Lilly moves to acquire Kelonia for in vivo CAR-T platform

Eli Lilly has announced plans to acquire Kelonia Therapeutics in a deal worth up to $7 billion, aiming to expand its capabilities in in vivo CAR-T cell therapies. Kelonia’s proprietary gene delivery system uses engineered lentiviral particles to generate CAR-T cells directly inside the patient’s body, potentially bypassing the complex manufacturing steps required for traditional ex vivo approaches.

The company’s lead candidate, KLN-1010, is a one-time intravenous therapy targeting BCMA in multiple myeloma and is currently in Phase 1 trials. Early clinical data presented at the 2025 ASH Annual Meeting suggest promising tolerability and initial efficacy signals. The acquisition reflects growing interest in off-the-shelf and in vivo strategies designed to simplify CAR-T delivery, improve access, and reduce treatment timelines. Source

FDA approves first gene therapy for inherited hearing loss

The US Food and Drug Administration has approved Regeneron’s Otarmeni, the first gene therapy for patients with OTOF-related severe-to-profound sensorineural hearing loss. The dual AAV-based therapy delivers a functional copy of the OTOF gene directly to inner ear cells, aiming to restore otoferlin production and auditory signaling in this rare genetic condition.

The accelerated approval – granted in just 61 days under the agency’s National Priority Voucher program – was supported by data from the CHORD trial, in which 80 percent of patients achieved clinically meaningful hearing improvement, and 42 percent reached normal hearing levels, including the ability to detect whispers. The one-time therapy is administered via intracochlear infusion and represents the first approved gene therapy to restore a neurosensory function. Regeneron has also announced it will provide the treatment free of charge to eligible patients in the US. Source

CAR-T therapy delivers durable benefit in stiff person syndrome trial

Kyverna Therapeutics has reported positive primary analysis results from its registrational Phase 2 KYSA-8 trial of mivocabtagene autoleucel (miv-cel), showing statistically significant and durable clinical improvements in patients with stiff person syndrome (SPS). The data, presented in a late-breaking oral session at the American Academy of Neurology (AAN) Annual Meeting, indicate that a single infusion of the CD19-targeting CAR-T therapy improved mobility and reduced disability across all primary and secondary endpoints at 16 weeks.

The single-arm study enrolled 26 patients with inadequate responses to prior immunomodulatory therapies. The trial met its primary endpoint, with a 46 percent median improvement in the Timed 25-Foot Walk and 81 percent of patients achieving clinically meaningful gains. Notably, all patients discontinued chronic immunotherapies, while secondary measures of disability, stiffness, hypersensitivity, and mobility also showed significant improvement. Safety findings were consistent with CAR-T expectations, with no high-grade cytokine release syndrome or neurotoxicity events reported. Source

UCB targets regenerative epilepsy therapy with Neurona acquisition

UCB has announced plans to acquire Neurona Therapeutics in a deal worth up to $1.15 billion, marking a strategic move into regenerative medicine for epilepsy. The acquisition centers on Neurona’s lead asset, NRTX-1001, a neuronal cell therapy currently in Phase I/II trials for drug-resistant mesial temporal lobe epilepsy, a condition with significant unmet need and limited treatment options beyond symptomatic control.

NRTX-1001 is designed as a one-time, minimally invasive therapy that delivers inhibitory neurons directly into the brain to restore balance in overactive neural circuits. The cells produce GABA, aiming to reduce seizure activity by repairing dysfunctional networks rather than simply suppressing symptoms. Early data supporting the program have led to both FDA Regenerative Medicine Advanced Therapy (RMAT) and EMA PRIME designations, reflecting its potential to address a high-burden neurological disorder. Source 

CAR-T therapy shows deep, durable responses in early myeloma setting

A Phase 2 trial from Dana-Farber Cancer Institute has reported strikingly deep and sustained responses to CAR-T cell therapy in patients with high-risk smoldering multiple myeloma, marking the first study to test the approach in this early disease stage. The results, presented at the American Association for Cancer Research (AACR) Annual Meeting and published in Nature Medicine, showed that all 20 treated patients achieved minimal residual disease (MRD) negativity within two months of a single infusion of ciltacabtagene autoleucel (cilta-cel).

After a median follow-up of 15.3 months, all patients remained MRD-negative, with no disease progression or deaths reported. The study enrolled patients at high risk of progression to active multiple myeloma, a stage typically managed with observation rather than intervention. Safety findings were consistent with known CAR-T profiles, with no high-grade toxicities observed and only low-grade cytokine release syndrome reported. The data support the hypothesis that earlier use of CAR-T – when tumor burden is lower and immune function is more intact – may drive deeper, more durable responses, raising the possibility of long-term disease control or even cure with a one-time treatment. Source 

CAR-T therapy shows early activity in aggressive thyroid cancers

A first-in-human Phase I trial from MD Anderson Cancer Center has reported encouraging early responses for a CAR-T therapy targeting ICAM-1 in patients with advanced thyroid cancers. The results, presented at AACR, highlight progress in extending CAR-T approaches to solid tumors, particularly in anaplastic and poorly differentiated thyroid cancers with limited treatment options and poor prognosis.

Among patients treated at higher dose levels, objective responses were observed, including one complete response and one partial response, alongside disease control in more than half of evaluable patients. The therapy, AIC100, is a third-generation CAR-T designed to target ICAM-1 and includes a co-expressed imaging marker to enable in vivo tracking via PET. Safety findings showed no dose-limiting toxicities at initial dose levels and manageable cytokine release syndrome, although higher dosing was associated with pneumonitis in a small number of patients. The data support further clinical development, with a recommended dose identified for Phase II evaluation. Source