The gene therapy landscape continues to evolve rapidly, with several companies achieving key milestones in early 2025. These advancements span a range of therapeutic areas, from rare genetic disorders to more common conditions, highlighting the increasing potential of gene therapy to address unmet medical needs.
Regulatory Milestones
United Therapeutics' UKidney, an investigational gene-edited porcine kidney product intended to treat end-stage renal disease (ESRD) via xenotransplant, has received clearance of an investigational new drug (IND) application from the FDA. This marks a significant step forward in the field of xenotransplantation, offering a potential solution for patients awaiting kidney transplants.
Arbor Biotechnologies’ ABO-101, a lipid nanoparticle (LNP)-delivered CRISPR-based gene editing therapy, has also received IND clearance from the FDA, enabling a phase 1/2 study in primary hyperoxaluria type 1 (PH1).
Clinical Trial Updates
uniQure has been given the green light by an independent data monitoring committee (IDMC) to begin enrolling patients in the second dose cohort of the phase 1/2 EPISOD1 clinical trial (NCT06100276). This trial is evaluating AMT-162, an investigational adeno-associated virus (AAV) vector-based gene therapy, for the treatment of amyotrophic lateral sclerosis caused by mutations in superoxide dismutase 1 (SOD1-ALS).
Sarepta Therapeutics’ delandistrogene moxeparvovec-rokl (marketed as Elevidys), a marketed adeno-associated virus (AAV) vector-based gene therapy for patients with Duchenne muscular dystrophy (DMD), has shown sustained benefit in patients who are ambulatory who were treated in the phase 3 EMBARK clinical trial (NCT05096221), according to new results from part 2 of the study.
Spur Therapeutics’ (formerly known as Freeline Therapeutics) FLT201, an adeno-associated virus (AAV) vector-based gene therapy, has continued to show safety and efficacy in data from the phase 1/2 GALILEO-1 clinical trial (NCT05324943) evaluating the therapy for the treatment of Gaucher disease.
CAR-T Therapy Development
Adicet Bio’s ADI-100, its investigational allogeneic chimeric antigen receptor (CAR)-engineered gamma delta T-cell therapy being evaluated for the treatment of various autoimmune diseases, has received FDA fast track designation for refractory systemic lupus erythematosus (SLE) with extrarenal involvement. The therapy is currently being evaluated in a phase 1 clinical trial (NCT06375993) for patients with SLE, systemic sclerosis, antineutrophil cytoplasmic autoantibody associated vasculitis, idiopathic inflammatory myopathy, and stiff person syndrome.
Bristol Myers Squibb (BMS)’s Type II variation application for lisocabtagene maraleucel (liso-cel, marketed as Breyanzi), an autologous CD19-directed CAR T-cell therapy, has received a recommendation for approval in relapsed/refractory (r/r) follicular lymphoma (FL) from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP).
Other Notable Updates
RESTEM’s Restem-L, an investigational umbilical cord outer lining stem cells (ULSCs) product, has been granted fast track designation by the FDA for idiopathic inflammatory myopathy (IIM, also known as polymyositis and dermatomyositis, PM/DM).
ViGeneron has received rare pediatric disease designation (RPDD) from the FDA for VG901, a gene therapy intended to treat CNGA1-associated retinitis pigmentosa.
These developments underscore the ongoing momentum in the cell and gene therapy field, with regulatory approvals, clinical trial advancements, and novel therapeutic approaches paving the way for improved patient outcomes.
