This is an exciting time for the Dravet syndrome community. The first potentially disease-modifying treatment targeting the genetic cause of Dravet syndrome has completed initial human studies and is now advancing to a pivotal global Phase 3 trial. If the study confirms that this therapy is safe and effective, the next step will be seeking regulatory approval. While approval is likely still years away, there is much to consider—both about the current trial and the future availability of this treatment.
Today’s blog post provides answers to Frequently Asked Questions (FAQ) about this experimental therapy, zorevunersen (STK-001), including how it works, details about the upcoming Phase 3 study, and considerations regarding potential approval and access.
This information was compiled by DSF’s Scientific Director, Veronica Hood, PhD, based on publicly available data, as well as insights from professional and advocacy events. While DSF maintains a relationship with the study sponsor, Stoke Therapeutics, and frequently seeks information from them, Stoke Therapeutics did not contribute directly to these answers. Since clinical studies are complex and information evolves, we have made every effort to provide accurate details, but updates may emerge that affect the content in this post.
Background on Zorevunersen (STK-001)
What is zorevunersen?
Zorevunersen, previously called STK-001, is an experimental therapy being developed to address the underlying genetic cause of Dravet syndrome.
The majority of cases of Dravet syndrome are caused by mutations in one copy of the SCN1A gene. The SCN1A gene codes for a sodium channel, called Nav1.1, that is used by a specific set of cells in the brain. Mutations in SCN1A that cause Dravet syndrome are loss-of-function causing haploinsufficiency; this means half of the Nav1.1 sodium channels are lost or reduced in function, which leads to the seizures and symptoms of Dravet syndrome. Zorevunersen works to restore levels of the healthy Nav1.1 sodium channel by increasing expression from the healthy copy of the SCN1A gene.
Zorevunersen is part of a class of therapies called antisense oligonucleotides, or ASOs. ASOs are short sequences of genetic material (in this case, RNA) that bind to specific, targeted sequences of RNA in patient cells. Zorevunersen binds to a section of RNA that allows the SCN1A gene to be more productive, increasing the levels of Nav1.1. This therapy is not permanent and will not make any changes to an individual’s existing genetic material. It will be administered via intrathecal injection (like a lumbar puncture) and dosing will likely be required every four months.
What research led to the development of zorevunersen for Dravet syndrome?
The therapeutic approach for zorevunersen was first tested in cell lines after the discovery that SCN1A was a gene that could be targeted with this approach. Studies in mouse models of Dravet syndrome provided proof-of-concept that zorevunersen could treat the symptoms of Dravet syndrome, reducing spontaneous seizures, providing protection from heat-induced seizures, preventing mortality, and restoring neuronal signaling when mice were treated at early ages. Mice treated at later points after symptom onset, there were also improvements, but the therapy seemed to take longer to be effective. Testing in non-human primates provided evidence that zorevunersen could reach the areas of the brain where the treatment needs to be delivered, and that it appeared safe in an animal model with similar physiology to humans.
These studies have been published on the work leading up to human studies of zorevunersen:
- Lim KH, Han Z, Jeon HY, Kach J, Jing E, Weyn-Vanhentenryck S, et al. Antisense oligonucleotide modulation of non-productive alternative splicing upregulates gene expression. Nat Commun 2020;11:3501. https://doi.org/10.1038/s41467-020-17093-9.
- Han Z, Chen C, Christiansen A, Ji S, Lin Q, Anumonwo C, et al. Antisense oligonucleotides increase Scn1a expression and reduce seizures and SUDEP incidence in a mouse model of Dravet syndrome. Sci Transl Med 2020;12:eaaz6100. https://doi.org/10.1126/scitranslmed.aaz6100.
- Wengert ER, Wagley PK, Strohm SM, Reza N, Wenker IC, Gaykema RP, et al. Targeted Augmentation of Nuclear Gene Output (TANGO) of SCN1A Rescues Parvalbumin Interneuron Excitability and Reduces Seizures in a Mouse Model of Dravet Syndrome. Brain Res 2021;1775:147743. https://doi.org/10.1016/j.brainres.2021.147743.
- Yuan Y, Lopez-Santiago L, Denomme N, Chen C, O’Malley HA, Hodges SL, et al. ASO restores excitability, GABA signalling and sodium current density in a model of Dravet syndrome. Brain 2023:awad349. https://doi.org/10.1093/brain/awad349. **This study used a molecule very similar to zorevunersen but not the original therapy
What studies are completed for zorevunersen in humans?
