Sunday, February 26, 2012

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

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.

The Company

Founded in 2006, as an outgrowth of co-founder Thomas Chalberg’s PhD work in genetics while at Stanford University (along with pioneering work with gene therapy at Prof. Elizabeth Rakoczy’s lab at the Lions Eye Institute in Perth, Australia), Avalanche Biotech is a privately held biotechnology company that develops technologies and products for sustained delivery of therapeutic proteins to the eye to treat wet age-related macular degeneration (AMD), as well as other ophthalmic disorders, such as diabetic retinopathy, retinal degeneration, and glaucoma.

The company was incubated at the Berkeley Entrepreneurship Laboratory at the UC Berkeley Haas School of Business in 2010-2011 while Tom was completing his MBA at that school. The company set up offices in San Francisco’s SOMA district, near the UCSF Mission Bay Campus, in July 2011.

The company’s lead product treats wet AMD by using the body's own cells to produce therapeutic proteins on an ongoing basis after only a single injection. The goal is effective long-term treatment of wet AMD without the burden of frequent, ongoing intra-ocular injections.

In June 2011, Avalanche Biotechnologies licensed additional intellectual property from the University of California, Berkeley, property related to novel adeno-associated virus (AAV) vectors for use with the company's proprietary BioFactoryTM drug delivery platform. A BioFactory™ is a long-term ocular drug delivery technology that secretes a therapeutic protein over years following a single eye injection. The company’s intellectual property includes access to a pipeline of next-generation vectors for non-invasive drug delivery to the retina.

In November 2011, Avalanche began a collaboration with Lions Eye Institute in Perth to commercialize AAV-based approaches, developed using the company’s proprietary BioFactory™ drug delivery platform.

The People – Founders and Management Team

Thomas W. Chalberg, PhD
Co-Founder & Chief Executive Officer

Dr. Chalberg, earned his PhD in Genetics from Stanford University where, as a Howard Hughes Medical Institute Fellow, he focused on retinal diseases and novel technologies for gene therapy. He was interested in retinal diseases, including retinal degenerations and age-related macular degeneration (AMD). During his PhD program at Stanford, he worked with Dr. Mark Blumenkranz, his committee member and a co-founder of Avalanche Biotech, on creating a sustainable solution for sustained delivery of therapeutic proteins to the retina. They were joined by Mitchell Finer PhD, a biotechnology industry expert with experience in gene therapy, and Steven Schwartz MD, an expert in retinal disease and early-stage clinical development.

Prior to joining Avalanche, Dr. Chalberg worked on the ophthalmology team at Genentech, helping to launch Lucentis (ranibizumab by injection), a novel therapeutic for age-related macular degeneration. Dr. Chalberg holds an A.B. from Harvard University, where he graduated magna cum laude and Phi Beta Kappa. He earned a PhD in Genetics from the Stanford University School of Medicine and an MBA from the UC Berkeley Haas School of Business. Tom is a member of the Board of Visionary Scientists for Hope for Vision, a non-profit charity supporting vision research.

Dr. Chalberg joined Avalanche Biotechnologies, Inc. on a full-time basis as President and Chief Executive Officer on October 11, 2010.

Mark S. Blumenkranz, MD
Co-Founder & Director

Dr. Blumenkranz is an ophthalmologist and trained vitreoretinal surgeon at the Byers Eye Institute at Stanford University, where he has served as Chairman of Ophthalmology since 1997. Prior to that, he served on the faculty of the Bascom Palmer Eye Institute in Miami, Florida, and as founder and Director of the Vitreoretinal Fellowship Program at William Beaumont Hospital in Royal Oak, Michigan – two of the top retinal training programs in the United States. His primary clinical interests center on medical and surgical treatment of vitreoretinal diseases, with a specific emphasis on macular problems. Dr. Blumenkranz was also a founding member of the Eyetech Pharmaceuticals Scientific Advisory Board and a founder or director at several Silicon Valley-based biotech startups, including Oculex Pharmaceuticals, MacuSight, Optimedica, and PEAK Surgical. Mark received his B.A. and Master of Medical Science in Biochemical Pharmacology of Brown University and M.D. degree at Brown University followed by a Residency in Ophthalmology at Stanford.

Steven D. Schwartz, MD
Co-Founder & Director

Dr. Schwartz is an ophthalmologist and trained vitreoretinal surgeon at the UCLA Jules Stein Eye Institute, where he serves as Director of the Retina Service. At UCLA, he has served as principal investigator in a number of early-stage clinical trials for retinal diseases, including the initial studies for ranibizumab (Lucentis) and novel products in gene and cell therapy. Dr. Schwartz has held various key positions at Eyetech Pharmaceuticals, and has served on a number of Scientific Advisory Boards, including Genentech, Ophthotech, Optos, and Optimedica. Steve received his B.A. from UC Berkeley and his M.D. from USC School of Medicine, followed by a Residency in Ophthalmology at UCLA and vitreoretinal fellowship at Moorefield's Eye Hospital in London.

