Biogen, Inc.

Who is Biogen?

Biogen is a global biotechnology company founded in 1978 that focuses on developing treatments for neurological and neurodegenerative conditions. Their work includes research on rare diseases, including gene therapies that may be relevant to retinal disorders.

Mission and Approach

Biogen’s mission is to advance science to develop therapies that could improve patients’ lives. They prioritize innovation, patient safety, ethical research practices, and inclusion in both their workforce and research efforts.

Relevance to the CHM Community

Biogen is involved in gene therapy research, which is one of the approaches being studied for choroideremia. While their work in this area is ongoing, it contributes to the broader understanding of potential treatments for rare retinal diseases. For patients and families, this research helps provide information on the kinds of therapies under investigation and the design of clinical studies.

Safety and Research Ethics

Biogen’s trials follow established clinical research guidelines and prioritize participant safety and careful monitoring.

Important note: Please note that any information about clinical trials or gene therapies is for informational purposes only. The Choroideremia Research Foundation does not endorse any specific company, clinical trial, or genetic test. Patients should discuss any questions with their healthcare provider.

Biogen Sponsored Studies

A Safety Study of Retinal Gene Therapy for Choroideremia With Administration of BIIB111

NCT: NCT03507686
URL: https://clinicaltrials.gov/study/NCT03496012
Intervention: DRUG: BIIB111

Technical Summary: At 12 months, VA was generally maintained in both eyes following bilateral sequential subretinal injection of BIIB111. Across 66 participants, the mean BCVA change was small and not clinically significant (–2.8 letters +/- 16.0 SD) despite both eyes undergoing retinal detachment and subretinal injection. No meaningful deterioration was observed in microperimetry, autofluorescence, or OCT structure at month 12. Safety outcomes showed low rates of serious surgical complications (7.6%), mostly mild-to-moderate inflammation manageable with steroids. Retinal inflammation occurred in 45% of participants but was not correlated with significant vision loss. Importantly, no systemic immune reactions or additive toxicity were seen between the first and second eye. Overall, the study supports that bilateral gene therapy is feasible and well-tolerated, providing evidence that prior AAV2 exposure does not preclude safe sequential treatment.

Lay Language Summary: After one year, participants in the study generally maintained their vision in both eyes after receiving the BIIB111 gene therapy in each eye, one after the other. Among the 66 participants, the average change in visual acuity was very small and not considered clinically meaningful, even though both eyes went through a controlled retinal detachment and subretinal injection as part of the procedure. Other measures of vision and retinal health, including light sensitivity testing, autofluorescence imaging, and OCT scans, also stayed stable over the 12-month period. The treatment’s safety profile was encouraging. Serious surgical complications were uncommon, occurring in about 8 percent, and most inflammation was mild to moderate and responded well to steroid treatment. Retinal inflammation occurred in nearly half of participants but did not lead to significant vision loss. There were no signs of harmful immune reactions in the body, and treating the second eye did not add any safety concerns. Overall, the results suggest that treating both eyes with this type of gene therapy is possible and generally safe, and that previous exposure to the AAV2 vector does not prevent safe treatment of the second eye.

Efficacy and Safety of BIIB111 for the Treatment of Choroideremia

NCT: NCT03496012
URL: https://clinicaltrials.gov/study/NCT03496012
Intervention: GENETIC: BIIB111

Technical Summary: In this phase 3 trial, patients with choroideremia received a single subretinal injection of BIIB111 at either high dose (1.0×10¹¹ vg; n=69) or low dose (1.0×10¹⁰ vg; n=34) versus a no-treatment control (n=66). At 12 months, the study did not meet the primary endpoint: ≥15-letter BCVA gains occurred in 5% (3/65) of high-dose, 3% (1/34) of low-dose, and 0% (0/62) of controls; key secondary endpoints were therefore not tested for significance, though ≥10-letter gains were seen in 14–18% of treated eyes vs 2% of controls. Safety was largely ocular and manageable: most adverse events were mild/moderate; in the lay summary safety set (n=164), any AE occurred in 91–94% of treated participants vs 51% of controls, serious AEs in 17–26% vs 15%, and no deaths were reported which is consistent with vitrectomy/subretinal procedures.Overall, STAR shows that while subretinal BIIB111 was generally tolerable, it failed to demonstrate efficacy on BCVA at 12 months in this pivotal setting.

Lay Language Summary: In this Phase 3 study, people with choroideremia received a single injection of the gene therapy BIIB111 under the retina. Participants received either a high dose, a low dose, or no treatment. After one year, the main goal of the study, significant improvement in vision, was not met. A small number of treated participants did experience noticeable vision gains, and slightly more people in the treated groups had smaller improvements compared with the no-treatment group.The treatment was generally safe. Most side effects were related to the eye and were mild to moderate, and serious side effects were uncommon. No deaths occurred, and the safety profile was similar to what is expected with this type of eye surgery. Overall, this study showed that BIIB111 injections under the retina were generally well tolerated but did not significantly improve vision after 12 months in this trial.