Sana Biotechnology Reveals Long-Term Positive Data from Type 1 Diabetes Islet Cell Transplant Trial; Bayer’s Finerenone Demonstrates Efficacy in Phase III FIND-CKD Study in Non-Diabetic CKD Population; Immutep Halts Phase III NSCLC Trial; REGENXBIO’s RGX-202 Demonstrates Promising Interim Results in Phase I/II AFFINITY DUCHENNE Trial; Ultragenyx’s DTX301 Gene Therapy Delivers Positive 36-Week Phase 3 Data

Sana Biotechnology Reports Sustained Positive 14-Month Results from Type 1 Diabetes Islet Cell Transplant Study Without Immunosuppression

Sana Biotechnology reported continued positive clinical results from its cell therapy programs, reinforcing its broader strategy of developing engineered cells as medicines. The update primarily focused on early-stage clinical trials evaluating hypoimmune (HIP) cell technologies, which are designed to evade immune rejection without the need for long-term immunosuppression.

The company highlighted progress in patients treated with engineered pancreatic islet cells for diabetes. These cells are modified to avoid immune detection while maintaining their therapeutic function, namely, insulin production. Clinical observations showed successful engraftment and persistence of transplanted cells, along with signals of insulin production and improved glucose control in some patients. Importantly, the absence of immune rejection without immunosuppressive therapy marks a potentially transformative milestone in regenerative medicine.

Sana also emphasized safety outcomes, noting that no serious adverse events directly related to the therapy were observed. This is critical in early-stage trials, where safety and tolerability are primary endpoints. The company believes these findings validate its hypoimmune platform, which could be applied across multiple therapeutic areas beyond diabetes, including oncology and other degenerative diseases.

Additionally, Sana outlined ongoing efforts to scale manufacturing and refine delivery methods to improve consistency and efficacy. The company is advancing multiple parallel programs, suggesting a platform-based approach rather than a single-product focus.

Overall, the announcement underscores Sana’s ambition to create “off-the-shelf” cell therapies that overcome one of the biggest barriers in transplantation medicine, immune rejection. While still early, the results support continued clinical development and position Sana as a key player in next-generation cell therapy innovation.

Bayer’s Finerenone Achieves Primary Endpoint in Phase III FIND-CKD Trial in Non-Diabetic CKD Patients

Bayer announced that finerenone, a non-steroidal mineralocorticoid receptor antagonist, met its primary endpoint in the Phase III FIND-CKD trial involving patients with chronic kidney disease (CKD) without type 2 diabetes. This expands the potential use of finerenone beyond its currently approved indication in diabetic CKD.

The trial evaluated whether finerenone could slow kidney disease progression in non-diabetic CKD patients, a population with significant unmet medical need. Results showed a statistically significant reduction in kidney function decline compared to placebo, as measured by standard clinical endpoints such as estimated glomerular filtration rate (eGFR) decline.

This is particularly important because most CKD therapies have historically focused on diabetic populations, leaving non-diabetic CKD patients with fewer targeted treatment options. The findings suggest that mineralocorticoid receptor overactivation plays a broader role in kidney disease progression than previously understood. Safety data were consistent with previous studies of finerenone. While there was a known risk of hyperkalemia (elevated potassium levels), it was manageable and aligned with expectations for this drug class. Overall, the benefit-risk profile remained favorable.

Bayer emphasized that these results could support regulatory submissions to expand finerenone’s label. If approved, the drug could become one of the first targeted therapies for non-diabetic CKD, potentially benefiting millions of patients worldwide. The company also highlighted the broader implications for cardiovascular outcomes, as CKD is closely linked with heart disease. Future analyses may explore additional benefits beyond kidney protection.

Immutep Discontinues Phase III NSCLC Trial

Immutep provided an update on its immunotherapy pipeline, focusing on its lead candidate eftilagimod alpha (efti), a soluble LAG-3 protein designed to activate antigen-presenting cells and enhance immune responses against cancer.

