Providing Expertise to the

Regenerative
Medicine Industry

A living therapy is only as approvable as the process that makes it. We build the CMC, comparability, and evidence CBER actually reviews.

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The regulatory reality

Living therapies are regulated as biologics — and judged on the process.

Cell therapies, gene therapies, and tissue-engineered products are biologics, licensed through a BLA and reviewed by CBER's Office of Therapeutic Products (OTP). Unlike a small molecule, the product is often inseparable from the process that makes it, so your manufacturing, potency, and comparability story is your approval story.

Sponsors move fast on the clinical side and discover late that CMC is the critical path: a potency assay that was never qualified, a comparability gap after a process change, a vector supply that can't scale. We embed as senior leaders to align the regulatory strategy, the CMC package, and the evidence plan long before they collide at the pre-BLA meeting.

Where your product sits

Four modalities, four regulatory conversations.

The framework, the review division, and the evidence bar shift with the modality. We position each on the pathway it actually travels.

Cell Therapies

Autologous and allogeneic cell products: CAR-T, TILs, stem-cell and NK platforms, where donor variability and potency dominate the review.

CBER / OTPIND → BLA21 CFR 1271 / 610

Gene Therapies

In vivo AAV and lentiviral vectors and ex vivo gene-modified cells, where vector characterization, shedding, and immunogenicity drive the package.

CBER / OTPRMAT-eligible15-yr LTFU

Tissue-Engineered

Engineered tissues, scaffolds, and combination constructs, where the primary mode of action decides device-versus-biologic jurisdiction and the review center.

CBER / OCPCombination product21 CFR 3.2(e)

Genome-Edited

CRISPR and base/prime-edited products, where off-target analysis, editing fidelity, and durability shape both the CMC and the clinical evidence plan.

CBER / OTPOff-target strategyNovel endpoints
The expedited ladder

Every accelerated program is leverage — if you earn it early.

Regenerative products can stack expedited designations. Each one buys time, access, or flexibility, and each one raises the bar on CMC readiness.

Section 506(g)RMAT
Regenerative Medicine Advanced Therapy: all Breakthrough features plus early, intensive FDA interaction on surrogate endpoints and expanded ways to satisfy post-approval requirements. The designation built for this industry.
Section 506(a)Breakthrough
Preliminary clinical evidence of substantial improvement over available therapy, with organizational commitment from FDA and rolling review of the BLA.
Section 506(b)Fast Track
Serious condition and unmet need: more frequent meetings, rolling submission, and eligibility for accelerated approval and priority review.
Review clockPriority Review
A six-month review goal instead of ten when the product would significantly improve safety or effectiveness.
Subpart E / 601Accelerated Approval
Approval on a surrogate or intermediate endpoint reasonably likely to predict benefit, paired with a confirmatory trial obligation.
The critical path

CMC is where regenerative programs are won or lost.

Expedited clinical timelines compress the very work that takes the longest to get right. A potency assay you can't yet correlate to mechanism, a comparability gap after a scale-up, an out-of-spec vector lot: any one can stall a BLA that the clinical data had already earned. We treat CMC as the schedule-defining path, not the afterthought.

Scientist operating a closed-system cell-therapy manufacturing suite

Potency & bioassay

A matrixed potency strategy tied to mechanism of action, qualified before it becomes a pivotal-lot release gate.

Comparability

A pre-planned comparability protocol so a process or site change doesn't reopen the clinical package.

Analytical characterization

Orthogonal identity, purity, and safety assays that let you defend a living product against a moving specification.

Process validation

Aseptic process, closed-system control, and validation that holds at commercial scale, the aim from the first GMP lot.

After approval, the clock keeps running

A gene therapy commitment can outlast the trial by fifteen years.

Integrating vectors and durable edits carry delayed-risk obligations that most teams under-scope. We design the follow-up before the BLA, not after the letter.

15 yr

Long-term follow-up

FDA expects up to 15 years of follow-up for integrating vectors and genome-editing products, with a monitoring plan sized to the delayed-risk profile.

RCR

Replication-competent testing

Retroviral and lentiviral products carry defined RCR/RCL testing and reporting expectations across manufacturing and patient monitoring.

Reg.

Registries & real-world evidence

Patient registries and structured real-world data that satisfy post-marketing commitments and support label expansion over time.

How we help

Senior regenerative leadership, embedded in your program.

Fractional heads of regulatory and CMC who have taken advanced therapies from pre-IND to BLA, deployed where your gaps are.

RMAT Strategy Pre-IND & INTERACT IND & BLA Filings CMC Strategy Potency Assay Roadmap Comparability Protocols Pre-BLA Meetings LTFU Plans CDMO Oversight Chain of Identity Combination Jurisdiction Global Filings

Bring senior regenerative regulatory judgment onto your team.

From a first RMAT strategy to a BLA readiness review, we embed as the regulatory and CMC leadership advanced therapies require.

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