Bridging preclinical
to clinical
Evers Genetic Therapy Consultancy (EGTC) provides support from idea to IND. Hands-on experience in early development, specializing in CNS and Neurodegeneration drug development. EGTC excels in bridging preclinical to clinical stages through translational science and value stream mapping. With a deep knowledge of assay development, cell culture, delivery and in vivo proof-of-concept studies, EGTC aims to accelerate research to the patient.
About
Leveraging my extensive experience from conception to the clinic, I aim to assist others in accelerating their (genetic) therapies to reach patients in need. Whether it’s gene therapy, oligonucleotides, or another delivery vehicle, I am here to provide guidance on feasibility, delivery, and general translational strategies to the clinic. With deep expertise in the neuromuscular and neurodegenerative disorders, I bring a unique perspective to drive success in these challenging areas. In addition, I am skilled to taking on (interim) roles in team or department leadership.
My mission is to work as a team every day to generate results, innovate and advance medicinal products and to help patients and families in need.
Story telling event at Regent Theater Arlington, MA: Dr. Melvin Evers | What #HuntingtonsDisease Tells Us About Living https://youtu.be/hmnE7Y9hUHA
Experience
As the founder of Evers Genetic Therapy Consultancy, I utilize my extensive expertise to help others accelerate their genetic therapies for patients in need. My specialty encompasses genetic therapies (such as gene therapy and oligonucleotides), offering guidance on feasibility, delivery, and translational strategies to the clinic.
As Vice President of Global Research I led a cross-functional, global research team with a complex gene therapy portfolio in central nervous system, metabolic and liver-directed indications and spearheading innovations such as miQURE® and goQURE™.
My academic foundation includes a PhD in Human Genetics from Leiden University, focusing on genetic therapies for neurodegenerative disorders. Thesis: Developing Genetic Therapies For Polyglutamine Disorders (https://hdl.handle.net/1887/30254). I have worked with Prosensa Therapeutics on triplet repeat targeting using oligonucleotides (Now further developed by Vico Therapeutics), at the Leiden Genome Technology Center to perform next-generation sequencing, and founded an iPSC neuronal differentiation lab at the Leiden University Medical Center.
Whether it is project leadership with target nomination, candidate selection, value-stream mapping, target product profiling, regulatory strategy, or preclinical to clinical translation with preclinical proof-of-concept studies, large animal administration and distribution, in vitro, mechanism-of-action, assay and biomarker development, dose-translation, I am skilled to advise and assist to bring medicinal products as safe, efficacious, and fast as possible to the patients in need.
Responsible for pivotal preclinical studies with successful IND clearance for 6 currently in-human clinical stage programs. Here are some examples:
“uniQure Announces Completion of Enrollment in the First Cohort and Favorable Recommendation from the Independent Data Monitoring Committee for its Phase I/IIa Clinical Trial of AMT-191 for the Treatment of Fabry Disease” https://uniqure.gcs-web.com/node/11976/pdf
“uniQure Announces Alignment with FDA on Key Elements of Accelerated Approval Pathway for AMT-130 in Huntington’s Disease” https://uniqure.gcs-web.com/node/11906/pdf
“Vico Therapeutics Announces Positive Interim Phase 1/2a Clinical Data of VO659 in Treatment of Huntington’s Disease” https://vicotx.com/vico-therapeutics-announces-positive-interim-phase-1-2a-clinical-data-of-vo659-in-treatment-of-huntingtons-disease/
Some ongoing scientific advice:
Scientific Advisor CureQ consortium
https://cureq.nl/consortia/advisory-committee/
CureQ is a consortia of researchers, clinicians/neurologists, experts on Medical Ethics, data management and AI analysis as well as a representative from patient foundations. The consortium aims to enable polyQ targeting therapies to better predict onset and progression of disease of the different patient groups (early-onset, adult-onset and carriers of intermediate repeats).
Scientific Advisor JAMA Therapeutics
JAMA Therapeutics is at the forefront of creating a new category of treatments, utilizing and enhancing the natural delivery capabilities of extracellular vesicles. By leveraging our proprietary exosome platform technology, VINCI™, developed at the University Medical Center Utrecht, our aim is to revolutionize the way we treat a broad range of life-threatening diseases, thereby significantly improving human health.
https://jamatherapeuticscom.wordpress.com/
Leadership
Experience leader, heading a global team consisting of over 20 nationalities in different countries. Experience with agile methodology.
