508‑988‑0105 · info@axiomregenerative.us 375 Totten Pond Rd, Waltham, MA 02451
Axiom Regenerative Sciences Cell Therapeutics Development
Research programs

Cell-therapeutics research with a U.S. development pathway

Focused research in COPD, systemic lupus erythematosus, and kidney injury—connected to product control, nonclinical evidence, clinical design, and FDA submission planning.

Disease programs

Three programs with distinct biology, risks, and endpoints.

The modality and evidence plan must be tailored to the disease. MSCs, hematopoietic transplantation, engineered immune cells, and extracellular vesicles cannot be treated as interchangeable.

PROGRAM 01
Investigational

Chronic obstructive pulmonary disease (COPD)

Cell-based research for chronic pulmonary inflammation and injury must balance biological rationale with route-specific and cardiopulmonary risk.

Development questions

  • Which COPD phenotype and disease stage fit the proposed mechanism?
  • How will identity, viability, potency, dose, route, and product consistency be controlled?
  • Which pulmonary, thrombotic, immunologic, and infusion risks require nonclinical and clinical monitoring?
  • Which endpoints can separate safety, biological activity, function, exacerbations, and patient-reported benefit?

Selected U.S. registry context

Selected completed U.S. MSC studies include Phase 1 NCT04047810 and Phase 2 NCT00683722. Their registry presence and completion do not establish efficacy or approval.

NCT04047810 · Phase 1NCT00683722 · Phase 2
PROGRAM 02
Investigational

Systemic lupus erythematosus

SLE development requires modality-specific thinking across immune dysregulation, disease heterogeneity, organ involvement, infection risk, and background therapy.

Development questions

  • Is the hypothesis immunomodulation, immune reset, cell replacement, or another defined mechanism?
  • How will the protocol stratify disease activity, organ involvement, prior therapy, and concomitant immunosuppression?
  • What potency model connects the product to the proposed immune mechanism?
  • How will flare, infection, cytopenia, thrombosis, organ outcomes, and durability be monitored?

Selected U.S. registry context

Selected completed U.S. MSC studies include Phase 1 NCT03171194 and Phase 2 NCT02633163. Other modalities, such as hematopoietic transplantation or engineered immune cells, require separate risk and regulatory analyses.

NCT03171194 · Phase 1NCT02633163 · Phase 2
PROGRAM 03
Investigational

Kidney injury

Kidney-injury programs must define the clinical setting, timing, cause of injury, recovery window, and interaction with renal replacement and supportive care.

Development questions

  • Is the target acute kidney injury, inflammatory injury, post-surgical injury, or a defined chronic population?
  • What timing, route, dose, biodistribution, and pulmonary-trapping risks apply?
  • Which models and biomarkers support mechanism, potency, renal safety, and patient selection?
  • Which endpoints address survival, dialysis, renal recovery, adverse events, and durability without confounding?

Selected U.S. registry context

A selected U.S. registry example is NCT06654193, a recruiting Phase 1/2 study of allogeneic adipose-derived MSCs for acute kidney injury at the review date. Recruitment status can change.

NCT06654193 · Phase 1/2
U.S. pathway

From product definition to IND and BLA

The pathway depends on the exact product and intended use. The sequence below is a planning framework, not a legal determination or a promise that FDA will agree.

Product definition & regulatory classification

Define source, manipulation, composition, intended use, route, dose form, combination components, and whether the product is regulated as a drug/biologic requiring an IND and marketing application.

CMC and control strategy

Establish starting-material controls, donor and tissue requirements where applicable, manufacturing process, in-process controls, identity, purity, viability, potency, safety testing, specifications, stability, and comparability.

Nonclinical evidence

Generate pharmacology and toxicology evidence appropriate to the modality, including biodistribution, persistence, local and systemic toxicity, tumorigenicity, immunogenicity, thrombogenicity, and dose/route rationale as relevant.

FDA interaction & IND submission

Use focused questions to resolve material gaps, then submit the required manufacturing, nonclinical, clinical protocol, investigator, informed-consent, and IRB-related information. A sponsor generally waits 30 calendar days after FDA receives an IND before initiating the study, unless FDA places it on clinical hold.

Clinical development & lifecycle evidence

Conduct authorized studies under the protocol and applicable requirements, manage safety reporting and amendments, and build progressively stronger evidence for dose, benefit-risk, manufacturing consistency, and the intended population.

BLA review & post-approval obligations

If the total evidence supports marketing, a biologics license application may seek licensure for a specific product and indication. Approval, labeling, inspections, pharmacovigilance, and postmarketing commitments are separate from IND status.

Primary sources

Verify against the current record.

Clinical status and FDA policy can change. These official sources should be checked again before any publication, investor statement, protocol decision, or submission.

FDA: Investigational New Drug (IND) Application FDA: Approved Cellular and Gene Therapy Products FDA: Therapeutic Biologics Applications (BLA) eCFR: 21 CFR Part 312 — IND eCFR: 21 CFR 1271.10 — HCT/P criteria ClinicalTrials.gov

Clinical and regulatory landscape last reviewed: August 16, 2026.

Build the evidence and development plan before making the claim.

Tell us the modality, indication, development stage, and decision you need to make next.

Discuss a development program