I’ve been trying to find the words to describe the flood of emotions that have been swirling inside of me since talking with the team at Nanoscope Therapeutics about their recently announced vision restoring optogenetics therapy for late stage RP. I’ve alternated between crying, doing a happy dance, and shouting out thank you prayers to the universe. After trying to claw my way out of a dark pit for the past 17 years, I’ve finally found light. And I’m so full of hope and gratitude that I’m ready to burst.
In case you somehow missed it, Nanoscope Therapeutics, a clinical stage biotech based in Bedford Texas, issued a June 3rd press release detailing the results of their Phase 1/2a optogenetics clinical trial that took place in India over the past 2 years. By all accounts, it appears that they have cracked the code when it comes to restoring vision in late stage RP patients — regardless of gene mutation.
I’m talking functional vision restoration — not shadows, or dark blobs, or white stripes. Real, functional vision that has previously-blind patients navigating complex public transportation on their own, watching TV, reading newspaper headlines and, in one case, even riding a bike and recognizing facial features.
What’s more, their therapy isn’t dependent on implanted microchips, or on wearing external devices such as cameras or goggles. It involves a single injection of molecules into the eye targeting bipolar cells that lay just beneath the photoreceptors cells that are destroyed by RP. The therapy activates those bipolar cells, turning them into light sensitive cells that react to ambient light — the kind of natural light found in everyday living. And as far as researchers can tell, the vision restoration appears to be permanent.
“The results varied among patients, with some better than others,” notes Nanoscope’s Co-Founder and President, Samarendra Mohanty, who has been working on the development of the therapy since 2006. “But every patient who received the therapy has experienced significant improvement — both objectively and subjectively.”
The eleven patients involved in the phase 1/2a clinical trial underwent extensive testing over the course of a year, utilizing every vision-measuring metric the team could apply.
From a straight visual acuity standpoint, Mohanty said the most dramatic improvement saw a patient’s visual acuity jump from 20/2000 to 20/200. But that was just the beginning.
Patients in the trial also underwent visual field tests to measure peripheral vision, tests to measure light perception in progressively dim light conditions, and even tests to measure shape and determination accuracy. A variety of mobility tests were also performed to see how accurately a patient could identify a light source and moving objects. Dramatic improvements were seen across the board.
Along with experiencing dramatic visual improvements within four weeks of the therapy injection, Mohanty said that Nanoscope’s optogenetics therapy seeks to address another critical factor: the halting of further degeneration.
“After the photoreceptors cells die, the retina continues to degenerate,” notes Mohanty. “But we have preliminary indication of halting further degeneration and that’s very very exciting for us ”
The next step in bringing this therapy to patients everywhere is the newly announced Phase 2b clinical trial in the United States that will kick off within the next few weeks and be administered at several locations throughout the country (for more information, go to clinicaltrials.gov or contact the clinical trial coordinator, Kristen Peterson, at info@nanostherapeutics.com.). Like in India, the U.S. clinical trial is focused on late stage RP patients with bare-to-no light perception. To qualify, a patient’s visual acuity must be 20/1600 or worse.
“We wanted to start with them because we wanted to give hope to people who have absolutely no hope and have been left behind,” explains Nanoscope’s Co-Founder and CEO, Sulagna Bhattacharya, who watched her beloved uncle go blind as a child and experienced the devastating impact it had on him and her entire family. “This is why we started with RP. Our therapy also has potential to help Stargardt, and we are confident that it will also potentially benefit those with dry age-related macular degeneration. But most of the Dry-AMD patients still have a little vision left, and we wanted to start with those who were suffering the most.”
The phase 2b trial results are expected in the third quarter of 2022. If all goes according to plan, the FDA may be able to expedite the approval process so that Nanoscope can get their life-transforming therapy to patients as soon as possible.
There is too much to this story to capture in a single post. But I’ll cover it in depth in several segments over the next month. These segments will include:
- The fifteen-year story behind the development of this breakthrough therapy and Mohanty’s determination to figure out how to restore vision in ambient light — which was key in activating the bipolar cells and restoring meaningful vision
- The remarkable journey of Bhattacharya, who has devoted her life to finding a cure for blindness after her uncle lost his eyesight — even while enduring unimaginable loss and grief
- The team’s commitment to making this life-changing therapy affordable and accessible to patients everywhere, and the uphill challenge to achieve this
Like all of you who have been following this blog since its inception in early 2013, I’ve refused to believe that I am destined to live in a world of darkness. Even after my eyesight took a nose-dive in 2018 and my retinal specialist told me it was time to transition to blindness, I’ve maintained my determination to see. Now, though there is still a ways to go, it feels close enough to touch. I am so grateful to all of the scientists and researchers out there who are devoting their lives and careers to bringing back our eyesight and our lives. And I’m making it my mission to do everything I can to raise awareness and help get these life-changing treatments out to the world.
