ENCELTO officially hits the United States, offering MacTel patients a slice of hope.
One small injection for a patient, one giant leap for ophthalmology. The first use of ENCELTO (revakinagene taroretcel-lwey) outside a clinical trial marks a milestone for this cell-based gene therapy, created to slow idiopathic macular telangiectasia (MacTel).
Retina Consultants of Texas announces the news on its LinkedIn page.
For a quick recap, MacTel is like a delicate cake, quietly collapsing from the inside. The layers (photoreceptors) appear intact at first, but over time, they buckle and with them, vision fades. Until now, ophthalmologists could only watch the “cake” lose its structure, offering supportive care but no way to slow the damage.
That changed when Dr. Charles Wykoff of Retinal Consultants of Texas (USA) performed the first ENCELTO implantation in a real-world setting. “Performing the first ENCELTO surgery for a patient with MacTel outside of a clinical trial is an important milestone,” he said in a news release. “For the first time, we can offer a valuable and durable treatment option for patients, one that slows their loss of photoreceptors and maintains more visual function over time.”
READ MORE: The Promise of Gene Therapy in Retinal Disorders
The therapy, developed by Neurotech Pharmaceuticals (Rhode Island, USA) and backed by Phase III trial data, earned FDA approval earlier this year.
“We have been working toward this for years,” said Neurotech CEO Richard Small. “Seeing it become a reality is incredibly exciting for the Neurotech team and most importantly, for the patients who may benefit.”
How ENCELTO works
ENCELTO is a tiny, surgically implanted capsule placed in the vitreous cavity. Inside are genetically engineered retinal pigment epithelial cells that continuously release ciliary neurotrophic factor (CNTF), a protein that slows retinal degeneration, protects photoreceptors and helps shield them from immune attacks.
READ MORE: FDA Approves Neurotech’s ENCELTO for Treatment of Macular Telangiectasia Type 2
In the Phase III trial, ENCELTO slowed ellipsoid zone loss by roughly 55% over 24 months. To top it off, microperimetry testing revealed functional benefits, particularly in areas with the greatest photoreceptor preservation. Side effects were generally mild and transient, such as miosis and delayed dark adaptation, both of which resolved over time.1
Years in the making
This milestone caps decades of research into neuroprotective strategies for retinal disease. ENCELTO’s promise lies in its sustained drug delivery, supporting retinal structure continuously rather than relying on repeated injections or transient treatments.
READ MORE: Gene Therapy Shows Promise for AIPL1-Associated Retinal Dystrophy in Children
For ophthalmologists who have long watched MacTel steal vision layer by layer, ENCELTO offers something new: the possibility to preserve what remains. It can’t rebuild the cake, but it just might keep it from collapsing before the last slice is gone.
Editor’s Note: This content is intended exclusively for healthcare professionals. It is not intended for the general public. Products or therapies discussed may not be registered or approved in all jurisdictions, including Singapore.
Reference
- Chew EY, Gillies M, Jaffe GJ, et al. Cell-based ciliary neurotrophic factor therapy for macular telangiectasia type 2. NEJM Evid. 2025;4(8):EVIDoa2400481