  • Providing fundamental expertise in process development, non-clinical and clinical development of vaccines

  • Contributing to scientific advisory boards

  • Integrating the public health perspectives into the benefit-risk-calculation relevant to vaccines

  • Taking vaccines against emerging and re-merging infectious diseases through EMA’s PRIME procedure and FDA’s Fast Track, Breakthrough Therapy, Accelerated Approval or Priority Review programs

  • Paving the way towards next generation influenza vaccines

  • Integrating EMA Article 58 and WHO prequalification procedure

  • Interacting with competent authorities in low- and medium-income countries

  • Exploring and realizing novel regulatory pathways for vaccine licensure

  • Developing regulatory concepts for platform technologies facilitating faster vaccine production and licensure

  • Integrating activities of CMOs, CROs and other service providers

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

  • Providing regulatory and scientific support to successfully conduct national scientific advice, EMA scientific advice, FDA meetings and ROW authorities

  • Writing any kind of documents required for agency interactions and regulatory procedures

  • 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

  • Rescuing high-risk projects at the level of national authorities, EMA, FDA and beyond

  • 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 program 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