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What this is: A summary created by Elaaj Bio on the current status, timeline and other specific questions related to Elaaj Bio’s CDKL5 Gene Therapy Program. This FAQ was created, in part, to address questions from:
February 28, 2026: Webinar titled Update on ELJ-101: A Gene Therapy for CDD (the recording can be viewed here)
June 20, 2026: ELJ-101: AAV Gene Therapy for the Treatment of CDKL5 Deficiency Disorder, presented at the 7th CDKL5 Family Educational Conference hosted by the International Foundation for CDKL5 Research (the recording can be viewed here)
What this is not: Medical advice or a promise of trial timing, eligibility, or access. Details may change.
Where official details will appear: Program updates, when available, will be provided through official Elaaj Bio communication updates. Details may change.
Gene therapy programs typically move through stages or phases, such as from proof of concept in relevant models to IND enabling studies (safety, biodistribution, dosing) to manufacturing scale up to regulatory submissions and feedback and first in human study start-up. Because these steps can run in parallel (and sometimes need repeats), timing can change.
The first clinical trial for ELJ-101 is currently targeting enrollment in Spring/early Summer 2027.
By the end of 2026. Program updates, when available, will be provided through official Elaaj Bio communication updates. Details may change.
Eligibility is determined by the protocol and may include factors such as age range, confirmed diagnosis/genotype, clinical stability, seizure history, and safety considerations. Eligibility criteria are set in a study protocol and are usually finalized as the trial is prepared for registration and site activation.
We can’t confirm individual eligibility in advance. For the first clinical trial, we plan to enroll girls ≥ 4 years of age. There are currently no exclusions based on mutation type, including those with deletions or mosaicism. Additional definitive criteria are currently under consideration. The intention is to expand access to include all patients. Details may change. Once criteria are posted, we encourage families to discuss them with their clinician and the study team.
For the first clinical trial, we plan to enroll girls ≥ 4 years of age. No maximum age limits have been set as yet. Pending outcomes of the first study, we anticipate future enrollment in separate trials to include (1) girls ≥ 6 months of age, and (2) boys. Elaaj Bio's goal is for the drug to be approved for use in patients of all ages
We can’t confirm individual eligibility in advance. As of June 2026, there is no cap for the number and severity of seizures. For the overall severity of the condition, there will be no cap except that individuals who are ventilator dependent will be excluded. Details may change. Once criteria are posted, we encourage families to discuss them with their clinician and the study team.
In general, clinical trials commonly require investigational medications to be discontinued for a pre-defined period of time prior to starting a new treatment. Elaaj Bio’s study is designed to be as inclusive as possible and is anticipated to apply similar criteria. However, individuals with prior gene and cell therapies would be excluded. Details may change. Once criteria are posted, we encourage families to discuss them with their clinician and the study team.
Vagus nerve stimulation (VNS) is not an exclusion criterion. Major surgery, for scoliosis as one example, is not an exclusion criterion. Enrollment is possible after major surgery after a defined time period has passed. Details may change. Once criteria are posted, we encourage families to discuss them with their clinician and the study team.
Site selection may balance factors including clinical expertise with the condition, ability to perform required procedures and monitoring, infrastructure, and regulatory readiness. Enrollment happens through confirmed study sites once a trial is open. Participation rules, either within or outside the U.S., depend on where sites are located, local regulations, and the protocol.
Clinical trial sites have not yet been finalized. For the first study, we will focus on evaluating sites based on (1) CDKL5 Centers of Excellence Principal Investigators and (2) experience with gene therapy. More details on trial sites are expected to be available later in 2026
We cannot enroll participants until a study is open and sites are activated. We estimate that it will take 1-2 years to complete enrollment.
After the first study, the goal is to make the trial globally accessible, depending on where clinical trial sites are opened. Travel may be required but we cannot confirm details until sites are finalized. More details will be available later in 2026.
In general, study design addresses factors such as dose levels and endpoints. A study requires establishing rules, safety review checkpoints and careful monitoring. As of June 2026, we offer general information to address the following questions for the first study. Details may change.
We anticipate a target enrollment of up to twelve (12) individuals.
We anticipate intracisternal magna (ICM) injection will be used as the route to deliver the gene therapy.
In general, study protocols define dose levels details. The dose levels under consideration are within range of efficacy, safety and tolerability. More details will be available later in 2026.
Outcome measures vary by phase. At this time, we are considering primary and secondary outcomes, and exploratory measures. Safety is a primary objective for the first trial. Outcomes measures under consideration include seizure frequency and severity, communication, motor skills, adaptive function and behavior, quality-of-life, and sleep. More details will be available later in 2026.
All AAV gene therapies must be evaluated for safety concerns including immune responses to the vector/capsid, inflammation, lab abnormalities (e.g., liver enzymes), and procedure-related risks depending on route of administration.
Yes, there are potential risks. In other gene therapy trials (for other diseases), serious adverse events have occurred. This is why the first trial has safety as its primary objective. The non-human primate (monkey) and mouse data showed no serious problems, but neither primates nor mice are patients with CDD.
We cannot advise individual decisions online. If a study is available, the consent process and study clinicians will review risks, alternatives, and individual circumstances.
Long-term follow-up plans are typically described in the study protocol. Most gene therapy trials for other diseases include long-term follow-up to monitor durability and safety. More details will be available later in 2026.
We cannot call this treatment a “cure.” It has never been given to a person with CDD so we do not yet know how much benefit patients will receive. The mouse data are encouraging, but mice are not people. We can say that the data generated in preclinical studies give us hope that this gene therapy could provide meaningful improvement to patients.
We do not know. Researchers hope so, based on the fact that neurons in people with CDD are still alive (the disease is not degenerative). This will be one of the key questions the clinical trials aim to answer.
It’s too early to state pricing. If the therapy is approved, we will share access/support resources as they become available.
Your treating clinician is the best person to guide care decisions. This FAQ does not replace medical advice. We encourage families to consult their care team for individualized guidance.