Oligonucleotide and Peptide Therapeutics: Accelerate Your Oligo and Peptide Therapeutics to Market
with GenScript!
Join us at TIDES USA
2025 at Booth #515, held at the Manchester Grand Hyatt San
Diego from May 19-22, 2025. This premier event will showcase cutting-edge
science across the Oligonucleotide, Peptide, Genome Editing, and mRNA landscape. The
TIDES USA conference will feature over 150 industry leaders, offering
insights into the latest advances and developments in oligo and peptide therapeutics. The scientific agenda
spans the full spectrum of drug development, from discovery and preclinical stages to clinical trials, CMC,
manufacturing, and the commercialization of therapeutics and vaccines.
Don’t miss the opportunity to visit us at Booth #515, attend our session, and explore our
posters to discover how GenScript can accelerate your research and bring your therapeutic
innovations to market faster!
GenScript Activities
Spotlight Presentation
Date/Time: Thursday, May 22, 2025, 12:20 pm – 12:50 pm
Title: Precision to Production: Advancing Gene Therapies through Targeted mRNA
Delivery and Scalable CRISPR Solutions
Abstract:
This talk bridges cutting-edge innovations in mRNA delivery systems with scalable,
regulatory-compliant CRISPR tools to address the dual challenges of precision and practicality in gene
therapy development.
Part 1: Targeted Lipid Nanoparticles loaded with mRNAs for Gene Editing
Recent advancements in RNA therapeutics highlight the potential of mRNA-based tools for in
vivo gene editing with lipid nanoparticles (LNPs) co-loaded with gene-editing enzymes and sgRNA,
offering a safer alternative to traditional methods. A key challenge is achieving efficient,
tissue-specific delivery. We optimized LNP formulations for targeted delivery to T cells and hepatic
cells, significantly enhancing protein expression and gene-editing efficiency. Our ReadyEdit platform
supports prime editing, base editing, and Cas enzyme applications at clinically relevant loci like PCSK9
and TRAC. Our LNPs have achieved over 60% editing efficiency in HEK293T cells and 20% in liver cells.
Codon optimization of enzyme-coding mRNA resulted in a fivefold increase in expression, maximizing the
potential of mRNA-based gene editing. These findings hold significant potential for advancing research
in RNA-based therapeutics.
Part 2: Securing the CRISPR Pipeline with Scalable cGMP Guide RNA
The promise of CRISPR-based therapies hinges on reliable, high-quality reagents. GenScript
addresses this need with scalable cGMP guide RNA (gRNA) production tailored for diverse CRISPR
systems—Cas9, Cas12a, prime editing, and base editing. We will detail our state-of-the-art manufacturing
infrastructure, featuring dedicated production lines, rigorous quality control, and rapid 30-day
clinical-grade gRNA synthesis. We will also explore how comprehensive regulatory support, including Drug
Master Files (DMFs) and IND-enabling documentation, accelerates therapeutic development. Case studies
highlight partnerships with biotech innovators to streamline workflows from preclinical validation to
commercial-scale production.
Speaker 1: Dr. Shambhavi Shubham
Title: Sr. Research Scientist
Company: GenScript
Bio: Dr. Shambhavi Shubham is an accomplished scientist with 10+
years of expertise in CRISPR editing, prime editing, mRNA solutions, and NGS technology.
Currently, Dr. Shubham is a Senior Scientist II at GenScript, leading gene editing and mentoring
teams. Former Principal Scientist at Salish Bioscience, driving R&D for next-gen liquid biopsy
assays. Extensive experience in developing DNA aptamers, enzyme kinetics, and novel assays for
cancer research and diagnostics. Strong background in cross-functional collaboration and project
management. Proven ability to innovate in molecular biology, genomics, and biotechnology, with a
strategic perspective from previous roles in licensing and technology commercialization.
Speaker 2: Dr. Jianpeng Wang
Title: Senior Director of GMP Production Dept.
Company: GenScript
Bio: Dr. Jianpeng Wang received his doctor’s degree in
chemistry from the Chinese University of Hong Kong, he has more than 15 years’ experience
in peptide & nucleic acid chemistry and filed 20+ patents. Since joining GenScirpt in 2015, Dr.
Jianpeng Wang has led the nucleic acid R&D and manufacturing departments to develop, launch and
deliver the nucleic acid reagents for CRISPR. Specifically for the guide RNA, Dr. Jianpeng Wang
led the GenScript team to deliver more than 120 GMP batches, supported 40+ clinical programs,
and got 13 global IND approvals.
Posters
Topic: Targeted LNP for improving mRNA Delivery
Presenter: Dr. Shambhavi Shubham
Job Title: Sr. Research Scientist
Abstract: mRNA therapeutics present a groundbreaking opportunity
for in vivo gene editing applications. However, the success of these therapies hinges on the
ability to efficiently and precisely deliver RNA to target cells. In this study, we developed
and evaluated several innovative delivery platforms, including antibody-decorated lipid
nanoparticles (LNPs), peptide-decorated LNPs, and modified formulation compositions. We
systematically compared their delivery efficiency both in vivo for marker gene expression and in
vitro for gene editing. Our findings revealed that using a modified SM102-LNP could greatly
improve delivery efficiency for prime editing in vitro. Additionally, ligand-decorated LNPs show
great potential for stem cell gene editing in vivo, representing a critical advancement in
optimizing targeted RNA delivery. These results highlight the potential of tailored nanoparticle
platforms to enhance the therapeutic efficacy of mRNA-based gene editing, offering a path toward
more precise and effective treatments for a range of genetic disorders.
Topic: Harnessing Ultra-Long Guide RNA to Advance Gene Therapy
Presenter: Dr. Jianpeng Wang
Job Title: Senior Director of GMP Production Dept.
Abstract: CRISPR gene editing, exemplified by the approved therapy
CASGEVY™ for sickle cell disease, demonstrates therapeutic efficacy but carries risks of
off-target effects due to double-strand breaks (DSBs). To address this, Prime Editing—a
"search-and-replace" system avoiding DSBs—has emerged, utilizing three components: (1) pegRNA to
target sequences and encode edits, (2) Cas9 H840A nickase for single-strand nicking, and (3)
M-MLV reverse transcriptase to synthesize desired DNA from the RNA template. Recent
optimizations in these components have enhanced pegRNA purity and purification efficiency,
advancing precision in genome editing with reduced risks.
Most Popular Products
CRISPR Gene Editing
Antibodies
Protein Electrophoresis and Blotting
Molecular Biology
Stable Cell Lines
Cell Therapy
Diagnostics
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