Rocket Pharmaceuticals: cross-border access for the specialty portfolio

Quick orientation

Rocket Pharmaceuticals, Inc. is a US clinical-stage biotechnology company headquartered in Cranbury, New Jersey, focused on gene therapy for rare pediatric diseases. Rocket trades on NASDAQ as RCKT. The company's pipeline includes both lentiviral ex vivo gene therapies (for Fanconi anemia, leukocyte adhesion deficiency-I, and pyruvate kinase deficiency) and AAV-based in vivo gene therapies (for Danon disease and other cardiac and neurological indications). FDA approval scale is currently one marketed gene therapy product, Kresladi for severe leukocyte adhesion deficiency type I (LAD-I), with multiple Phase 2 and Phase 3 candidates approaching potential regulatory action.

Rocket portfolio in cross-border NPP scope

Kresladi (marnetegragene autotemcel) is an FDA-approved autologous lentiviral gene therapy for the treatment of severe leukocyte adhesion deficiency type I (LAD-I) in pediatric and adult patients (FDA approval December 2024). LAD-I is an ultra-rare inherited immunodeficiency caused by mutations in the ITGB2 gene encoding the beta-2 integrin subunit CD18. Affected children experience life-threatening recurrent bacterial infections, impaired wound healing, and very high early mortality without hematopoietic stem cell transplantation. Kresladi is a one-time autologous gene-corrected cell therapy: the patient's CD34-positive hematopoietic stem cells are collected, transduced ex vivo with a lentiviral vector carrying functional ITGB2, and reinfused after conditioning chemotherapy. US list pricing has been reported in the range of US$3.4 million per one-time treatment.

Cross-border coordination of autologous gene therapies is operationally distinct from coordination of off-the-shelf pharmaceutical product. Autologous gene therapy requires the patient to travel to or coordinate cell collection at an authorised treatment center capable of CD34 mobilisation, leukapheresis, conditioning chemotherapy, and gene-corrected cell reinfusion. The product itself is not transportable in the same way as a vial of biologic. Reserve Meds does not provide turnkey autologous gene therapy logistics; for Kresladi specifically, we provide patient navigation and referral support to US-based authorised treatment centers but do not handle the cell manufacturing or product shipment.

Rocket's pipeline includes RP-A501 (AAV gene therapy for Danon disease), RP-A601 (PKP2 cardiomyopathy), FA-RPL102 (Fanconi anemia gene therapy), and RP-L301 (pyruvate kinase deficiency gene therapy). These are investigational and not in NPP scope.

Why patients route through cross-border NPP

Severe LAD-I is ultra-rare globally, with estimated incidence of less than one per million live births. Affected children frequently die in early childhood without curative therapy. Historically, allogeneic hematopoietic stem cell transplantation has been the only curative option, but matched donor availability and transplant-related mortality limit access. Kresladi as an autologous gene therapy is a paradigm shift but operationally complex: cross-border patient navigation requires coordination of pre-treatment evaluation, travel to a US treatment center, and post-treatment follow-up care.

Reserve Meds supports international LAD-I families with patient navigation: identifying authorised US treatment centers, supporting medical record transfer for eligibility review, and providing destination-country regulatory documentation for cross-border medical travel. We do not coordinate the product itself; the autologous cell manufacturing and administration occur within the authorised US treatment center supply chain.

Common cross-border destinations

Patient travel for Rocket gene therapy is inbound to US authorised treatment centers. Inbound patient origins for LAD-I navigation referrals are primarily the Saudi Arabia, the United Arab Emirates, India, Qatar, and Turkey. Consanguineous-marriage demographics in some of these jurisdictions contribute to higher LAD-I incidence.

Manufacturer engagement posture

Reserve Meds does not coordinate Rocket Pharmaceuticals product directly. We provide patient navigation support for international LAD-I families seeking access to US authorised treatment centers offering Kresladi. We are not affiliated with Rocket Pharmaceuticals. For Rocket commercial teams interested in international patient pathways, our BD contact is [email protected].

What Reserve Meds provides

For Rocket gene therapy navigation, Reserve Meds coordinates: identification of US authorised treatment centers; 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

Caregivers of children with confirmed severe LAD-I (ITGB2 mutation) seeking US treatment center access can begin a navigation request now. Start a navigation request ›

Next step for Rocket teams

Rocket Pharmaceuticals medical affairs or commercial teams interested in international patient navigation partnerships can reach our BD team at [email protected].

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