Stem Cells in Ophthalmology Update 9 First Patients Treated

As I reported back on June 16th, Advanced Cell Technology had enrolled the first two patients in its Phase I/II clinical trials using retinal pigment epithelial (RPE) cells derived from embryonic stem cells (hESCs) for treating Stargardt’s Macular Dystrophy (SMD) and for the treatment of the dry form of age-related macular degeneration (Dry AMD). The company announced today that these first patients had now successfully received their first dose of the stem cells.

(Editors note: Please also see the addendum attached at the end of this piece.)

Here is the announcement:

ACT Announces First Patients Undergo Embryonic Stem Cell Transplantation Treatment for Stargardt's Disease and Macular Degeneration at UCLA's Jules Stein Eye Institute


MARLBOROUGH, Mass., July 14, 2011 /PRNewswire/ -- Advanced Cell Technology, Inc., today announced the dosing of the first patients in each of its two Phase 1/2 clinical trials for Stargardt's macular dystrophy and dry age-related macular degeneration (dry AMD) using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs). The patients were treated Tuesday (July 12) by Steven Schwartz, M.D., Ahmanson Professor of Ophthalmology at the David Geffen School of Medicine at UCLA and retina division chief at UCLA's Jules Stein Eye Institute. Robert Lanza, M.D., chief scientific officer of ACT, attended the procedures. Both patients successfully underwent the outpatient transplantation surgeries and are recovering uneventfully.

Both the Stargardt's trial and the dry AMD trial will enroll 12 patients each, with cohorts of three patients each in an ascending dosage format. Both trials are prospective, open-label studies designed to determine the safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation into patients with Stargardt's and dry AMD at 12 months, the studies' primary endpoint.

"This first treatment milestone is welcomed by scientists, stem cell advocates and patients hoping for cures," said Gary Rabin, interim chairman and chief executive officer of ACT. "The two trials could not have started any smoother, and we are very pleased to announce that the procedures went well. The dosing of the first patients represents an important milestone for ACT and opens the doors to a potentially significant new therapeutic approach to treating the many forms of macular degeneration. We believe that these procedures represent a key step forward in therapeutic stem cell research, and the capacity to treat a variety of devastating diseases."

Dr. Schwartz, the studies' principal investigator, explained, "One patient in each clinical trial, the Stargardt's trial and the dry AMD trial, has undergone surgical transplantation of a relatively small dose (50,000 cells) of fully-differentiated retinal pigment epithelial (RPE) cells derived from human embryonic stem cells. Early indications are that the patients tolerated the surgical procedures well. The primary objective of these Phase 1/2 studies is to assess the safety and tolerability of these stem cell-derived transplants. We will be carefully monitoring our patients over the course of the trials. We are privileged to be collaborating with ACT and honored to be working with these pioneering patients."

Dry AMD, the most common form of macular degeneration, Stargardt's and other forms of atrophy-related macular degeneration are usually untreatable. Safe and effective therapies are greatly needed for the treatment of these common forms of blindness. Disease progression of both Stargardt's and dry AMD includes thinning of the layer of RPE cells in the patient's macula, the central portion of the retina and the anatomic location of central vision. With RPE cell death comes the loss of macular photoreceptors and loss of central vision. Currently both conditions are untreatable and often lead to legal blindness over a multi-year course. ACT's Stargardt's and dry AMD therapies treat these conditions by transplanting RPE cells in the patient's eyes before the RPE population is lost.

"Today -13 years after the discovery of human embryonic stem cells - the great promise of these cells is finally being put to the test," said Dr. Lanza. "The initiation of these two clinical trials marks an important turning point for the field.  While we will continue writing research papers and carrying out more research, it's time to start moving these exciting new stem cell therapies out of the laboratory and into the clinic.  Tens of thousands of people continue to die every day from diseases that could potentially be treated using stem cells. In the meantime, we intend to accelerate our efforts to translate new embryonic stem cell (ES) and induced pluripotent stem (iPS) cell therapies into the clinic. It has taken years of extensive research to get to this point. Our research and preclinical studies have demonstrated the safety and effectiveness of such therapies.  We hope these cells may provide a treatment option not only for degenerative eye diseases, but for a wide spectrum of other debilitating conditions, ranging from diabetes to vascular and autoimmune diseases. Our team remains committed to moving the field of regenerative medicine forward from bench to bedside."


