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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 the Webinar titled Update on ELJ-101: A Gene Therapy for CDD (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 early 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. There are currently no exclusions based on mutation type. The intention is to include all patients, including those with deletions. However, the definitive criteria for the first clinical trial have not yet been established. Once criteria are posted, we encourage families to discuss them with their clinician and the study team.
No age limits have been set as yet. Elaaj Bio's goal is for the drug to be approved for use in patients of all ages.
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.
The sites of the clinical trial have not yet been selected. Elaaj Bio is currently evaluating potential trial sites in multiple countries. 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.
Clinical sites have not yet been chosen. The goal is to make the trial globally accessible, but this will depend on where the 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. At this time, we cannot confirm details, however, we offer general information to address the following questions.
In general, study protocols define dose levels details. 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. Other outcomes that will be measured include communication, motor function, and developmental endpoints. 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.