Clinical Research Focus. 48th Edition

FDA Approves First Gene Therapy for Wiskott–Aldrich Syndrome

The FDA has approved Waskyra, the first cell-based gene therapy for Wiskott–Aldrich syndrome, marking a major milestone for patients with this rare, life-threatening genetic disease. The decision reflects regulatory flexibility for rare conditions and is supported by clinical trial and expanded access data showing significant reductions in infections and bleeding, offering a new treatment option where none previously existed.

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12 Stories of Courage and Cure: A Holiday Message from Cromos Pharma

As we step into a new year, we’re pleased to share “12 Stories of Courage and Cure” — a short video that reflects on defining moments in medical research where courage, perseverance, and c       ompassion came together to move science forward and change lives. These stories highlight not only scientific breakthroughs, but the people behind them — patients, researchers, and physicians whose determination and humanity continue to shape progress in medicine. We hope this video serves as a moment of reflection and inspiration as we look ahead to another year of discovery, responsibility, and meaningful impact.

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Cancer Vaccines 2025, Part II: The Rise of DNA Cancer Vaccines

Cancer vaccines are entering a new era — and it’s no longer just about mRNA. A powerful second wave is gaining momentum: DNA cancer vaccines. They’re stable, fast to produce, easy to store, and now moving from early experiments into real human trials across melanoma, lung, ovarian, prostate, and virus-associated cancers.

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EMA Recommends Conditional Approval of Anktiva for BCG-Unresponsive Bladder Cancer

The EMA has recommended granting a conditional marketing authorisation in the EU for Anktiva (nogapendekin alfa inbakicept) to treat adults with high-risk, BCG-unresponsive non-muscle invasive bladder cancer. Supported by clinical trial data showing durable complete responses in a majority of patients, the recommendation addresses a major unmet need for those facing limited options beyond bladder removal surgery and reflects the EU’s regulatory pathway for early access to promising therapies.

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The Most Transformative FDA Approvals of 2025: Therapies Redefining Clinical Research and Future Trial Strategy

FDA approvals offer more than scientific milestones — they reveal how clinical trials are evolving. The 2025 highlighted a clear shift toward ultra-precise patient populations, biomarker-driven development, and accelerated regulatory pathways. From oncology and hematology to RNA therapeutics and infectious disease prevention, these approvals point to where clinical trial strategy is headed next.

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NIH Trial Shows 94% Survival With Expanded Cord Blood Transplant in Severe Aplastic Anemia

A clinical trial led by the NIH has demonstrated a 94% survival rate in patients with severe aplastic anemia treated with an expanded umbilical cord blood transplant using omidubicel. The approach enabled rapid and durable blood cell recovery in high-risk patients lacking suitable donors and has now received approval from the FDA, marking a major advance for a life-threatening condition with limited treatment options.

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Kyverna’s CAR-T (miv-cel) Delivers Registrational Win in Stiff Person Syndrome, Sets Up FDA Filing in 1H 2026

Kyverna Therapeutics reported positive topline data from its registrational KYSA-8 Phase 2 trial of miv-cel in stiff person syndrome, showing significant clinical improvement after a single dose and a favorable safety profile. The company plans to submit a BLA to the FDA in the first half of 2026, potentially making miv-cel the first approved CAR-T therapy for an autoimmune disease.

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Immunome’s Varegacestat Delivers Phase 3 Win in Desmoid Tumors, NDA Planned for 2026

Immunome announced positive topline results from its pivotal Phase 3 RINGSIDE trial, with varegacestat reducing the risk of disease progression or death by 84% versus placebo and achieving a 56% objective response rate in desmoid tumors. The oral therapy was generally well tolerated, and the company plans to submit a New Drug Application to the FDA in Q2 2026, positioning varegacestat as a potential new standard of care for this rare disease.

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