HCP supportEnroll patients

Support that’s customized because every patient has distinct needs

As a healthcare professional (HCP), you know better than anyone that each patient has medical, information, and support needs that are unique. That’s why we assign each patient a dedicated support team to help ensure the best possible experience. While the program is optional, we encourage patients to take advantage of this service.

We can help your patients by:

  • Providing a caring support team from confirmed diagnosis through postsurgery follow-up
  • Ensuring a single point of contact to help navigate insurance coverage and connect your patients with financial assistance resources as needed
  • Helping to coordinate visits to the ocular gene therapy treatment center of your chosing
  • Answering any nonmedical questions along the way

How to enroll patients

First, determine if LUXTURNA® (voretigene neparvovec-rzyl) is right for your patient. The prescription gene therapy is for patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. Genetic testing is the only way to confirm this gene mutation. Your patients must also have enough viable retinal cells as determined by the treating physician(s), in order to be eligible for treatment. If your patient has not had a genetic test, learn more here.


1Mutations in both copies of the RPE65 gene

Your patient has confirmed biallelic RPE65 mutation-associated retinal dystrophy and viable retinal cells as determined by you, their treating physician. Have they been connected with an ocular gene therapy treatment center?


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2Fill out enrollment form

Submitting this form allows Spark® Therapeutics to enroll your patient in the program and investigate their insurance coverage.

Download enrollment form

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3Schedule consultation

Your patient’s support team will help connect them with an ocular gene therapy treatment center to schedule their consultation to determine eligibility for treatment.

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4Completion of a Statement of Medical Necessity (SMN)

Once a patient has met all eligibility requirements for treatment with LUXTURNA, an ocular gene therapy treatment center specialist fills out a Statement of Medical Necessity (SMN) form for your patient. An SMN confirms eligibility to receive treatment and Spark Generation Patient Services will then begin an insurance benefits investigation.

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5Your patient’s journey

Once an enrollment form or a SMN has been received along with the signed and completed patient consent, your patient services team will communicate the next steps with you and your patient.

Download enrollment formDownload SMN form

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Participation in Spark Therapeutics Generation Patient Services® is voluntary. Your patient may choose to participate in all, some, or none of the services offered. Participating or deciding not to participate in these services will have no effect on your patient’s ability to get treatment or the nature of your patient’s treatment or care. Spark Therapeutics Generation Patient Services does not provide medical advice.

Patient Mentor Program: support from someone who’s been there

If your patient is considering treatment with LUXTURNA, it may be helpful to hear from someone who has been there. Spark Therapeutics Generation Patient Services can connect your patient with a patient (or caregiver of a patient) who was previously treated with LUXTURNA.

Mentors are happy to share their stories, but they cannot give medical advice. If your patients or their caregivers have medical questions about inherited retinal disease or the risks and benefits of LUXTURNA, they should talk to you.

Eligibility requirements

To be connected with a mentor, a patient must be a US resident 18 years of age or older and be a patient, or a parent or legal guardian of a patient, who has been diagnosed with a biallelic RPE65 mutation–associated retinal dystrophy. The patient/guardian must sign the consent form.

To learn more about how to get started with a mentor, download this brochure and call Generation Patient Services toll-free at 1-833-SPARK-PS (1-833-772-7577).

Download brochure

Photographs are not of actual patients or caregivers


Warnings and Precautions

  • Endophthalmitis may occur following any intraocular surgical procedure or injection. Use proper aseptic injection technique when administering LUXTURNA, and monitor for and advise patients to report any signs or symptoms of infection or inflammation to permit early treatment of any infection.
  • Permanent decline in visual acuity may occur following subretinal injection of LUXTURNA. Monitor patients for visual disturbances.
  • Retinal abnormalities may occur during or following the subretinal injection of LUXTURNA, including macular holes, foveal thinning, loss of foveal function, foveal dehiscence, chorioretinal atrophy, and retinal hemorrhage. Monitor and manage these retinal abnormalities appropriately. Do not administer LUXTURNA in the immediate vicinity of the fovea. Retinal abnormalities may occur during or following vitrectomy, including retinal tears, epiretinal membrane, or retinal detachment. Monitor patients during and following the injection to permit early treatment of these retinal abnormalities. Advise patients to report any signs or symptoms of retinal tears and/or detachment without delay.
  • Increased intraocular pressure may occur after subretinal injection of LUXTURNA. Monitor and manage intraocular pressure appropriately.
  • Expansion of intraocular air bubbles Instruct patients to avoid air travel, travel to high elevations or scuba diving until the air bubble formed following administration of LUXTURNA has completely dissipated from the eye. It may take one week or more following injection for the air bubble to dissipate. A change in altitude while the air bubble is still present can result in irreversible vision loss. Verify the dissipation of the air bubble through ophthalmic examination.
  • Cataract Subretinal injection of LUXTURNA, especially vitrectomy surgery, is associated with an increased incidence of cataract development and/or progression.

Adverse Reactions

  • In clinical studies, ocular adverse reactions occurred in 66% of study participants (57% of injected eyes), and may have been related to LUXTURNA, the subretinal injection procedure, the concomitant use of corticosteroids, or a combination of these procedures and products.
  • The most common adverse reactions (incidence ≥ 5% of study participants) were conjunctival hyperemia (22%), cataract (20%), increased intraocular pressure (15%), retinal tear (10%), dellen (thinning of the corneal stroma) (7%), macular hole (7%), subretinal deposits (7%), eye inflammation (5%), eye irritation (5%), eye pain (5%), and maculopathy (wrinkling on the surface of the macula) (5%).


Immune reactions and extra-ocular exposure to LUXTURNA in clinical studies were mild. No clinically significant cytotoxic T-cell response to either AAV2 or RPE65 has been observed. In clinical studies, the interval between the subretinal injections into the two eyes ranged from 7 to 14 days and 1.7 to 4.6 years. Study participants received systemic corticosteroids before and after subretinal injection of LUXTURNA to each eye, which may have decreased the potential immune reaction to either AAV2 or RPE65.

Pediatric Use

Treatment with LUXTURNA is not recommended for patients younger than 12 months of age, because the retinal cells are still undergoing cell proliferation, and LUXTURNA would potentially be diluted or lost during the cell proliferation. The safety and efficacy of LUXTURNA have been established in pediatric patients. There were no significant differences in safety between the different age subgroups.

Please see the US Full Prescribing Information for LUXTURNA.


LUXTURNA (voretigene neparvovec-rzyl) is an adeno-associated virus vector-based gene therapy indicated for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy.

Patients must have viable retinal cells as determined by the treating physicians.

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