Mitchell H. Finer, PhD
Co-Founder & Senior Consultant

Dr. Finer has over 25 years of experience in biotechnology, building and managing companies from discovery through market launch, in addition to pioneering the development of human monoclonal antibodies, and cell and gene therapies. Currently, Dr. Finer serves as a Senior Consultant to Avalanche and is the Chief Scientific Officer of bluebird bio. Previously, he served as senior vice president of development and operations for Novocell (now Viacyte), a stem cell engineering company researching treatments for diabetes and other chronic diseases. Dr. Finer has served as CEO of Intracel Holdings LLC and Genteric Inc., and as vice president of research and development for the Gencell division of Aventis Pharma (now Sanofi). He was also a founder and vice president of research for Cell Genesys Inc., and a founder of Abgenix. Mitch received a B.S. in biochemistry and molecular biology from the University of California at Berkeley and a Ph.D. in biochemistry and molecular biology from Harvard University. He completed a postdoctoral fellowship at the Whitehead Institute for Biomedical Research.

Advisory Boards

On October 1, 2010, Avalanche announced the founding members of its Scientific and Clinical Advisory Board. The Scientific Advisory Board is chaired by Dr. Elizabeth Rakoczy, Winthrop Professor of Molecular Ophthalmology at the Lions Eye Institute, University of Western Australia. Joining her is Dr. Mitchell Finer, Chief Scientific Officer of bluebird bio and a co-founder of Avalanche.

The Clinical Advisory Board will be chaired by Dr. Ian J. Constable, Founder and Professor at the Lions Eye Institute, University of Western Australia. Joining him is Dr. Mark S. Blumenkranz, Chairman of Ophthalmology at the Byers Eye Institute at Stanford, Dr. Steven D. Schwartz, Chief of the Retina Division at the UCLA Jules Stein Eye Institute, Dr. Judy Gordon, a clinical regulatory consultant with extensive experience in ophthalmology products, and Dr. Steven Butler, Consulting Senior Biostatistician.

On September 1, 2011, Avalanche announced the addition of three renowned scientific leaders in academia and industry to its Scientific Advisory Board: Dr. Jean Bennett, F.M. Kirby professor of ophthalmology at the University of Pennsylvania; Dr. Estuardo Aguilar-Cordova, CEO of Advantagene; and Dr. J. Fraser Wright, Director of the Clinical Vector Core at Children's Hospital of Philadelphia. They will join an august group of colleagues on Avalanche's Scientific and Clinical Advisory Boards.

The Technology

The Avalanche Approach

Currently, wet age-related macular degeneration is treated with frequent injections of an anti-VEGF protein directly into the vitreous of the eye, requiring frequent office visits to assess progress and for retreatment injections with the drug, as many as 8-12 retreatments per year. This creates a burden for both patients and physicians, and limits access for those who are not able to comply with frequent visits and injections.

Avalanche’s lead product is AVA-101, currently in clinical testing for the treatment of wet AMD. Following a single injection, AVA-101 creates an Ocular BioFactoryTM that continuously secretes a therapeutic protein over an extended period, avoiding the need for frequent intraocular injections of recombinant anti-VEGF protein. The therapeutic protein is a potent inhibitor of vascular endothelial growth factor (VEGF), a clinically validated target in wet AMD. Drug delivery technology for the Ocular Biofactory™ has been pioneered under the leadership of Elizabeth P. Rakoczy, Winthrop, Professor of Molecular Ophthalmology at the Lions Eye Institute. As of mid-December 2011, Phase I/II clinical trials are in progress to evaluate safety and efficacy of a single subretinal injection of AVA-101 into eyes of patients with exudative age-related macular degeneration (wet AMD).

The Ocular BioFactoryTM

Avalanche has developed a proprietary drug delivery system which uses the body's own cells to produce therapeutic protein on an ongoing basis after only a single injection This, in effect, creates a "BioFactoryTM" in the patient's eye to treat wet AMD.

The Ocular BioFactoryTM

How it Works

Avalanche delivers its therapeutic treatments through the use of a vector made from a small biological nanoparticle called Adeno-Associated Virus (AAV). Humans are commonly exposed to AAV without any known safety issues or association with disease. AAV-based therapies are in development for a wide range of diseases including heart failure, Parkinson's disease, Alzheimer's disease, and amyotrophic lateral sclerosis (ALS), among others, along with a litany of retinal diseases in addition to the treatment of AMD.

To create a safe therapeutic vector, viral genes are removed from AAV and replaced with specific genes encoding a therapeutic protein that specifically treats AMD. AAV can infect a variety of retinal cell types and remain stable, resulting in long-term therapeutic protein expression in a variety of retinal cell types. In clinical studies, AAV appears safe and well-tolerated when injected into the retina.