Data has been collected from four studies in the US and UK involving 81 patients with Dravet syndrome who have mutations in the SCN1A gene receiving at least one dose of zorevunersen (at the time, called STK-001) ranging from 10mg to 70mg.
US Studies:
- MONARCH was a Phase 1/2a study in the US involving children and adolescents ages 2 to 18 that assessed safety, tolerability, and pharmacokinetics of zorevunersen. The study also assessed the efficacy of zorevunersen as an adjunctive anti-seizure treatment.
- SWALLOWTAIL is an ongoing, Open-Label Extension study in the US where participants that completed the MONARCH study are eligible to continue treatment with zorevunersen while long-term safety and tolerability of repeated doses is evaluated, as well as impacts on seizure frequency, behavior, cognition, and quality of life.
UK Studies:
- ADMIRAL was a Phase 1/2a study in the UK involving children and adolescents ages 2 to <18 that assessed safety, tolerability, and pharmacokinetics of zorevunersen The study also assessed the efficacy of zorevunersen as an adjunctive anti-seizure treatment, as well as overall clinical status and quality of life.
- LONGWING is an ongoing, Open-Label Extension study in the UK where participants that completed the ADMIRAL study are eligible to continue treatment with zorevunersen while long-term safety and tolerability of repeated doses is evaluated, as well as impacts on seizure frequency, behavior, cognition, and quality of life.
What is known about the effectiveness of zorevunersen in Dravet syndrome?
Reductions in seizure frequency have been reported throughout the length of these trials across all ages and dose ranges. The most recent data update demonstrated that initial doses of 70mg led to the most substantial reductions in seizure frequency that were sustained at 6 months after the last dose of zorevunersen. Additionally, these reductions in seizures were maintained in the Open-Label Extension studies where patients continued to receive 30 to 45mg doses every 4 months.
Continued treatment with zorevunersen in the Open-Label Extension studies has led to improvements in measures of behavior and cognition such as receptive and expressive communication, personal skills, interpersonal relationships, and fine motor skills. Assessments by clinicians and caregivers additionally indicated improvements in the overall condition of the patient. These improvements suggest that zorevunersen could be the first therapy to be truly disease-modifying, significantly reducing seizure frequency as well as improving other symptoms of Dravet syndrome.
What do we know about the safety of zorevunersen in Dravet syndrome?
81 patients were treated with zorevunersen in the Phase 1/2a studies. 74 patients transitioned to the Open-Label Extension (OLE) studies, and as of June 2024, 61 patients remained in the study. Some participants have now received doses for over three years and over 600 doses of zorevunersen have been administered.
30% of patients have experienced a treatment-emergent adverse event (TEAE) that was related to the study drug. The most common were CSF protein elevations and procedural vomiting. Procedural vomiting is common following lumbar punctures and was not unexpected as a reported symptom related to the administration of the therapy. CSF protein elevations have been common, with a greater number reported from the Open-Label Extension studies, with 70% of patients having at least one laboratory report of elevated CSF protein (>50 mg/dL). Notably, no clinical symptoms have emerged correlating with these laboratory reports of elevated CSF protein. One patient did discontinue treatment due to the elevated CSF protein laboratory result, and at the 6-month follow-up, the levels had returned to normal. Elevated CSF protein levels have been observed with several types of ASO therapies and it is not fully understood.
22% of patients have experienced a treatment emergent serious adverse event (TESAE). TESAEs can include hospitalizations for any reason, and are not always related to the treatment. All but 1 event in these trials has been found to be unrelated to the study drug STK-001. As previously reported by Stoke Therapeutics, there was one patient who received multiple doses of 70mg STK-001 in the ADMIRAL study and experienced Suspected Unexpected Serious Adverse Reactions (SUSARs) that the study clinician attributed to STK-001.
Phase 3 EMPEROR Study for Zorevunersen
What is the next phase of study for zorevunersen?
Stoke Therapeutics has received regulatory approval to begin a Phase 3 study for zorevunersen in the United States (FDA), Europe (EMA), and Japan (PMDA). The Phase 3 study will be called the EMPEROR Study. The study will evaluate the effectiveness and safety of zorevunersen in patients with Dravet syndrome caused by loss-of-function mutations in SCN1A. This is a pivotal, global study that is intended to gather the evidence required to seek approval (if it is effective) from regulators.
