Pedro M.

Preclinical Development and Oligonucleotide lead in research collaboration with Soufflé Therapeutics. Due diligence (oligonucleotide) projects.

R&D and Oligonucleotide Lead for establishing research collaboration with Soufflé Therapeutics. Led scouting, evaluation, and scientific due diligence for oligonucleotide extra-hepatic modality.

Provided scientific bioanalytical, biodistribution and DMPK strategy for nucleic acid-based and targeting modalities and supported preclinical projects: oligonucleotide conjugates, mRNA/LNPs, and RNA-targeting small molecules. Represented the bioanalytical function in global project teams (Bayer AG) and international expert groups in the nucleic acids field (mRNA, ASO, siRNA). Contributed to three white papers and commented on new or revised guidelines, e.g. IQ, and FDA.

As an active member of the IQ Nucleic Acids Working Group (on behalf of Bayer Pharma as oligo/RNA expert), I participate in the following subgroups in the last three years: a) Therapeutic mRNA/LNPs (I have presented a webinar covering bioanalytical aspects of mRNA therapies) b) ASO/siRNAs c) Immunogenicity of mRNA/Oligonucleotides d) Drugsafe Oligo-based Therapeutics As members of the IQ Consortium Nucleic Acids Working Group, we regularly discuss best practices related to ADME/DMPK (biodistribution, DDI, formulations, and bioanalytical characterization) and clinical pharmacology strategies for nucleic acid-based modalities. As IQ members, we also work to harmonize approaches and strategies for risk assessment, bioanalysis, and PK/PD for nucleic acid-based modalities. We identify risks related to the developability of nucleic acid-based modalities and develop clear guidance on expectations for preclinical and clinical development. We develop risk-based strategies that can drive preclinical, translational, and bioanalytical strategies to support clinical development.

At ProQR, I’ve co-invented a novel RNA “editing” therapeutic modality (TRIDENT™ technology), demonstrated it up to in vitro Proof of Concept (PoC): human primary cells and animal disease mode cells. Additionally, I’ve co-written, applied for, and secured two grant awards: one from the Gilbert Family Foundation for Neurofibromatosis Type 1 and another from the Cystic Fibrosis Foundation, totaling $1.7 million in funding - together with an academic partner. I’ve established and overseen multiple scientific external collaborations (including concept development and Statement-of-Work writing), and supported business development activities (preparing slide decks and presenting to major pharmaceutical venture arms). These projects were focused on oligonucleotide technology development. I have also worked side by side with the patent attorney in identifying patentable inventions and help with the filling and prosecution process. Over a period of 5 years, I collaborated with Professor Yi-Tao Yu at the University of Rochester, my academic partner. Note: ProQR is a Dutch NASDAQ-listed RNA editing biotech company.

Led a group of scientists working in the Nonclinical development of a Cystic Fibrosis drug. RNA expert for different preclinical teams (PK, ADME, Toxicology, in vivo, biomarkers, bioanalysis). Co-invented new antisense oligonucleotides for treatment of Huntington's disease (patent WO/2019/043027).

Operated and maintained automated AKTA oligonucleotide synthesizer, including troubleshooting, synthesis cycle optimizations. Synthesized diverse novel chemically modified oligonucleotides (custom nucleoside analogues from Glen Research) for RNA editing assays. Managed all aspects of synthesis operations: argon gas systems, phosphoramidite solution preparation and replacement, and column selection. Ensured synthesis consistency with high coupling efficiencies and purity standards. Delivered multiple lectures on oligonucleotide chemistry and synthesis to TU-Darmstadt PhD/Master students. Trained lab members on hands-on oligo synthesis and equipment operation and maintenance. Development of RNA editing assays with chemically modified guide RNAs. SHAPE mRNA chemical probing technology of therapeutic transcripts to understand structural and thermodynamic impact of oligo-mRNA interactions. Managed research collaboration with Oligonucleotide biotech company (ProQR Therapeutics)