The Avalanche Difference

As opposed to the current standard of care, which is effective for a short period of time, the Ocular BioFactoryTM continues to express therapeutic protein, gathering momentum and reaching peak expression after 4-6 weeks. At that point, the treatment continues to be maintained on an ongoing basis. The result is a continuous, steady-state level of therapeutic protein, which eliminates the need for frequent re-injections. Based on preclinical studies, the therapeutic effect will be maintained for at least 18 months and has the potential to last for several years following treatment from a single injection.(1)


Avalanche collaborates with leading academic institutions and pharmaceutical companies to develop and market its drug delivery platforms. We are looking for strategic partnerships with companies focused on ophthalmology and drug delivery for eye disease.

Our Partners Include:

Lions Eye Institute

In 1975 the Lions Save-Sight Foundation (LSSF) established the Lions Chair in Ophthalmology at the University of Western Australia (UWA). Professor Ian Constable AO was appointed to this position and subsequently established the Lions Eye Institute (LEI). As LEI's inaugural Managing Director, Professor Constable spearheaded its efforts to eradicate blindness worldwide. LEI conducts first class scientific research into blindness and incorporates one of Australia's largest ophthalmic practices. The Institute also houses the Lions Eye Bank and the LSSF.

As noted above, Avalanche announced its collaboration with the Lions Eye Institute in November 2011, and its first human clinical trial is being undertaken at the institution.

Merck Sharp & Dohme Corporation

Merck (known as MSD outside the United States and Canada) is a global healthcare leader working to help the world be well. Through prescription medicines, vaccines, biologic therapies, and consumer care and animal health products, Merck works with customers and operates in more than 140 countries to deliver innovative health solutions.

The therapeutic protein used in the Ocular BioFactoryTM is exclusively licensed by Avalanche from Merck Sharp & Dohme Corp, formerly Merck & Co., Inc.

The Clinical Trial

The initial clinical trial, Safety and Efficacy Study of rAAV.sFlt-1 in Patients With Exudative Age-Related Macular Degeneration (AMD), NCT01494805, is currently recruiting participants at the Lions Eye Institute, in Perth, Western Australia, and was established on December 14, 2011.

It is a Phase I/II controlled dose-escalating trial to establish baseline safety and efficacy of a single subretinal injection of AVA-101 (rAAV.sFlt-1) into eyes of patients with exudative age-related macular degeneration.

The study will involve 24 patients aged 65 or above who have wet AMD. The patients will be radomized to receive one of two doses of the agent (16 patients) or assigned to the control group (8 patients). Patients in all three groups will be eligible for rescue therapy with ranibizumb (Lucentis). The study is estimated to be completed in three years (December 2014), with a primary completion date of December 2012 (i.e., final data collection for primary outcome measurement). (For more information, please follow the NCT hyperlink above.)

The Future

The company is involved in researching several other therapeutic proteins for the following indications, according to the Clinical Trials and Milestones graphic shown below:

AVA--201 – is in research for the treatment of specific cells in the retina, including possible treatment for rare genetic diseases.

AVA--301 – is in research for the treatment of diseases associated with photoreceptor cells.

Editor’s Comments – As I noted in my introduction, if this approach is successful, it could result in a “game change” in the way wet AMD is treated. Instead of multiple injections to gain control of neovascularization, one injection at the first signs of neovascularization could stop it dead in its tracks and the effect could last, potentially, for several years.

In addition, it is conceivable that patients at risk of developing wet AMD could receive a therapeutic injection of AVA-101 to prevent the occurrence of wet AMD! This would be a definite paradigm shift in the treatment of AMD.

With the human clinical testing now underway, we should have an indication of initial results with this unique approach within about a year.

Stay tuned!

For more on the technical aspects of the company’s technology, please see reference 2.


1. AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness ; Bennett, J., Ashtari, M., Wellman, J., Marshall, K. A., Cyckowski, L. L., Chung, D. C., McCague, S., et al.; (2012). Science translational medicine, 4(120), 120ra15–120ra15. doi:10.1126/scitranslmed.3002865

2. Preclinical safety evaluation of subretinal AAV2.sFlt-1 in non-human primates; Lai, et al, Gene Therapy, (10 November 2011) | doi:10.1038/gt.2011.169

Tuesday, February 21, 2012

AMD Update 17: Dame Judi Dench Has Macular Degeneration

Over the weekend, several articles appeared in the British press and also here in the States, about Dame Judi Dench announcing that she has macular degeneration – dry in one eye and wet in the other – and that she was going blind, although she was receiving treatment for the wet AMD in the hopes of preserving some of her sight in that eye.