EMPEROR is a double-blind, sham-controlled study that lasts 52 weeks following an 8-week baseline assessment period. Participants will be divided equally between treatment and sham control groups (50% in each). Participants and the clinicians following them in the study will be “blinded” to the treatment groups, meaning no one will know who is in which group so that it does not impact their assessments of how the patient is doing while in the study. Participants will receive four procedures throughout the 52-week period. The experimental group will receive two doses of zorevunersen at 70mg (Day 1 and Week 8) and two doses at 45mg (Week 24 and Week 40). Zorevunersen is delivered intrathecally, using a procedure similar to a lumbar puncture. Participants assigned to the sham control group will follow the same procedure schedule, but will not receive a dose of the medication following the lumbar puncture. After week 52, eligible participants (regardless of sham control or treatment group) will have the option to enter the Open-Label Extension study where participants will receive up to 45mg doses of zorevunersen every 4 months.
When will the Phase 3 EMPEROR Study begin?
Regulatory approval has been given for Phase 3 to begin. Currently, the sponsor (Stoke Therapeutics) is working to activate individual study sites to begin recruitment. It is anticipated that sites in the United States will become active first, likely by mid 2025, given the involvement of many sites in the previous Phase1/2a study. The remaining study sites will continue to be activated into 2026.
Where will study sites be located for the Phase 3 study?
The Phase 3 EMPEROR Study will have approximately 60 study sites in the United States, United Kingdom, Europe, and Japan.
It is anticipated that many of the same study sites from the Phase1/2 studies in the United States will be used again for the Phase 3 study. It is likely that sites in the United States that took part in the previous study will begin opening first, projected by summer 2025, with a roll-out to international sites in Europe and Japan into 2026.
Information about study site locations will be communicated as it is confirmed, and sites like clinicaltrials.gov will be updated accordingly with study site location information.
What is a sham procedure and why is it required?
A sham control procedure in the EMPEROR study means that the patient will get the same procedure, a lumbar puncture, that is given for the experimental medication (zorevunersen), but they will not receive the actual medication. Participants will be divided equally, at random, between treatment and sham groups (50% in each). Participants and the clinicians following them in the study will be “blinded” to the treatment groups, meaning no one will know who is in which group so that it does not impact their assessments of how the patient is doing while in the study. Participants will receive four procedures throughout the 52-week period (on Day 1, Week 8, Week 24, and Week 40). The study groups will not be revealed, or ‘unblinded,’ to the study team collecting and analyzing the data until the final patient has completed the 52 week study.
A sham control arm allows for the clearest assessment of how safe and effective zorevunersen is for the treatment of Dravet syndrome. A control group can reduce the impact of unintentional bias, which is particularly relevant when some of the assessments in a study are more subjective (like the impression of how the patient’s symptoms are improving by the caregiver and clinician). Importantly, the sham control arm can also help to understand the potential effects zorevunersen has on symptoms that are independent from the procedure. All of the regulatory agencies (FDA, EMA, and PMDA) were all aligned in the requirement for a control arm in these studies.
It is evident that the possibility of being in the sham control arm may place a significant burden on participants and their families during this long study. Stoke Therapeutics listened closely to feedback from advocates on their trial design plans. Ultimately, this trial design was required by all regulators in order to feel confident about the data determining if zorevunersen is safe and effective. DSF continues to advocate for the patient voice to be included in the regulatory process for drug development and to seek research mechanisms that may reduce the future requirements for the numbers of patients that need to be in a control arm of a study. However, we also recognize the importance of studies to show a therapy is safe and effective before making it commercially available to the broader patient population.
How many participants will receive the sham procedure?
The study has a 1:1 ratio of patients randomly assigned to the experimental therapy versus sham procedure groups, meaning 50% of patients will be assigned to the sham group. The study aims to enroll 150 patients, meaning 75 will be randomly assigned to the sham group.
Who will know which participants are receiving the sham?
The administration team performing the lumber puncture are the only ones who will know to which group the participant has been assigned. The study doctor that makes assessments of the patient will not know which group the patient is in, nor will the patient or their caregiver/family. The study will not be unblinded until after the final patient completes the 52-week study, at which point the individuals performing the data analysis will have the groups revealed to them. Doctors and participants will not know which group they are in until the participant has completed the entire Phase 3 study.
How long will participants receive the sham procedure?
Participation in the Phase 3 study requires an 8-week baseline assessment period, followed by a 52-week study period, during which participants will receive four procedures. Those who are in the sham group will have four lumbar punctures throughout the 52-week period (on Day 1, Week 8, Week 24, and Week 40).