Addendum: Along with the news from the company and UCLA shown in the press release above, the Los Angeles Times covered the story and presented some interesting additional information. I have attached the LA Times writeup as an addendum to this piece.


Stem cell clinical trials to treat eye diseases begin at UCLA

By Daniela Hernandez,
Los Angeles Times/For the Booster Shots blog
July 14, 2011, 2:01 p.m.

After more than 20 years of research, doctors at UCLA's Jules Stein Eye Institute have begun treating the first patients in clinical trials for two progressive eye diseases that cause blindness: dry age-related macular degeration and Stargardt's macular dystrophy.

The patients were given an injection of specialized eye cells that were derived from embryonic stem cells. Dr. Steven Schwartz, who is leading the trial at UCLA, performed both stem cell transplant surgeries Tuesday. The two patients are said to be recovering without complications.

According to Dr. Robert Lanza, chief scientific officer at Advanced Cell Technologies Inc., which developed the cells and is sponsoring the trials, "you could feel the excitement in the air and that history was being made."

Surgery began for the first of two patients, a 77-year old woman with dry macular degeneration, around 9:30 a.m. Tuesday and took about half an hour. In the afternoon, a 27-year old woman with Stardgart's macular dystrophy underwent the same procedure. Both patients are legally blind.

Doctors will monitor these two patients over the coming weeks. Another set of surgeries is scheduled to start in August.

Schwartz explained how these trials will help patients and what they mean for regenerative medicine. His comments were edited for space and clarity.

What are dry age-related macular degeneration and Stargardt's macular dystrophy?

Twenty percent of age-related macular degeneration is wet macular degeneration and it is treatable.  The other 80% of people have an untreatable, progressive visual loss leading to legal blindness called dry, or atrophic, macular degeneration.

The retinal cells, the rods and cones, and the underlying retinal pigment epithelium atrophy. As they atrophy, there is a long period of time when they are compromised and then they're gone.

Stargardt's is a genetic disorder and it strikes earlier in life. Patients start to notice visual changes as early as their teens and as late as their forties. There are a number of known genetic abnormalities in the photoreceptors that are toxic over time.

In this trial, what cells in the eye are you replacing with stem cells?
Advanced Cell Technologies Inc. has been able to take [embryonic] stem cells and differentiate them into highly functional retinal pigment epithelium that do everything they're supposed to do. Our strategy of giving the eye brand-new, ready-to-go retinal pigment epithelium is designed for areas that are compromised, not for the areas that are gone. So we need to catch it early enough for this treatment to work.

Will the patients regain vision?

The patients' central vision is already gone. Not rescuable. So the patients we're enrolling in this trial know they will not be getting their central vision back.

If not to restore vision, what is the goal?
This is a safety trial. It's not designed to improve vision. It may; and if we see a signal, that would be great news and we're hoping we will. It's plausible biologically, but that's not what we're looking for.

What results are you hoping to see?

I hope what happens is that we find this is safe and that we can optimize the dosing, and that allows us to move into eyes that are earlier in disease. That could have a real visual upside.

How long is the surgery?

Under an hour. It's an outpatient procedure done with local anesthesia. It's a surgery that we've done before - not with the injection of these stem cells, but we've accessed the eye before, and that's one of the things that I've had a lot to do with surgically.

What does this trial mean for medicine?

We're super-privileged to be taking this first step. It's the unknown. These patients are doing a service for mankind. It's inspirational.

Stem Cells in Ophthalmology Update 10 ACT Expands Trials for Embryonic Stem Cells for Stargardt’s to the UK

In a news announcement today, Advanced Cell Technology said it had received approval to expand its stem cell treatment for Stargardt’s Macular Dystrophy to Moorfield’s Hospital in the UK.

As reported by the Guardian, “The Massachusetts-based company Advanced Cell Technology (ACT) announced the trial ... will run alongside a similar study that began in July at the Jules Stein Eye Institute at the University of California, Los Angeles.

Only one patient has been treated so far in the US trial for Stargardt's disease. The results from both studies are expected next year.”