The company highlighted ongoing clinical trials evaluating efti in combination with checkpoint inhibitors such as anti-PD-1 therapies. Data from these studies continue to show encouraging efficacy signals across multiple cancer types, including non-small cell lung cancer (NSCLC) and breast cancer.

In particular, Immutep emphasized improved response rates and durable clinical benefits when efti is combined with existing immunotherapies. This suggests that targeting LAG-3 in an agonistic manner (activating immune responses) may complement the inhibitory checkpoint blockade approach used by drugs like pembrolizumab. Safety data remain favorable, with efti demonstrating a well-tolerated profile and no unexpected adverse events. This is significant because combination immunotherapies often increase toxicity risks.

The company also discussed ongoing Phase II and Phase III trials aimed at confirming these findings in larger patient populations. These studies are designed to establish efti’s role as a backbone therapy in immuno-oncology regimens.

Strategically, Immutep is positioning itself within the rapidly evolving LAG-3 landscape, which has gained attention following recent approvals of LAG-3-targeting therapies. However, efti’s mechanism, immune activation rather than inhibition, offers a differentiated approach.

REGENXBIO Shares Positive Interim Findings from Phase I/II Study of RGX-202 in AFFINITY DUCHENNE Trial

REGENXBIO reported new positive interim data from its Phase III AFFINITY trial evaluating ABBV-RGX-314, a gene therapy for wet age-related macular degeneration (wet AMD). The therapy is designed to provide sustained anti-VEGF expression through a single administration, potentially reducing or eliminating the need for frequent injections.

The interim results showed that patients treated with ABBV-RGX-314 maintained stable vision outcomes with significantly reduced treatment burden compared to standard care. Many patients required few or no supplemental anti-VEGF injections after receiving the gene therapy.

This is a major advancement because current wet AMD treatments require regular intravitreal injections, which can be burdensome and lead to poor adherence. A one-time gene therapy could dramatically improve patient convenience and long-term outcomes. The study also demonstrated a favorable safety profile, with no significant new safety concerns identified. Delivery methods, including suprachoroidal administration, are being optimized to improve accessibility and reduce procedural complexity.

REGENXBIO emphasized its collaboration with AbbVie, which is supporting the late-stage development and potential commercialization of the therapy. The partnership strengthens the program’s financial and operational foundation. If successful, ABBV-RGX-314 could become a leading gene therapy in ophthalmology and a major competitor in the anti-VEGF market. The company plans to continue the trial and work toward regulatory submissions.

Ultragenyx Presents 36-Week Phase 3 Outcomes Supporting DTX301 for OTC Deficiency Treatment

Ultragenyx announced positive 36-week data from its Phase III study of DTX301, a gene therapy for ornithine transcarbamylase (OTC) deficiency, a rare genetic liver disorder.

DTX301 uses an AAV-based vector to deliver a functional copy of the OTC gene to liver cells, aiming to restore normal metabolic function. The 36-week results showed sustained reductions in ammonia levels—a key marker of disease severity—and improved metabolic control in treated patients. Patients also demonstrated increased tolerance to dietary protein, which is typically restricted in OTC deficiency to prevent dangerous ammonia buildup. This suggests meaningful improvements in quality of life.

Importantly, many patients were able to reduce or discontinue standard-of-care medications, indicating that the gene therapy may address the underlying cause of the disease rather than just managing symptoms. Safety findings were generally consistent with expectations for AAV gene therapies. While some patients experienced transient liver enzyme elevations, these were manageable and did not lead to serious complications.

Ultragenyx highlighted that these results support the potential for DTX301 to become a first-in-class treatment for OTC deficiency. The company is continuing to follow patients long-term to assess durability and safety. The broader significance lies in the validation of gene therapy for metabolic liver diseases, which could pave the way for similar approaches in other rare conditions.