Will participants know if they had the sham procedure or experimental therapy (zorevunersen)?
No, participants will not be unblinded to which group they were in during the Phase 3 study until after completion. If participants enter the Open-Label Extension study, their group would be revealed as they would need to have the two higher loading doses (70mg) prior to transitioning to the maintenance doses (45mg).
Will those receiving the sham procedure eventually be able to receive zorevunersen?
Yes. Eligible participants will have the option to transition to the Open-Label Extension study where participants will receive up to 45 mg doses of zorevunersen every 4 months. If participants were in the sham group, they will receive the two loading doses prior to the lower maintenance dosing.
What is the dosing schedule for the Phase 3 and Open-Label Extension studies?
EMPEROR is a double-blind, sham-controlled study that lasts 52 weeks following an 8-week baseline assessment period. Participants will be divided equally between treatment and sham groups (50% in each). Participants will receive four procedures throughout the 52-week period. The experimental group will receive two doses of zorevunersen at 70mg (Day 1 and Week 8) and two doses at 45mg (Week 24 and Week 40). Participants assigned to the sham procedure control group will follow the same procedure schedule, but will not receive a dose of the medication. After week 52, eligible participants (regardless of sham or treatment group) will have the option to enter the Open-Label Extension (OLE) study where participants will receive up to 45mg doses of zorevunersen every 4 months.
How long is the Phase 3 study expected to last?
The current projected timeline is that the final patient will complete the study in 2027 and the pivotal data readout will be ready in the second-half of 2027 for global regulatory filings.
These timelines are all tentative and clinical study timelines often change due to unanticipated delays in different parts of the process such as study site activations, recruitment, or safety concerns. The timelines for regulatory approval after the study may also differ depending on the individual country and their regulatory body.
Where can you find more information on the Phase 3 study?
You can find the study listing at clinicaltrials.gov. The study ID is NCT06872125. The listing has basic information about the study, including participation criteria, the study plan, and a listing of study site locations. The listing is new, and updates to the site locations will be made soon as study sites are confirmed and activated.
A study website is also coming soon, and will be an additional resource for information about participation: https://www.emperorstudy.com/
Participating in the EMPEROR Study
Who is eligible to participate in the Phase 3 study?
The Phase 3 EMPEROR study will enroll patients with a confirmed diagnosis of Dravet syndrome with a documented causal, loss-of-function mutation in the SCN1A gene between the ages of 2 to 18 years of age. Patients must be on at least one stable anti-seizure medication and have tried at least two prior anti-seizure therapies (including medications, VNS, or ketogenic diet). Patients must still be experiencing breakthrough major motor seizures.
Since zorevunersen works by increasing levels of the sodium channel Nav1.1, patients with gain-of-function mutations in the SCN1A gene are not eligible to participate, nor are patients taking maintenance anti-seizure medications that act primarily as a sodium channel blocker (such as phenytoin, carbamazepine, oxcarbazepine, lamotrigine, lacosamide, rufinamide, or cenobamate). Participants also cannot be currently treated with a neuromodulation technique other than VNS.
Additional criteria may be required and will be discussed with potential participants by the study team.
You can read more under “Participation Criteria” at the listing on clinicaltrials.gov.
What ages will be included in the Phase 3 study?
The EMPEROR Study will enroll patients with Dravet syndrome that are 2 years or older up to age 17 years (must be under the age of 18 years). While the previous US-based study included patients aged 2 to 18 years, this slight adjustment to under 18 was made to meet alignment among global regulators. Patients that have entered the studies within that target age range will still be eligible to continue in the studies after reaching the age of 18 years.
Patients from the previous Phase 1/2a study that are over 18 years have continued to be able to access zorevunersen through the Open-Label Extension study.
Is there a minimum seizure threshold required to participate? Why?
The primary outcome that the EMPEROR study will be measuring to determine if zorevunersen is effective is the change in number of major motor seizures, so participants must be experiencing a baseline level of breakthrough seizures to participate so that the effect of the experimental therapy can be assessed during the study period. The number of seizures required during the baseline observation period will be communicated to the participant by the study site team.
Can participants be in other studies or on experimental medications?
No, participants cannot be enrolled in other clinical trials or taking experimental medications. If you have a question about a specific medication, these should be directed to the clinician at the study site.
Will mutations in other genes exclude a patient from participating?