Here is the company’s announcement:

ACT Receives Approval for First Human Embryonic Stem Cell Trial in Europe

Moorfields Eye Hospital in London is Site for Phase 1/2 Trial to Treat Stargardt's Macular Dystrophy

MARLBOROUGH, Mass. - Sept. 22, 2011 - Advanced Cell Technology, Inc., a leader in the field of regenerative medicine, announced today that it has received clearance from the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) to begin treating patients as part of a Phase 1/2 clinical trial for Stargardt's Macular Dystrophy (SMD) using retinal pigment epithelium (RPE) derived from human embryonic stem cells (hESCs). ACT received similar approval from the the Gene Therapy Advisory Committee (GTAC), which has responsibility for the ethical oversight of proposals to conduct clinical trials involving gene or stem cell therapies in the U.K. The European Medicines Agency (EMA) previously granted Orphan Drug designation for the company's RPE cell product for use in treating SMD.

"This is another important milestone for ACT and for the field of regenerative medicine," said Gary Rabin, chairman and CEO of ACT. "We are pleased that the Moorfields Eye Hospital in London has agreed to participate as a site for this study as we continue to assess the capabilities of hESC-derived RPE cells to repair the retina and reduce the impact of these devastating eye diseases. We recently announced the dosing of the first patients in our Phase 1/2 clinical trials for Stargardt's macular dystrophy and dry age-related macular degeneration (dry AMD) with hESC-derived RPE cells in the U.S., and both patients successfully underwent the outpatient transplantation surgeries. (Editors Note: See Updates 8 and Update 9) Clearance from the MHRA to begin an SMD trial in the U.K. is the first step in our European clinical trial program. Europe not only represents the world's second-largest pharmaceutical market, but it is also home to some of the best eye hospitals and surgeons in the world. Building international relationships around our clinical programs, such as with Professor James Bainbridge at Moorfields Eye Hospital is very important to our strategy of developing new regenerative medicine therapies."

Stargardt's Macular Dystrophy affects an estimated 80,000 to 100,000 patients in the U.S. and Europe, and causes progressive vision loss, usually starting in people between the ages of 10 to 20. Eventually, blindness results from photoreceptor loss associated with degeneration in the pigmented layer of the retina, the retinal pigment epithelium. The first patient to be treated in the U.S. with stem cell-derived RPE cells was a young woman who was already legally blind as a consequence of this disease. This newly-approved clinical trial in Europe will be a prospective, open-label study designed to determine the safety and tolerability of RPE cells derived from hESCs following sub-retinal transplantation to patients with advanced SMD, and it is similar in design to the FDA-cleared U.S. trial initiated in July.

"This is the first time an embryonic stem cell trial has ever been approved anywhere else in the world," said Robert Lanza, M.D., ACT's chief scientific officer. "Stargardt's disease is currently untreatable, and is one of the leading causes of juvenile blindness in the world. Collectively, degenerative eye diseases afflict over 25 million people in the U.S. and Europe alone. These diseases have a devastating impact on patients and their families, which has been a strong motivating factor for developing this new treatment. In Stargardt's disease, the loss of RPE cells in the patient's macula causes a loss of photoreceptors - the cones and rods with which we see - leading to blindness. We believe that transplanting new, healthy RPE cells may provide an effective treatment for SMD and perhaps other macular degenerative diseases such as dry AMD. We are excited to start these trials in Europe, and look forward to analyzing the data we continue to collect in our ongoing trials to determine the engraftment and function of the transplanted RPE cells."

The trial will be led by Professor James Bainbridge, consultant surgeon at Moorfields Eye Hospital and Chair of Retinal Studies at University College London.

"Stargardt's disease is a form of macular degeneration that causes disabling loss of sight in young people and is currently untreatable," said Professor Bainbridge. "There is real potential that people with blinding disorders of the retina including Stargardt's disease and age-related macular degeneration might benefit in the future from transplantation of retinal cells. The ability to generate retinal cells from stem cells in the laboratory has been a significant advance and the opportunity to help translate such technology into new treatments for patients is hugely exciting. Testing the safety of retinal cell transplantation in this clinical trial will be an important step towards achieving this aim."

About Macular Degeneration and SMD

Degenerative diseases of the retina are among the most common causes of untreatable blindness in the world. As many as 30 million people in the U.S. and Europe suffer from macular degeneration, which represents a $25-30 billion worldwide market that has yet to be effectively addressed.

Menu 21 A List of Writeups on Gene Therapy Used in Ophthalmology

As with my menu on stem cells used in ophthalmology (Menu 20), here is one for the current articles on the use of gene therapy in ophthalmology, with links to the full writeups.