Certified Board of Director (2023 Board Company)
Communication
Experienced in patient, scientific, medical, regulatory and investor-oriented communication. Hosting of patient days, contributing to investor calls and R&D days, presenting at clinical, scientific advisory boards and international conferences. Presenting to and interacting with regulatory agencies.
Quotes
“I’m sure that your enthusiastic personality, combined with your thorough in-depth knowledge will be valued, and that many can benefit from your expertise in this area.”
“Your brilliance and leadership will carry you wherever you go!”
“Your experience and people oriented personality will bring so much value to any company.”
“Your experience will be invaluable to anyone developing a gene therapy.”
Publications
https://www.researchgate.net/profile/Melvin-EversPatents
Over 10 patents as co-inventor, current list of published patents:
Nucleic acid regulation of snca. WO2023198663A1 · Issued Oct 23, 2023WO2023198663A1 · Issued Oct 23, 2023
The present invention relates to a nucleic acid comprising two or more RNA encoding sequences, wherein at least one of the sequences comprises a guide sequence substantially complementary to part of the SNCA gene. The invention also relates to associated AAVs, compositions, pharmaceutical compositions and uses in treatments thereof.
Nucleic acid regulation of c9orf72. WO2023198702A1 · Issued Oct 23, 2023WO2023198702A1 · Issued Oct 23, 2023
The present invention relates to a nucleic acid comprising two or more RNA encoding sequences, wherein at least one of the sequences comprises a guide sequence substantially complementary to part of the C9orf72 gene. The invention also relates to associated AAVs, compositions, pharmaceutical compositions and uses in treatments thereof.
Novel systems for nucleic acid regulation. WO2023198662A1 · Issued Oct 19, 2023WO2023198662A1 · Issued Oct 19, 2023
The present invention relates to a nucleic acid comprising two or more RNA encoding sequences, wherein the each of the RNA comprises a hairpin and a guide sequence substantially complementary to part of a gene of choice, and to associated AAVs, compositions, pharmaceutical compositions and uses in treatments thereof.
Targeting misspliced transcripts in genetic disorders. US20220213482A1 · Issued Jul 7, 2022US20220213482A1 · Issued Jul 7, 2022
The present invention relates to repeat expansion disorders. Missplicing is understood to be a general phenomenon that can occur in repeat expansion disorders wherein the DNA and/or RNA sequence of repeat sequences in expanded repeat disorders can cause such aberrant transcription and/or aberrant splicing, resulting in misspliced transcripts, i.e. transcripts that do not have the putative splicing as observed e.g. for corresponding non-diseased genes. Such misspliced transcripts can be in particular associated with disease. Hence, the current invention now provides means and methods for targeting misspliced transcripts which is highly useful for the treatment of expanded repeat disorders. The present invention relates to repeat expansion disorders.
RNAi induced reduction of ataxin-3 for the treatment of Spinocerebellar ataxia type 3. US US20200199625A1 · Filed Jan 1, 2019US US20200199625A1 · Filed Jan 1, 2019
The current invention relates to gene therapy approaches for the treatment of SCA3, in particular RNAi based gene therapy approaches utilizing a total knockdown approach. The inventors provide for selected target regions and/or target sequences for which highly efficient knockdown of the ATXN3 gene expression can be advantageously obtained in human neuronal cells and in mouse models relevant for SCA3.
Antisense oligonucleotide directed removal of proteolytic cleavage sites, the HCHWA-D mutation, and trinucleotide repeat expansions. US 20170226521 · Issued Aug 10, 2017US 20170226521 · Issued Aug 10, 2017
Described are methods for removing a proteolytic cleavage site, the HCHWA-D mutation or the amino acids encoded by a trinucleotide repeat expansion from a protein comprising providing a cell that expresses pre-mRNA encoding the protein with an anti-sense oligonucleotide that induces skipping of the exonic sequence that comprises the proteolytic cleavage site, HCHWA-D mutation or trinucleotide repeat expansion, respectively, the method further comprising allowing translation of mRNA produced from the pre-mRNA.
Antisense oligonucleotide directed removal of proteolytic cleavage sites from proteins. US 20170175117 · Issued Jun 22, 2017US 20170175117 · Issued Jun 22, 2017
The invention relates to means and methods for removing a proteolytic cleavage site from a protein comprising providing a cell that expresses pre-mRNA encoding the protein with an anti-sense oligonucleotide that induces skipping of the exonic sequence that encodes the proteolytic cleavage site, the method further comprising allowing translation of mRNA produced from the pre-mRNA.