The CANDID study evaluates neurocognitive, functional, and quality of life scales to optimize endpoint selection for CDKL5 deficiency disorder trials.

The first peer-reviewed results from the Loulou Foundation-led CANDID study are now published in Epilepsia.

CANDID is a 3-year, non-interventional study that seeks to identify motor, cognitive, and behavioral measures that may inform future trial design for CDKL5 Deficiency Disorder (CDD).

The baseline study publication includes 112 enrolled participants, with 111 included in the baseline analyses. Participants had a mean age of 8.3 years, 93% were female, and median seizure onset was 1.5 months.

Among the measures evaluated, the authors concluded that Vineland-3 and Bayley-4 were feasible for use in CDD and scores could be derived in most participants. The Gross Motor Function Measure-88 (GMFM-88), by contrast, showed floor effects and operational limitations that make it less suitable for CDD trials.

The paper also provides a snapshot of study progress: 90% of participants had reached the 2-year visit, and the first participant had completed the study.

“The results from this paper mark an important milestone for clinical trial readiness in the CDD field”, notes Dr. Liogier d’Ardhuy, Chief of Translational Science of the Loulou Foundation and the CANDID study lead. “We now have an early view of which non-seizure clinical measures may be most practical, informative, and suitable in CDD trials.”

Additional longitudinal data are expected as follow-up continues.

Elaaj Bio participates in CANDID as one of seven biopharmaceutical partners in the study’s pre-competitive consortium.

Learn more about CANDID, an endpoint-enabling study designed to support future clinical trials in CDD at candidstudycdkl5.info and in the open access publication 10.1002/epi.70095.

Elaaj Bio a biotechnology company focused on development of transformative treatments for CDKL5 Deficiency Disorder (CDD) — today announced a collaborative agreement with Gemma Biotherapeutics (“GEMMABio”), a leading global genetic medicines company. The companies will work together to advance ELJ-101, an adeno-associated virus (AAV)-based CDKL5 gene therapy, to the clinic.

Elaaj Bio is a wholly owned subsidiary of the Loulou Foundation, a private UK-based foundation dedicated to the development of therapeutics for CDD. The news of this agreement was announced at the CDKL5 Forum (October 27-28, 2025), the Loulou Foundation’s annual international conference on CDD research and drug development, which is held in Boston each fall.

ELJ-101 is a clinical candidate designed to provide functional expression of CDKL5 in the brain. It is administered directly to the central nervous system (CNS) by intra-cisterna magna injection and deploys the hu68 AAV capsid. It was developed in the academic laboratory of James M. Wilson, MD, PhD with support from the Loulou Foundation and Elaaj Bio, which funded the nonclinical studies. The program is licensed to Elaaj Bio, and the company will support its progression to clinical trials and beyond.

"We are thrilled to have GEMMABio’s support as we advance ELJ-101 to the clinic," said Russ Addis, Chief of Pipeline Strategy and Head of Genetic Medicine at the Loulou Foundation. “This partnership boosts our efforts toward our primary goal of achieving transformative therapeutics for CDD patients and families.” Dr. Addis will present an update on ELJ-101 development at the CDKL5 Forum today.

Under the terms of the collaboration, GEMMABio will provide advice and access to additional datasets to complement and support Elaaj Bio’s investigational new drug (IND) submission to the FDA for ELJ-101. GEMMABio will also give recommendations on the clinical protocol and clinical operations for the ELJ-101 program. Elaaj Bio will share regulatory feedback and clinical data to support and inform additional GEMMABio programs using the same AAV platform technologies.

"It is gratifying to continue our longtime collaboration with Elaaj Bio and the Loulou Foundation as ELJ-101 advances to IND submission," said James M. Wilson, MD, PhD, who serves as President and CEO of GEMMABio and whose research team developed ELJ-101. "Transparency and knowledge sharing are essential to moving these platform-based, transformative therapies ahead as quickly as possible to patients in need. This collaboration will benefit not only the CDD community, but also other rare disease communities whose future therapeutic options may rely on similar CNS platform technology."

CDKL5 Deficiency Disorder is among the most prevalent monogenic neurodevelopmental and epileptic disorders, with an incidence rate of approximately 1 in 41,000 live births. Infantile spasms present shortly after birth, and progress to largely intractable epilepsy, along with neurodevelopmental delay impacting multiple domains. Current anti-seizure treatments are only partially effective and there is no treatment for the neurodevelopmental delay.