(Updated May 25, 2014)

Gene Thearpy

The Use of Gene Therapy in Treating Retinitis Pigmentosa and Dry AMD Nov. 2010

After several discussions with Sean Ainsworth, the founder of RetroSense, and much online research, I think I have learned a little about what gene therapy is about, and its application in ophthalmology, especially in the possible restoration of vision in those who suffer from retinitis pigmentosa (RP). Thanks to Sean for whetting my appetite -- here is what I have learned.

Gene Therapy Update 1: First Clinical Trial for a Form of Retinitis Pigmentosa (RP) Approved to Begin Oct. 2010

In an announcement today, Oxford BioMedica said that it had gained approval from the FDA to begin a Phase I/IIa Clinical Trial for a form of Usher’s Syndrome, Type 1B, which leads to progressive retinitis pigmentosa combined with a congenital hearing defect.

Gene Therapy in Ophthalmology Update 2: Foundation Fighting Blindness Funds Six New Gene Therapy Projects  Oct. 2010

In a news release that I found on the net, I learned that the Foundation Fighting Blindness was going to put $8.25 million into six gene therapy projects, either already underway or about to start. The release contains good information about several projects that I knew about, and others that I did not.

Gene Therapy in Ophthalmology Update 3: Genetic Testing of RP Patients Necessary in Order to Direct Treatment  Nov. 2011

In  another of the presentations made during the Retina SubSpecialty Day Meeting, Dr. Stephen Tsang presented on factors and the genetics of retinitis pigmentosa. His paper was based on the article previously published by he and his co-author, Kyle Wolpert, that appeared in the November 2010 issue of Retinal Physician.

Gene Therapy in Ophthalmology Update 4: Table of Companies and Institutions Participating Nov. 2011

Again, this table is currently out-of-date. See Update 16.

Gene Therapy in Ophthalmology Update 5: A Complement-Based Gene Therapy for AMD Dec. 2011

A writeup about a start-up company, Hemera Biosciences, with a gene therapy approach to treating dry AMD.

Gene Therapy in Ophthalmology Update 6: First-Ever Clinical Trial for the Autosomal Recessive Form of Retinitis Pigmentosa (arRP) is Underway Dec. 2011

Thanks to my friends at the Foundation Fighting Blindness, I learned about this first human clinical trial using gene therapy for treating recessive retinitis pigmentosa.

Gene Therapy in Ophthalmology Update 7: 2012 the Year for Gene Therapy? Jan. 2012

In this opus, I discuss my reasons why I think 2012 is going to be the year for gene therapy and also presented my table of current clinical trials underway. (Again, note there is now an updated table available via Update 16.)

Gene Therapy in Ophthalmology Update 8: Promising Results in the Treatment of Leber’s Congenital Amaurosis (LCA)  Jan. 2012

A report on the progress being made in treating Leber’s using gene therapy, as reported by the Foundation Fighting Blindness.

Gene Therapy in Ophthalmology Update 9: Oxford BioMedica/OHSU Preparing to Treat First Usher Syndrome Patient & Oxford BioMedica Ophthalmic Program Update Mar. 2012

A report on the start of the program to treat Usher Syndrome patients at OSHU, and an update on other ophthalmic programs underway by Oxford BioMedica.


Gene Therapy in Ophthalmology Update 10: Gene Therapy Research in Dogs Cures X-Linked Retinitis Pigmentosa – Paves the Way for Similar Treatment in Humans  Mar. 2012

Researchers at several universities and laboratories collaborated to treat dogs afflicted with the x-linked form of retinitis pigmentosa, to deliver the therapeutic RPGR gene specifically to the diseased rods and cones, which led to functional and structural recovery. This is the first proof that this condition is treatable in an animal model and the researchers feel the results are promising and relevant for translation to humans afflicted with this disease.


Gene Therapy in Ophthalmology Update 11: Clinical Trial Details May 2012

In attempting to determine how many patients had been treated with gene therapy for eye disorders, I quickly found that no one was keeping track – at least no one that I could find.

So, I decided to try and get this data. I have now found reliable data for more than two-thirds of the 16 clinical trials underway and present this information in my new table. My latest table (available via Update 16) contains all of the newest data.

Editors Note: See Update 16 for access to the latest versions of the three tables.


Gene Therapy in Ophthalmology Update 12: First Gene Therapy Approval on the Horizon Jul. 2012

As Andrew Pollack writes in today’s NYTimes, “After more than two decades of dashed expectations, the field of gene therapy appears close to reaching a milestone: a regulatory approval. The European Medicines Agency has recommended approval of a gene therapy to treat a rare genetic disease.”