Patients with mutations in other genes must be considered on a case-by-case basis by the clinician investigators at the study site, as well as by review of an external committee confirming the diagnosis. In general, mutations in genes that are related to epilepsy or other symptoms of Dravet syndrome may be exclusionary, but mutations in unrelated genes could potentially be acceptable.
How long does study participation last?
Participation in the Phase 3 study includes an 8-week baseline assessment period followed by a 52-week study period. Eligible participants may elect to join the Open-Label Extension (OLE) following completion of the Phase 3 study where they can continue to have access to zorevunersen.
How many patients will be included in the Phase 3 study?
The Phase 3 study will involve 150 patients with Dravet syndrome across approximately 60 sites worldwide, with the number of participants at each site varying. Given the strong interest from our patient community around this Phase 3 trial, it is important to remember that enrollment will be extremely limited.
Why was this number chosen?
The number of participants needed was carefully calculated to ensure that there would be sufficient data to statistically assess with confidence whether zorevunersen is reducing seizures and improving scores on developmental scales (the Vineland-3). They took into account the typical ‘placebo’ response that has been observed in other interventional studies of anti-seizure therapies for Dravet syndrome and used the natural history data collected in the BUTTERFLY observational study to understand the level of information needed for the developmental scale measurements. Because zorevunersen is still an experimental therapy, exposing the minimum number of individuals while still gathering the necessary data is the goal.
Does this number include participants from the previous studies?
No, participants in the Phase1/2a study and those that have continued into the Open-Label Extension study are not included in the target enrollment of 150 participants for the Phase 3 study.
How are participants selected across sites? How is the order determined?
In short, participants are selected by the clinicians overseeing the study at each site.
There are many moving parts and roles in the management of a study. The study sponsor, in this case Stoke Therapeutics, develops the overarching protocol for the study, which includes the enrollment criteria and number of participants that will be enrolled. However, the sponsor does not select the individual patients for the study. The clinician-lead at each study site is responsible for selecting patients that are eligible to participate. The sponsor and any clinical research organization they have hired to help manage the trial will work amongst sites with participants ready to enroll to maintain a balance in the ages of participants as well as ensuring a balanced randomization of participants into the sham control versus treatment arms.
Can other medications be adjusted while participating in the Phase 3 study?
Daily maintenance medications should remain stable during the study, particularly during the initial 28 weeks, during which time the change in seizures is being measured for the primary endpoint for the study. However, rescue medications are allowed while in the study and acute changes to medications could occur if there is a sudden increase in seizure activity. Participants will be closely monitored by the study team and safety is of primary importance.
Medication changes are allowed in the Open-Label Extension study.
Will participants continue to have access to zorevunersen after the study?
Following completion of the Phase 3 study (8 weeks of baseline assessment followed by 52 weeks of the interventional study), eligible participants will have the option to transition to the Open-Label Extension study.
Participants in the Open-Label Extension study will receive up to 45mg doses of zorevunersen every 4 months.
Only participants that have completed the earlier phase of the study (Phase 1/2a or Phase 3) are eligible to enter the Open-Label Extension study. There is no other way to access zorevunersen prior to a regulatory approval.
Is there a way to access zorevunersen outside of the study? (i.e. Compassionate Use)
Only participants that have completed the earlier phase of the study (Phase 1/2a or Phase 3) are eligible to enter the Open-Label Extension study. There is no other way to access zorevunersen prior to a regulatory approval.
While the current data from the previous Phase1/2a is promising, zorevunersen is still an experimental therapy and the goal during the Phase 3 study is to expose the minimum number of patients while gathering the clearest insight into the safety and effectiveness of zorevunersen.
Can patients with an SCN1A gain-of-function mutation participate?
Since zorevunersen acts by increasing the expression of SCN1A and as a result the production of more Nav1.1 sodium channels, this could be contraindicated for patients with gain-of-function SCN1A mutations and they are not eligible to participate in the EMPEROR Study.
Are patients eligible to participate in the study in a country where they do not currently reside and/or have citizenship?
Participation in a clinical study from another country can be quite complicated. EMPEROR is a particularly long study requiring regular follow-up and dosing. International participation in the study would be dependent on many factors, including the ability to consistently travel and the safety of the participating patient. Requests for international participation in the study would be considered on a case-by-case basis from the individual study site and the sponsor. It is almost important to consider that this is not a one-time therapy and maintaining access to the therapy after regulatory approval could pose additional challenges.
