Dual FDA designations support advancement of GEn1E’s novel oral precision therapy GEn-1123 for a rare devastating neuromuscular disease with critical unmet need

GEn1E Lifesciences Inc., a Phase 2 clinical-stage biotechnology company advancing AI-driven precision medicines for immunology, inflammation, and rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted both Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) to GEn-1123 for the treatment of Duchenne Muscular Dystrophy (DMD).

The dual designations mark important regulatory milestones for GEn1E and support the advancement of GEn-1123, a novel oral dual signal modulator designed to target key inflammatory and degenerative drivers of DMD progression.

DMD is a rare, severe and progressive neuromuscular disorder caused by the mutation of dystrophin gene, resulting in irreversible muscle degeneration, as well as chronic inflammation, immune-cell infiltration, myofiber death, impaired regeneration, and fibrosis — processes that contribute to progressive decline of skeletal, cardiac, and respiratory muscle function.

GEn-1123 is designed to rebalance dysregulated inflammatory signaling by modulating pro-inflammatory pathways while promoting anti-inflammatory, pro-survival, and regenerative biology. The therapy was advanced using GEn1E’s proprietary GRID Platform, which integrates patient endotyping, multi-omics, biomarker, and clinical data to drive targeted therapeutic development.

“Receiving both Orphan Drug and Rare Pediatric Disease Designations for GEn-1123 marks important milestones for GEn1E and further supports our precision medicine approach to address rare and inflammatory diseases,” said Dr. Ritu Lal, Founder and CEO of GEn1E Lifesciences. “DMD remains a devastating disease for young patients and their families. We believe GEn-1123 has the potential to become an oral small-molecule differentiated therapy that addresses key drivers of disease progression in patients who remain underserved by current treatment options.”

The FDA grants Orphan Drug Designation to therapies intended for the treatment of rare diseases or conditions affecting fewer than 200,000 people in the United States. The designation provides certain development incentives, including potential tax credits for qualified clinical trials, exemption from certain FDA fees, and the potential for seven years of market exclusivity upon regulatory approval.

The FDA grants Rare Pediatric Disease Designation to therapies intended to treat serious or life-threatening diseases that primarily affect individuals aged 18 years or younger and meet statutory rarity criteria. Upon potential approval of a qualifying application for GEn-1123, GEn1E may be eligible to receive a Priority Review Voucher (PRV), subject to applicable FDA requirements.

About GEn1E Lifesciences

GEn1E Lifesciences Inc. is a Phase 2 clinical-stage biotechnology company based in Palo Alto, California, with a laboratory in Mountain View, California, advancing AI-driven precision medicines for immunology, inflammation, and rare diseases. The company’s lead program, GEn-1124, a host-directed, small-molecule dual-signal modulator, advanced from discovery to Phase 2 in ~2.5 years and has received U.S. FDA Fast Track designation. GEn1E is also advancing a pipeline of four novel precision therapies targeting immunological, inflammatory, and rare diseases.

At the core of GEn1E’s strategy is its proprietary “Platform-in-a-Mechanism™” model, which is applied across the entire drug development process. This platform incorporates patient-first, AI-powered endotyping models to better define disease subpopulations, guide therapeutic design, and enhance the probability of technical and regulatory success. By integrating advanced computational biology with translational science, GEn1E is able to pursue programs with strong mechanistic rationale and clinical, omics and biomarker-driven development pathways.

Learn more about how the company is advancing a new generation of precision medicines for patients at www.gen1e.com.

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“Receiving both Orphan Drug & Rare Pediatric Disease Designations for GEn-1123 marks important milestones for GEn1E and further supports our precision medicine approach to address rare and inflammatory diseases,” said Dr. Ritu Lal, Founder and CEO of GEn1E