Let’s hope that it Will continue working for the next phases!
Exactly!
Dear Ingrid and Jeanne,
I am so happy for you and will look forward to hearing about your future treatment in this groundbreaking development for RP. I have a different situation where my retinal detachment while traveling in Italy in 2012 resulted in secondary glaucoma due to the silicon oil placed in my eye to attach the retina. But like you, I have hope for a future of better vision and reversing the 20/800 in my left eye for more functional vision.
I will keep a good thought going for you two!
Hey Joan,
I am holding you in my thoughts and channeling an answer for all of us. <3
God bless you!! You’re a really nice person
Amazing,thanks so much for posting
Oh Hi Ingrid!
Wow that is great news Ingrid. I’m so happy for you and will be praying for all to go well.
Gary and I are on our way home from Uganda because of the lock downs here for Covid-seeming community spread now. I was explaining to some of our leaders here just the other day, that meeting in South Sudan years ago, when our politics differed—but we could still be friends. (Seemed a novel thought to them then!) Now we have leaders from many tribes, different militaries, even countries.) Would love to meet up with you when we are back.
Love to catch-up!
Thanks so much, Louise. I agree completely and would so love to catch up when you get back.
XO
Hello Ingrid, I am following you for few yeas now, wanted to go in that clinic in China but changed my mind after you experienced it and now waiting for gene testing and possible RP65 mutation to be eligible for Luxthurna gene therapy if possible. But the chances I have the RP65 mutation is very low.
Your article gives me a lot of hope again. Thank you so much for sharing this with us. It is indeed sooooooooo promising. I have RP and left with around 20% of sight, I am based in Quebec, Canada.
Thank you for your determination that help all of us.
Manon
Thank you so much for your words, Manon. I am feeling so hopeful. We’ve got this. 🙂
Hi Ingrid, So promising. Thank you so much to keep us posted. I was finally gene tested and have the homozygous for HGSNAT c. 1843G gene…which is associated with late-onset retinal distrophy and occured so far only in french Canadian people ! quite rare gene then.
Hope this new therapy will be soon accessible for you !
Manon
Such GREAT news!!!! I’m so glad to be reading this!
Hi Ingrid!
This is amazing news! It sounds so promising. I am so excited for you! I’m looking forward to hearing more about it as things progress.
So very happy to hear from you, my little friend. ❤️
Lori
Thanks, Lori. So great to hear your words. Sending love to you.
Dar Ingrid,
Thanks for your post and for always communicating what you learn in your travels with RP. It helps us all. Sending hugs from Graz.
Hey Kathryn, So great to hear from you. And thank you — I’m so hoping to come over there and SEE you soon.
Hi Ingrid,
Steve here. Great news with many thanks to you for keeping us in the loop ! Let’s keep our ‘eyes’ on this (smile). A (the) cure is in sight. All is well here and hoping you are enjoying the summer and the reduced restrictions re. Covid. (interesting that the Covid vaccines are based on messanger RNA first pioneered for vision ? (smile Crispr C-9) Ok. well we’ll be in touch and stay forcused.
Warm regards, Steve in T.O. http://www.singersvoice.ca
correction: focused
Thanks so much, Steve. And exactly!
Dear Ingrid: Your emotions cross my chest as if they were mine. This news could have reached me by any other means, but God wanted it to be your blog that I read first. And that’s only fair. I am glad to keep my hopes in the shadow of your search and your struggle. Maybe in a few years my daughter will be able to see again. God bless you.
Your words made me tear up. Yes, I just know your daughter is going to see. Take care.
Hi Ing, I’m so excited to hear this news. Your passion for the science is so deep and you’re making it accessible to the world in a way they need. You know i’m tracking your journey and look forward to a trip where we can truly see each other!
Thanks so much, Doug. As soon as my eyesight is back I’m coming to Pittsburgh and we are going to celebrate big time. XO
Hi Ingrid,
May God bless you with sight for the good work you are doing for people with genetic eye disorders.
Best Regards
VJ
Thank you so much for that. We are all going to see — I just know it.Take care.