The therapy recommended for approval in Europe, called Glybera, was developed by uniQure, a Dutch company. It treats lipoprotein lipase deficiency, a disease that affects only several hundred people in the European Union and a similar number in North America.

People with the disease have a genetic mutation that prevents them from producing an enzyme needed to break down certain fat-carrying particles that circulate in the bloodstream after meals. Without the enzyme, so much fat can accumulate that the blood looks white rather than red.

The reason I believe that this is important is because it brings “legitimacy” to the whole field of regenerative medicine. As readers of this online Journal are aware, my interest is in the field of ophthalmology. As you may be further aware, I am currently tracking eleven clinical trials involving the use of stem cells to treat ophthalmic disorders and sixteen gene therapy clinical trials. Several of these are showing promising results and the above approval, when it comes, will bring increased attention to the whole of this field, including the ophthalmic trials.

Gene Therapy in Ophthalmology Update 13: New Clinical Site for Usher Syndrome Clinical Trials  Jul. 2012

The Foundation Fighting Blindness and Oxford BioMedica announced funding for a second clinical site to conduct a gene therapy trial for Ushers Syndrome. The site will be the Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts in Paris, and will join the ongoing clinical trial being held at the Oregon Health & Science University's Casey Eye Institute.

Gene Therapy in Ophthalmology Update 14: Early Positive Results in Ongoing Gene Therapy Wet AMD and Stargardt’s Disease Studies Aug. 2012

Last week, Oxford BioMedica and its partner Sanofi announced positive results in their ongoing gene therapy clinical trials for wet AMD and Stargardt’s disease. In an interim review of their Phase I (RetinoStat) and Phase I/IIa (StarGen) trials, the Data Safetly Monitoring Board (DSMB), an independent panel of specialists in the fields of ophthalmology, virology and vectorology, gave the go ahead to proceed to a final patient cohort in the Phase I study in the case of the RetinoStat trial, and to a third patient cohort in the Phase I/IIa study of the StarGen trial.

Gene Therapy in Ophthalmology Update 15: First Gene Therapy Treatment Approved! Nov. 2012

As I first wrote back in July (Update 12: First Gene Therapy Approval on the Horizon), the first approval of a gene therapy application in medicine was expected soon. It has now been accomplished. On November 2nd, the European Medicines Agency gave final approval to a gene therapy approach to treat a rare genetic disease.

The therapy, given approval in Europe, called Glybera, was developed by uniQure, a Dutch company. It treats lipoprotein lipase deficiency (LPLD), a disease that affects only several hundred people in the European Union and a similar number in North America.

The reason I am noting this accomplishment in this space, where I normally write about treatments for ocular diseases is, because it brings “legitimacy” to the whole field of regenerative medicine. As readers of this online Journal are aware, my interest is in the field of ophthalmology. As you may be further aware, I am currently tracking twenty one clinical trials involving the use of stem cells (or cell threapy) to treat ophthalmic disorders and sixteen gene therapy clinical trials. Several of these are showing promising results and the above approval will bring increased attention to the whole of this field, including the ophthalmic trials.


Gene Therapy in Ophthalmology Update 16: Current Tables Now Online Jan. 2013/May 2014

Access to the three updated tables of information about the companies and institutions active in gene therapy, the ophthalmic applications being pursued, and the clinical trials underway and completed.


Gene Therapy in Ophthalmology Update 17: Hemera Biosciences Obtains Initial Funding Mar. 2013

Hemera biosciences has obtained initial funding, along with the issuance of a US Patent covering their technology and can now begin manufacturing its drug, soluble CD59 (protectin), perform animal toxicology, and initiate a phase 1 clinical study.

To review, HMR59 is a gene therapy using an AAV2 vector to express a soluble form of a naturally occurring membrane bound protein called CD59 (sCD59), which blocks MAC. Membrane attack complex is the final common pathway of activation of the complement cascade, and is composed of complement factors C5b, C6, C7, C8 and C9 that assemble as a pore on cell membranes. The MAC pore induces ionic fluid shifts leading to cell destruction and ultimate death. 

HMR59 works by increasing the production of sCD59 by ocular cells. The sCD59 released from the cells will circulate throughout the eye and penetrate the retina to block MAC deposition and prevent cellular destruction. By blocking MAC, the remainder of the upstream complement cascade is left intact to perform its normal homeostatic roles.


