Johnson & Johnson's Gene Therapy Fails Phase 3 Trial in Rare Eye Disease, Casting Shadow Over the Field

Johnson & Johnson bought the full rights to a gene therapy for a rare disease years ago, but now, this pharmaceutical giant may be experiencing“Buyer's Remorse”The moment. According to5Month2The results published on the day showed that in a study targetingXChained Retinitis Pigmentosa (XLRP) Patient's3In the clinical trial, the gene therapy failed to improve the patients' visually guided mobility.

 

LUMEOSA total of95Number of patients, including58Name received different doses (low dose or high dose) ofbotaretigene sparoparvovec(hereinafter referred to asbota-vec) Treatment.bota-vecIt is an investigational gene therapy that uses adeno-associated virus to target retinitis pigmentosa.GTPEnzyme Regulatory Protein (RPGR) A functional copy of the gene is delivered to the retina.

XLRP It is a severe and rare type of retinitis pigmentosa, which is a progressive eye disease that leads to the damage of photoreceptors over time, potentially resulting in blindness. This condition usually manifests during childhood and primarily affects boys.LUMEOSOnly a small number of female patients were enrolled in the trial.

 

In the process of all receivingbota-vecAfter a pooled analysis of the treated patients, the primary endpoint of the trial——Improvement in Patients' Visual Navigation Ability Through Virtual Mazes——Failed to achieve the expected goal. However, Johnson & Johnson stated that this result “The trend is favorable.”。

 

All Acceptbota-vecAll treated patients experienced at least one treatment-related adverse event, among which86%The events were considered mild or moderate. According to the announcement,53%The patients experienced at least one episode related tobota-vecRelated adverse events.

Nevertheless,bota-vecImprovements were shown in several secondary endpoints of the trial, including patient-reported vision and scores on the visual acuity chart. Although these resultspValue less than0.05, but Johnson & Johnson stated that these values are only used for descriptive purposes and are not statistically significant.

According to Johnson & Johnson, those receiving treatment55Number of patients with22The name showed improvement in two or more endpoints, while no patients in the control group showed improvement. As of4Month25Johnson & Johnson is still conducting trials for patients who were initially involved in the late-stage trial.3Follow-up study.

bota-vecIt was originally developed by Janssen, a division of Johnson & Johnson, in collaboration with Genzyme Corporation.MeiraGTxCo-developed.2023Year12At the end of the month, Johnson & Johnson reached a deal worth up to4.15The entire rights to the program were acquired through a deal worth hundreds of millions of dollars.

 

Johnson & Johnson's setback in the gene therapy field is not an isolated case. Recently, there have been multiple failures or exits in the gene therapy sector, drawing widespread attention from the industry.

2025Year2In the month, Pfizer announced the cessation of itsBHemophilia Gene TherapyBeqvezDevelopment and Commercialization.BeqvezIn2024Year4Month ObtainedFDAApproved for marketing,Priced as high as350Million USD, but due to weak market demand, no patients have received commercial treatment since its approval.In addition, Pfizer in2024The year also ended withSangamo TherapeuticsCooperativeAHemophilia Gene Therapygiroctocogene fitelparvovecThe development. As of now, Pfizer's product pipeline no longer includes any gene therapies in clinical or commercial stages.

Roche also for its gene therapy divisionSpark TherapeuticsConducted“Fundamental Restructuring”，Lead to337Employees unemployed,Remaining310Employees were merged into the Roche parent company.

2025Year5Month,VertexPharmaceutical company announces it will no longer use adeno-associated virus (AAV) as the delivery vector for gene therapy projects。The reason behind this decision isAAVThe immunogenicity issues and production process complexity of vectors exposed during clinical trials and commercialization.VertexThis shift also highlights the uncertainty in the field of gene therapy regarding the choice of technical pathways.

Bluebird Bio, a pioneer in the field of gene therapy (Bluebird Bio) also failed to escape the calamity.2025Year2Month21Recently, Bluebird Bio announced that it has been acquired by global investment firm Carlyle (Carlyle) andSK CapitalAcquisition. The company's valuation is only2900Million USD.

Bluebird Bio was once a star company in the field of gene therapy, and its stock price2018Annual up to per share230USD, Market value approaching300billion dollars. However, due to its gene therapy productsZynteglo、SkysonaAndLyfgeniaThe commercialization progress has been slow, with a limited patient population and facing high research and development and operational costs, the company's financial situation has continued to deteriorate. In addition, Bluebird Bio has repeatedly encountered difficulties due to safety issues, further undermining market confidence.

Gene Therapy: A Frontier Technology in the Medical Field

Despite setbacks in the field of gene therapy, it still shows great potential in the treatment of certain diseases. For example,ZolgensmaThe success shows that not all gene therapies cannot be commercialized. In the future, the development of gene therapy requires more innovation and breakthroughs to truly realize its value in disease treatment.

Reference:fierce biotechetc.

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