CD34+ selection, transduction, and formulation for ex-vivo hematopoietic gene therapy — with vector and GMP release testing inside one organization.
Hematopoietic stem cell (HSC) gene therapy — ex-vivo HSC or CD34+ gene therapy — collects mobilized CD34+ cells, transduces them with a vector carrying a corrective or therapeutic gene, and returns them to the patient after conditioning so the engineered stem cells repopulate the blood system. It is a one-time, potentially curative approach used in inherited hematologic, metabolic, and immune disorders. The constraint is that the cell population is small, precious, and not replaceable.
GeneFab is a stem cell CDMO for GMP HSC and CD34+ ex-vivo gene therapy manufacturing. We cover CD34+ selection, transduction optimization against vector we manufacture ourselves, closed formulation and cryopreservation, and the vector copy number, identity, and potency analytics a hematopoietic release package requires.
HSC programs enter with a construct, a transduction protocol, or a target indication. These are the pieces we own.
Mobilized peripheral blood, bone marrow, and cord blood–derived material. Selection strategy, chain of identity, and material acceptance criteria are defined during tech transfer, before the first engineering run.
Payload and regulatory element design from our synthetic biology group, with promoter selection appropriate to hematopoietic lineages and screening in primary CD34+ cells.
Transduction efficiency against vector copy number limits, with vector we manufacture ourselves so potency and process conditions are developed together rather than inherited.
Closed wash, concentration, and cryopreservation into the final container, designed to protect a cell population you cannot make more of.
Identity, purity, potency, and safety assays developed against the process, with core biosafety testing performed in house.
An integrated GMP project team — process, analytical, QA, and program management in one group — carries the process from engineering runs into GMP manufacturing.
You cannot re-collect a patient's mobilized CD34+ cells because a process step was too aggressive. Cell recovery is a design constraint on every unit operation, not a metric you report afterwards.
Every handling step is a loss. Process design minimizes both the number of steps and the time cells spend in them.
Transduction efficiency optimized against a vector made in the same organization, so both sides of the equation are ours to tune.
Cell recovery treated as a process attribute with a target, tracked from the first engineering run.
What sponsors ask before choosing a stem cell CDMO for a hematopoietic program.
Yes. We manufacture hematopoietic stem cell and CD34+ cell therapies under GMP, including CD34+ selection, transduction, formulation, and GMP release testing.
Mobilized peripheral blood apheresis, bone marrow–derived material, and cord blood–derived material, fresh or cryopreserved. Material specifications and chain of identity are defined during tech transfer.
Yes. We manufacture lentiviral vector in adherent and suspension formats and run the cell process in the same organization, so transduction conditions and vector potency are developed together.
Both are treated as process attributes with analytics developed alongside the process, so the trade-off between efficiency and copy number limits is characterized rather than discovered late.
Process design minimizes handling steps and time, and cell recovery is tracked as a target attribute from the first engineering run.
A technical exchange under CDA, then a gap assessment of your process and analytics, a tech transfer plan, and engineering runs before GMP. Requesting a quote starts that conversation.