Gene Therapy in Ophthalmology Update 18: A RetroSense Update  Mar. 2013

Since I first wrote about RetroSense in November 2010, I have learned that they are using a unique technology, called Optogenetic Therapy to treat retinitis pigmentosa and dry AMD. Optogenetics combines gene therapy and optical methods to provide vision where there is none.

The gene therapy allows the delivery of an “opsin” that converts second- or third-order non-light sensitive cells to become light sensitive to mimic the function of rods and cones.


Gene Therapy in Ophthalmology Update 19: A New Virus Vector for Safer Delivery of Gene Therapies Jun. 2013

Researchers at UCal Berkeley have found a gene therapy vector that can deliver genes deep into the retina via intravitreous delivery, instead of using a needle to deliver the virus sub-retinally.

This eliminates the need for a vitrectomy, anesthesia and a hospital stay to treat patients, allowing for a simple short office visit and injection into the vitreous, similar to the way anti-VEGF drugs for age-related macular degeneration are currently delivered.


Gene Therapy in Ophthalmology Update 20: Oxford BioMedica Gene Therapy Clinical Trials Resume Oct. 2013

As I have recently noted, both Oxford BioMedica and Genzyme had stopped recruiting for their respective gene therapy clinical trials this summer. Oxford announced the reason for its stoppage, but no word from Genzyme (and no response to my attempts to find out).

Well, Oxford announced today that it had resumed its clinical trial after receiving clearances from both the FDA and the French regulatory agency, ANSM.


Gene Therapy in Ophthalmology Update 21: New Gene Therapy Company, Spark Therapeutics, Launches Oct. 2013

Children’s Hospital of Philadelphia has spun out a new gene therapy company, Spark Therapeutics, that has taken over CHOP’s gene therapy programs. The new company takes over the advanced clinical trial for treating Leber’s Congenital Amaurosis, as well as an earlier stage trial for treating hemophilia B.

The Phase III clinical trial for Leber’s, is expected to be completed in mid-2015, and could become the first FDA-approved gene therapy treatment in the U.S.

Miscellaneous

A Golden Retriever Named Trevor and Retinitis Pigmentosa Apr. 2011

Recently, I encountered a unique referral source, goldenretrevor/pra-research. This piqued my curiosity and I went to the site and took a look. It turns out that the site is run by the owner of a Golden Retriever, named Trevor, along with two Labrador Retriever siblings. It seems that Trevor had been diagnosed with photo receptor cone disease (prcd), associated with progressive retinal atrophy (PRA). This was discovered when the dog was a puppy and the owner decided to look into this disease to see if there was anything that could be done to prevent him from going blind.

In doing extensive research, the owner, Katie McCormick, discovered that there was little research being done in the field of PRA in animals, but that PRA is genetically similar to retinitis pigmentosa (RP) in humans, as one study noted, "Identical mutation in a novel retinal gene causes progressive rod-cone degeneration (prcd) in dogs, and retinitis pigmentosa in man." And, there was lots of research being done on RP.

In her blog entry on PRA Research, Katie describes how she set up a “Google Alert” using the terms “progressive retinal atrophy” and “retinitis pigmentosa” – which is how she found my Journal article on The Use of Gene Therapy in Treating RP and Dry AMD.


A Novel Gene Therapy Approach to Treating the Wet Form of AMD: The BioFactoryTM From Avalanche Biotech  Feb. 2012

I originally contacted this company in November 2010, when they were still in “stealth mode” and weren't able to share details about what they were doing. Recently, the company got back in touch to provide an update, having announced, in December 2011, a clinical trial of their gene therapy approach to treating the wet form of AMD.

Since their approach is unique, and possibly “game changing” for the treatment of the wet form of AMD, I asked if I could prepare a writeup about the company and its technology for publication in my online Journal, and the co-founder and CEO Tom Chalberg agreed to answer my questions, as best as he could. So, here in their own words is what Avalanche Biotech is all about.


An Update on Avalanche Biotechnologies: A Potential Longer-Lasting Wet AMD Treatment? May 2014

With the news of a collaboration between Avalanche and Regeneron, we decided to update our initial report on Avalanche to describe what the collaboration is all about, as well as a brief update of the clinical trial underway using Avalanche’s Ocular BioFactory. Could this approach to treating wet AMD lead to fewer injections – once every 18 months or several years – in controlling this sight-robbing disease?