Genetic medicine · Discovery through lifecycle management
VectorSim LLC works with startups, clinical-stage and commercial companies, CDMOs, and CROs on AAV, siRNA, and bioconjugate programs. Hands-on support for candidate selection, process development, GMP manufacturing, PPQ, BLA submission, and everything in between.
At a glance
Our team supported the process and CMC work behind a U.S. FDA Platform Technology Designation granted to a viral vector platform, and we bring that experience to designation requests, briefing books, and platform strategy for new programs.
Services, in the order your program moves
Each stage below is a place we can step in: to lead, to augment your team, or to deliver a scoped workstream. Most clients start with one stage and stay for the next.
Target → lead candidate → proof of concept
We help teams move from a target to a defensible lead. That includes our siRNA screening platform, which ranks candidate sequences for a target by predicted efficacy, off-target risk, and cross-reactivity, and it includes navigating CROs and CDMOs to generate proof-of-concept material without losing months.
Submit a target and receive a ranked list of top candidate sequences scored on predicted efficacy, off-target effects, cross-reactivity across species, and immunostimulatory motifs, so wet-lab screening starts with the right handful of sequences instead of hundreds.
| Rank | Candidate | Predicted efficacy | Off-target | Cross-reactivity |
|---|---|---|---|---|
| 01 | siR-0417 | Low | Human · NHP · Mouse | |
| 02 | siR-1122 | Low | Human · NHP | |
| 03 | siR-0088 | Moderate | Human · NHP · Mouse | |
| 04 | siR-2301 | Low | Human | |
| 05 | siR-0956 | Low | Human · NHP |
Preclinical → IND-enabling
Upstream, downstream, and analytical development designed as a platform from the start, with the risk analysis, scale-down models, and phase-appropriate quality systems needed to support an IND and the campaigns that follow.
Technology transfer → GMP clinical supply
We navigate and manage CDMOs and CROs for GMP material production, lead technology transfer, review the batch records, and put QC and data analysis in place so every batch is understood before it is released.
PPQ → BLA → approval
From process performance qualification through BLA submission, with FDA meeting strategy and briefing books that get the right questions answered. We write the CMC sections, prepare the team for the meeting, and manage the responses that follow.
Our team supported the process and CMC work behind a U.S. FDA Platform Technology Designation granted to a viral vector platform, and can help you build the data package and briefing documents for your own request.
Commercial → post-approval → next generation
Post-approval changes, comparability, continuous process verification, and second-generation processes handled with the same rigor as the original filing, plus the Six Sigma tools to make improvement measurable.
Who we work with
The support is right-sized to the organization. A three-person virtual biotech and a commercial manufacturer need different things from us, and we scope accordingly.
Senior technical leadership from candidate selection through IND, so early decisions do not become late problems.
A tech transfer, a PPQ campaign, a BLA module, an FDA meeting: defined scope, senior hands, clean handoff.
We help partners scope, execute, and de-risk programs for their customers, and we help sponsors get the most out of their partners.
How we engage
Resolve a defined technical question, decision, or program risk in days, not quarters.
Add experienced discovery or CMC capacity to your team through a critical development phase.
Scope and execute a specific workstream with clear deliverables: a briefing book, a qualified scale-down model, a tech transfer package.
About VectorSim LLC
VectorSim LLC was founded by Nermin Ibreljic, Founder and Managing Member, who spent his industry career as a process engineer in gene therapy manufacturing, most recently as a Principal Engineer, with hands-on experience in process development, scale-up, technology transfer, and CMC on AAV programs from early development through commercial manufacturing.
We bridge industrial bioprocess engineering and graduate-level biotechnology teaching, and we bring Six Sigma Black Belt discipline to the data. VectorSim is not a software company learning biology. It is bioprocess engineers doing the work they have done in-house, now for your program.
Our mission is to compress the time between a promising candidate and an approved medicine, so the next generation of genetic medicines reaches patients faster.
Start with the immediate challenge
Share the modality, the stage, and the decision in front of your team. We will tell you honestly whether and how we can help, usually within a business day.