If a participant leaves the study, can they still access zorevunersen?
Participation in the Phase 3 study is the only way to gain access to zorevunersen prior to regulatory approvals. Transition to the Open-Label Extension studies is only possible upon completion of the Phase 3 study.
Future Access to Zorevunersen
Will zorevunersen be effective for adults with Dravet syndrome?
We do not currently know how effective zorevunersen may be in adults with Dravet syndrome. There are currently participants that entered the Phase1/2a studies in their late teens and now continue to receive zorevunersen in the Open-Label Extension studies as adults.
There is currently not enough data from the studies to determine if zorevunersen is more or less effective in older age groups. Mouse studies suggest that genetic interventions even at older ages may still be effective for the treatment of at least seizures and mortality in Dravet syndrome, but it is unclear how those mouse studies may translate to human patients with Dravet syndrome.
Stoke Therapeutics has expressed it is their hope for everyone who can benefit from zorevunersen to have access, but that may take time to understand who will benefit and to gather the data required for approvals.
Why are adults not included in the current trial?
Due to subtle differences in how global regulatory authorities approve therapies for different demographics, the age limit for the current study needed to be limited to those under the age of 18. Additional studies would be needed to look specifically at the effectiveness of zorevunersen in adults.
Will adults have access to zorevunersen if it is approved by regulators?
At this time it is difficult to know the specific indication that regulators will approve and there could be differences between agencies. In addition to the approval specifics, there will likely also be information that payers (insurance companies in the United States medical system) need to review that may impact how the therapy is reimbursed. It is likely that additional studies in adults with Dravet syndrome may be required.
Will there be future studies to investigate the efficacy of zorevunersen in adults?
Stoke Therapeutics has said they are looking at information related to how studies might need to be designed to investigate the efficacy and safety of zorevunersen in additional age groups. However, their current focus is on beginning the current Phase 3 EMPEROR Study.
Will zorevunersen be investigated in children with Dravet syndrome under age 2?
Stoke Therapeutics has said they are looking at information related to how studies might need to be designed to investigate the efficacy and safety of zorevunersen in additional age groups. However, their current focus is on beginning the current Phase 3 EMPEROR Study.
When will zorevunersen be approved?
It is very difficult to predict timelines regarding the study, regulatory filings, and regulatory approvals. In terms of the study, the current projected timeline is that the final patient will complete the study in 2027 and the pivotal data readout will be ready in the second-half of 2027 for global regulatory filings. We do not currently know which regulatory agencies and countries will be sought for approval following completion of the Phase 3 studies, and the timelines for each agency might be quite different to review the filing and make a decision on approval.
In the US, zorevunersen has received Breakthrough Drug Designation from the FDA. How will this speed up the study process?
FDA Breakthrough Drug Designation is intended for a drug that is developed to treat a serious condition and that has preliminary clinical evidence that indicates that the drug may demonstrate substantial improvement on a clinically significant endpoint over currently available therapies.
FDA granted Breakthrough Drug Designation for zorevunersen in December 2024. This designation allows for intensive guidance and frequent interactions with FDA throughout the remainder of the development process, the involvement of senior FDA staff in the review process, the ability to submit portions of the marketing application as they are completed (versus waiting to submit all at once), and faster priority review versus standard application timelines with the FDA.
Will zorevunersen be available to patients world-wide once it is approved?
We do not currently know which regulatory agencies and countries will be sought for approval following completion of the Phase 3 studies, and the timelines for each agency might be quite different to review the filing and make a decision on approval.
How will zorevunersen be accessed once it is approved?
Access to zorevunersen will likely look quite different depending on the location of the patient. In the United States, there may be key differences depending on geographical location and the type of medical insurance coverage the patient has. Genetic-based therapies have historically been quite expensive given the advantages they may offer over traditional medical treatments for select diseases and the expensive process of developing and administering them. While it remains to be seen how the process and costs may play out for zorevunersen, DSF is working now on understanding the challenges that may emerge following an approval for a therapeutic like this so we can be ready to help ensure the most number of patients that may benefit are able to access it.
Stoke Therapeutics recently announced a partnership with Biogen. What does this mean for the development of zorevunersen?
Stoke recently announced a partnership with Biogen to work on the development and commercialization of zorevunersen. This partnership is expected to lend international expertise and financial stability as they work to move zorevunersen and the Phase 3 studies forward as quickly as possible and throughout the commercialization process after regulatory approvals.
