CRISPR Therapeutics: cross-border access for the specialty portfolio
Quick orientation
CRISPR Therapeutics AG is a Swiss-incorporated, US-headquartered (Boston, Massachusetts) biotechnology company developing CRISPR/Cas9 gene-editing therapies. The company is a co-developer (with Vertex Pharmaceuticals) of the first FDA-approved CRISPR/Cas9 gene-edited therapy. CRISPR Therapeutics trades on NASDAQ as CRSP. FDA approval scale is one co-marketed product (Casgevy), with a pipeline that includes additional hematology, oncology, and in vivo gene-editing candidates.
CRISPR Therapeutics portfolio in cross-border NPP scope
Casgevy (exagamglogene autotemcel), co-developed and co-commercialised with Vertex Pharmaceuticals, is an FDA-approved CRISPR/Cas9 gene-edited autologous hematopoietic stem cell therapy for: sickle cell disease in patients twelve years of age and older with recurrent vaso-occlusive crises (FDA approval December 2023); and transfusion-dependent beta thalassemia in patients twelve years of age and older (FDA approval January 2024). Casgevy is a one-time therapy: the patient's CD34-positive cells are collected, edited ex vivo with CRISPR/Cas9 to disrupt BCL11A, and reinfused after myeloablative conditioning. The edited cells produce elevated fetal hemoglobin which substitutes for the missing or mutated adult hemoglobin in beta thalassemia or sickle cell disease.
US list pricing for Casgevy has been reported at US$2.2 million per one-time treatment. The product is administered only at authorised treatment centers with hematopoietic stem cell transplantation capability and CRISPR/Cas9 gene therapy infrastructure.
Cross-border coordination of autologous gene therapies is operationally distinct from coordination of off-the-shelf pharmaceutical product. Reserve Meds does not coordinate the gene therapy product itself, but provides patient navigation support for international families seeking access to US authorised treatment centers.
CRISPR Therapeutics pipeline includes CTX112 (allogeneic CAR-T for B-cell malignancies), CTX131 (allogeneic CAR-T for solid tumors), and in vivo gene-editing candidates for cardiovascular and other indications. These are investigational and not in NPP scope.
Why patients route through cross-border NPP
Sickle cell disease affects approximately 100,000 people in the United States and an estimated several million globally, with particularly high prevalence in sub-Saharan Africa, the Middle East, India, and parts of the Mediterranean. Transfusion-dependent beta thalassemia is concentrated in similar geographies. Until Casgevy and Lyfgenia (bb1111, Bluebird Bio), the only potentially curative therapy was allogeneic hematopoietic stem cell transplantation, which requires a matched donor. Casgevy uses the patient's own cells, eliminating donor-matching requirements and graft-versus-host disease risk.
Outside the United States, Casgevy is approved in the United Kingdom, the European Union, and select other markets, with commercial availability rolling out. For international families with confirmed sickle cell disease or transfusion-dependent beta thalassemia diagnoses and the clinical and financial readiness to pursue gene therapy, Reserve Meds provides navigation support to US authorised treatment centers. The decision pathway typically involves multi-disciplinary review at the treating center including hematology, transplant medicine, fertility preservation counselling, and financial counselling.
Common cross-border destinations
Patient travel for CRISPR Therapeutics gene therapy is inbound to US authorised treatment centers. Inbound patient origins for sickle cell disease and beta thalassemia navigation are primarily the India, the Saudi Arabia, the United Arab Emirates, Qatar, and Turkey. Consanguineous-marriage and population-genetic factors in several of these jurisdictions contribute to high beta thalassemia prevalence.
Manufacturer engagement posture
Reserve Meds does not coordinate Casgevy product directly. We provide patient navigation support for international families seeking access to US authorised treatment centers offering Casgevy. We are not affiliated with CRISPR Therapeutics or Vertex Pharmaceuticals. For CRISPR Therapeutics commercial teams interested in international patient pathways, our BD contact is [email protected].
What Reserve Meds provides
For CRISPR Therapeutics navigation, Reserve Meds coordinates: identification of US authorised treatment centers offering Casgevy; medical record transfer support for eligibility evaluation; destination-country regulatory documentation for cross-border medical travel; coordination with treating-physician referrals; and a dedicated coordinator for the navigation process. We do not coordinate product shipment for autologous gene therapy.
