<p>There are diseases so rare and so severe that most people have never heard of them. Leukocyte Adhesion Deficiency Type I — LAD-1 — is one. It is a life-threatening genetic immune disorder in which the body cannot make functioning immune cells that stick to blood vessel walls to fight infection. Without treatment, severe LAD-1 is <strong>near-uniformly fatal in early childhood</strong>.</p>
<p>The only curative option currently available is an allogeneic haematopoietic stem cell transplant — a bone marrow transplant from a matched donor. But finding compatible donors is difficult, transplant carries significant risk, and the procedure's success depends heavily on access to specialist care.</p>
<p>Now, a gene therapy may be about to change everything. The FDA has set a decision date of <strong>March 28, 2026</strong> for <strong>Kresladi</strong> (marnetegragene autotemcel), developed by Rocket Pharmaceuticals.</p>
<h2>100% Survival at 12 Months</h2>
<p>The clinical data behind Kresladi is striking. In a global Phase 1/2 study, every enrolled patient who received the therapy achieved <strong>100% overall survival</strong> at 12 months — and this survival was maintained throughout the full duration of follow-up, however long they were tracked. The therapy also met all primary and secondary endpoints, with patients showing substantial reductions in severe infections and improvements in skin lesions and wound healing — two hallmarks of LAD-1 disease severity.</p>
<p>Kresladi is a one-time therapy. It uses the patient's own stem cells, collected, genetically modified in a laboratory to deliver a working copy of the <strong>ITGB2 gene</strong> (which produces the missing protein), and then infused back. Because it uses the patient's own cells, there is no need to find a matched donor — and no risk of graft-versus-host disease, one of the most dangerous complications of conventional bone marrow transplants.</p>
<h2>A Long Road to This Moment</h2>
<p>The path to this approval was not without setbacks. The FDA issued a Complete Response Letter in June 2024 — not over safety or efficacy concerns, but over Chemistry, Manufacturing, and Controls requirements (the rigorous quality standards applied to gene therapy production). Rocket Pharmaceuticals addressed those concerns, and the FDA accepted the resubmission in October 2025, setting the March 28, 2026 review date.</p>
<p>Kresladi carries several special FDA designations: Regenerative Medicine Advanced Therapy (RMAT), Rare Paediatric Disease, and Fast Track — all markers of its potential to address a serious unmet medical need.</p>
<h2>What Approval Would Mean</h2>
<p>If the FDA grants approval, Kresladi would become the first gene therapy approved for LAD-1 in the United States. For families of children with severe LAD-1, it could represent the difference between a lifetime of bone marrow transplant searches and their own cells — corrected once, giving their child a functional immune system.</p>
<p>The rare disease community is watching the March 28 decision date closely.</p>
<p><em>Sources: Rocket Pharmaceuticals · FDA · FierceBiotech · CGTLive · Cell & Gene Therapy Live · PharmaVoice (March 2026)</em></p>
