Paulina Marzec

LoQus23 is a DDF-formed company focussing on targeting DNA damage repair pathways with small molecules to treat Huntington’s disease and other triplet repeat diseases. Highlights: Leading a project aiming at target ID and validation Developing cell-based assays for target engagement and proof of principle studies Leading a biomarker development program Managing CROs for the design and delivery of decision-making experiments for support of the drug discovery program Line management experience

Freeline is a clinical-stage biotechnology company focused on developing transformative gene therapies for chronic debilitating diseases. Freeline uses its proprietary, rationally designed AAV vector and capsid (AAVS3), along with novel promoters and transgenes, to deliver a functional copy of a therapeutic gene into human liver cells, thereby expressing a persistent functional level of the missing or dysfunctional protein into a patient’s bloodstream. The company is currently advancing FLT201, a highly differentiated gene therapy candidate that delivers a novel transgene, in a Phase 1/2 clinical trial in people with Gaucher disease type 1. Freeline has additional programs in research, including one focused on GBA1-linked Parkinson’s disease that leverages the same novel transgene as FLT201. Highlights: Agile Team leader in matrix environment overseeing design and delivery of in vivo studies for characterizing rAAV dynamics in mice. Leading and executing Proof of Principle studies both in in-vivo and ex-vivo phase. Presenting data on Project Team meetings and providing recommendations to project teams. Successfully set up an in vivo model for Proof of Principle studies as well as molecular biology techniques in In Vivo team for downstream analysis of rodent tissues. Mentored and coached junior scientists in the team. Implemented solutions and improvements in pre-clinical development projects and studies by liasing with CROs and Royal Veterinary College.

Won a two-year fellowship to work on an authorship project investigating telomere protection in stem cells. I presented the data at the EMBO telomere conference in Lisbon as a selected speaker and received very good feedback resulting in two collaborations. Based on my contributions to the project I am co-corresponding author of the paper that breaks a dogma in the telomere field by showing that telomere protection and t-loop formation in stem cells is TRF2-independent. Mentored and supervised two PhD students on shared projects. Learnt from the experience how to motivate others and resolve conflicts.

Discovered mechanism of Targeted Telomeric Insertions (TTI) driving development of complex sarcomas while gaining expertise in telomere biology and chromatin composition techniques. Supervised and taught a master student on placement project. Collaborated with clinicians to deliver results from tumor biopsies. Won the Helene Starck Prize (2013) for young scientists in recognition for scientific achievements. Presented the data as a selected speaker at EMBO telomere conference in Brussels which set up my recognition in the telomere field.

CIRS is a NGO in Cracow (Poland) which support development of society acting through support of local culture, science and entrepreneurship. Spotted an opportunity to apply for generous funding from EU programme "Leonardo da Vinci" for a summer training abroad that was specifically for a group of students. Overcame university's reluctance to support the initiative by liaising directly with the NGO. Proactively recruited students in my year, co-ordinated research of potential biotech companies and wrote proposals. Negotiated terms of training agreements with biotech companies. Motivated students in keeping up the team effort by setting up an example and highlighting our progress. Won the funding for 15 students including myself for training in 5 different countries.

Three years in a row among top 5% students of the year. Specialised in cell and molecular biology. Studied for a year in Montpellier, France as a part of master thesis project focusing on DNA replication in early stages on cancer development. Passed MSc viva with distinction.