Cell and Gene Therapy Consulting

Aclairo’s Cell and Gene Therapy group has extensive experience providing strategic advice, program support, and regulatory writing for many CGT modalities, including AAV, lentivirus, LNP-delivered nucleic acids, in vivo and ex vivo gene editing, and cell therapies.

Cell and Gene Therapy Consulting Overview

Aclairo consultants in the CGT group support the development of complex therapeutic modalities by guiding nonclinical strategy, regulatory planning, study design and data interpretation. Programs involving viral vectors, gene editing technologies, and cell-based therapies require specialized expertise to navigate evolving regulatory expectations and scientific challenges. Aclairo’s cell and gene therapy consulting team provides strategic guidance across the development lifecycle, helping sponsors design efficient nonclinical programs, prepare for regulatory interactions, and ensure that studies are aligned with global regulatory expectations. Our experience spans a wide range of platforms, including viral and non-viral delivery systems, enabling tailored approaches for each program.

When is Cell and Gene Therapy Consulting Needed?

Cell and gene therapy consulting is typically engaged at key decision points in program development, including:

Early-stage program planning and feasibility assessment
Preparation for INTERACT or pre-IND meetings
Design and placement of nonclinical safety and biodistribution studies
Implementation of analytical and biomarker strategies
When a development candidate has been identified
Addressing regulatory concerns or feedback
Candidate in-licensing or out-licensing opportunities

Expertise in Cell and Gene Therapy Development

Aclairo’s Cell and Gene Therapy group have extensive experience providing strategic advice, program support, and regulatory writing for many modalities, including AAV, lentivirus, LNP-delivered nucleic acids, in vivo and ex vivo gene editing, gene-modified cell therapies (in vivo and ex vivo CAR-T), and cell therapies.

The CGT team has experience with products developed for many indications and routes of administration, including:

Direct intraparenchymal administration of AAV for neurological diseases
ICV and IT-administered AAV for neurology indications
Systemic administration of AAV for many metabolic disorders
Systemic administration of LNP-encapsulated in vivo gene editors for both rare and common diseases
Intratumoral administration of oncology gene therapies
Ex vivo lentiviral transduction of various immune cells for oncology and immunology indications

We routinely assist clients with:

Developing an efficient nonclinical package to meet regulatory requirements
Drafting effective briefing documents and questions for INTERACT and preIND meetings
Identifying critical endpoints to include in nonclinical studies
Design and placement of nonclinical studies
Study monitoring
Generation of Investigator’s Brochures, INDs, and CTAs to initiate clinical trials across geographic regions

Related Services

Nonclinical Drug Development Operations Scientific & Regulatory Writing

Frequently Asked Questions

What is included in nonclinical cell and gene therapy consulting?
Cell and gene therapy consulting typically includes nonclinical strategy development, regulatory document preparation, study design, biomarker strategy and preparation for health authority interactions; however, we tailor support based on the needs of the client.
When should I engage a nonclinical cell and gene therapy consultant?
Consulting support is most valuable early in development, particularly before INTERACT or pre-IND meetings and during nonclinical program design.
Which consultant will support my program?
We match consultants to clients based on expertise, bandwidth, client preference, and the level of support needed. You will have one primary point of contact, but all of Aclairo is available to pitch in as issues arise.
How do nonclinical development plans for CGT products differ from small molecule drugs?
CGT programs are more tailored to the biology and indication and have fewer required “checkbox” studies. Because they are often higher-risk, single dose treatments, preclinical identification of potentially efficacious doses is more important for CGT products as the number of dose levels used in clinical trials is typically much smaller than for small molecules.