Clinical governance and prescription review
Every prescription for a product on this page is reviewed by a US-licensed pharmacist before dispensing. Reserve Meds does not substitute generic equivalents for branded specialty products without an explicit prescriber instruction, and we do not dispense any product whose chain-of-custody we cannot serialize back to the FDA-authorised manufacturer or its licensed wholesale partner. Our clinical review confirms indication-on-label appropriateness based on the documentation submitted by the treating physician, identifies any standard drug-drug interaction or contraindication concerns, and confirms that the requested dose, dosing schedule, and dosage form match the prescription. Where the prescription is unclear or where the indication on the prescription is off-label, our pharmacist contacts the prescriber directly. We do not provide medical advice to patients beyond drug-information counseling at dispensing.
Supply chain integrity
Supply integrity is the foundation of Reserve Meds operations. Every shipment carries a serialized DSCSA pedigree document confirming the chain of ownership from the FDA-registered manufacturer or its authorised distributor through a licensed US wholesale distributor to Reserve Meds and onward to the named patient. We do not source through parallel-import channels, third-country re-import, or unverified online wholesale markets. Cold-chain shipments are packed in qualified shippers with temperature data-loggers reading and logging temperature throughout the journey; data-logger reads are reviewed on arrival, and any out-of-specification excursion triggers our quality-hold protocol. For frozen products such as gene therapies and certain antibody-drug conjugates, we use validated frozen-shipper systems with dry-ice replenishment as required.
Destination-country regulatory pathways
Cross-border named patient access operates under jurisdiction-specific regulatory frameworks. In the United Arab Emirates, the Ministry of Health and Prevention (MOHAP) and Dubai Health Authority operate import-permit pathways for unregistered or restricted medicines on a named-patient basis. Saudi Arabia, Qatar, Kuwait, Bahrain, and Oman each operate analogous Ministry of Health named-patient frameworks with country-specific documentation requirements. India operates a personal-import pathway under the Drugs and Cosmetics Rules for quantities consistent with personal therapeutic use, typically up to three months supply per shipment. Turkey, Egypt, Jordan, Lebanon, and Morocco each operate distinct named-patient or import-permit pathways. Reserve Meds maintains current regulatory intelligence for each of these jurisdictions and prepares the documentation package required for each shipment.
What to expect through the coordination process
Patients beginning a request receive an initial coordinator contact within one business day of submission. The coordinator reviews the prescription, identifies the documentation required for the destination country, and provides an indicative cost range. Once required documents are uploaded - prescription, prescriber license verification, patient identification, and any indication-specific documentation such as genetic test results or biomarker reports - we issue a firm quote inclusive of product cost, packaging, shipping, regulatory documentation, and our coordination fee. Patients confirm acceptance of the firm quote before any sourcing or shipping is initiated, and Reserve Meds does not collect any deposit at intake. Once the firm quote is accepted and payment is received, the coordinator initiates sourcing through our licensed wholesale partners and prepares the destination-country regulatory package. Typical end-to-end timing from accepted quote to delivery is two to four weeks for ambient-shipped products and three to five weeks for cold-chain shipments, with variability driven by destination-country customs and regulatory turnaround times.
Not affiliated with the manufacturer
Reserve Meds is an independent operator of named patient program coordination. We are not a subsidiary, affiliate, authorised distributor, or licensed representative of any pharmaceutical manufacturer, and no statement on this page should be read as a partnership, endorsement, or representation by the named manufacturer. Trade names and product references on this page are used for informational purposes to identify the FDA-approved products that Reserve Meds coordinates through US specialty wholesale channels on behalf of named individual patients.
Privacy and data handling
Patient data submitted to Reserve Meds is handled under our HIPAA-aligned privacy policy and our destination-country data protection commitments. We minimise data collection to what is necessary for prescription verification, regulatory documentation, and shipping logistics. Patient identifiable information is shared with US wholesalers, destination-country regulatory authorities, and shipping carriers only as required to complete the coordination, and is not used for any marketing purpose.
Next step for patients
Patients or caregivers with confirmed sickle cell disease or transfusion-dependent beta thalassemia seeking US treatment center access for Casgevy can begin a navigation request now. Start a Casgevy navigation request ›
Next step for CRISPR teams
CRISPR Therapeutics medical affairs or commercial teams interested in international patient navigation partnerships can reach our BD team at [email protected].
Reserved for you.