I considered writing about this, but couldn’t find the right words – then I read what my friend Steve Rose of the Foundation Fighting Blindness had written on his blog, Eye on the Cure – and realized that he had the story just right.

So, with his permission, here is what he wrote:

Dr. Stephen Rose
Chief Research Officer
Foundation Fighting Blindness

Feb 20, 2012 05:30 pm

She's best-known, these days, as "M," director of the British Secret Service, in the last half-dozen James Bond films. But at 77, actress Judi Dench didn't earn the title Dame Commander of the Order of the British Empire solely for turning the notion of "Bond girl" on its head. In fact, she earned that title back in 1988, after delivering what was already a career-and-a-half's worth of stage and film performances, in everything from Shakespeare to the musical Cabaret. Since then, she's worked non-stop, playing, among other notable roles, Queens Elizabeth (Shakespeare in Love) and Victoria (Mrs. Brown) on film and starring in a long-running BBC hit comedy, As Time Goes By.

So it was with sadness that this past weekend I read the wire reports that Dame Judi has age-related macular degeneration, otherwise known as AMD. She reportedly can't read scripts anymore, nor can she see clearly the faces of fellow actors and, more important, family and friends.

That sadness, however, is mixed with hope. And those of you acquainted with the Foundation probably know why.

AMD is a retinal degenerative disease that affects the macula - the part of the retina that allows us to see fine detail. Worldwide, AMD is the most common cause of blindness in those 50 and older. More than 1.6 million in England are affected, and roughly 10 million people in the United States either have AMD or are at risk for developing it.

It's no surprise that Dame Judi mentioned, in an interview, that her mother had AMD, seeing as Foundation-funded researchers discovered that genetics appears to be a major factor in more than half of AMD cases.

Dame Judi also mentioned having both kinds of AMD - wet and dry - one in each eye. Dry AMD, which accounts for 90 percent of all cases, is caused by the accumulation of protein- and fat-containing "drusen," or waste deposits. It's true that drusen can become apparent with age and not cause vision loss, and it usually takes many years for a person to lose vision to dry AMD. People with dry AMD don't usually experience a total loss of central vision, but they do lose significant ability to read, drive and do work that requires seeing fine detail (like Dame Judi). And they are at greater risk for developing the more severe wet form.

Wet AMD is the one which causes more vision loss. It's "wet" because abnormal blood vessels grow beneath the macula, leaking blood and fluid and doing damage to photoreceptor cells. Wet AMD often progresses rapidly and can cause substantial loss of central vision.

So, why am I hopeful? Well, Ms. Dench did mention she's getting injections, and that they may be helping stop the vision loss. Although the article didn't go into detail, there are now a few treatments available to people with wet AMD - Lucentis and, more recently, a drug called Eylea, are FDA-approved here in the United States, and some ophthalmologists are using Lucentis' cousin, Avastin - developed to treat colon cancer - off label. All these were made possible by Foundation-funded studies supplying research leading to their development. Essentially, these treatments dry up those blood vessels, halting vision loss and, in some cases, even restoring some vision for people with wet AMD.

One last detail that's not surprising, considering Dame Judi's longevity in a career usually not kind to women of certain ages: her pluck. She says she has every intent to continue working, as people can read her scripts and directors can make accommodations for her on film. At the Foundation, and at AMD Alliance International, of which we're a member, we have seen that same kind of determination in many people with AMD, including those I spoke with a couple weeks ago during a seminar in Florida. These are resourceful, optimistic and determined people who continue to inspire us in our search for treatments and cures for all retinal diseases.

Dame Judi was quoted as saying she has no plans to retire soon. We, at the Foundation, certainly hope not, and we look forward to many more great performances.

Monday, February 20, 2012

Stem Cells in Ophthalmology Update 17: Recent ACT Updates

While I was out of the country on vacation, Advanced Cell Technology issued four news releases about their ongoing human embryonic stem cell trials. Here is a compilation of what transpired during my absence.

Jan. 25, 2012 -- ACT Announces that Additional Patient with Stargardt's Disease Has Undergone Embryonic Stem Cell Transplantation at UCLA's Jules Stein Eye Institute

Advanced Cell Technology announced the dosing of an additional patient in its Phase 1/2 trial for Stargardt's macular dystrophy using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs). The patient was treated on Tuesday (Jan. 24) 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. The patient successfully underwent the outpatient transplantation surgery and is recovering uneventfully.

"This is the fourth patient worldwide treated with ACT's hESC-derived RPE cells," said Gary Rabin, chairman and chief executive officer of ACT. "We are pleased to be on schedule with our clinical programs to test the safety and tolerability of ACT's stem cell-derived RPE cells. We are working with the best ophthalmology institutes to evaluate the capacity of our cell therapy which may have the ability to treat a variety of devastating diseases."

The clinical trial will enroll 12 patients each, with cohorts of three patients each in an ascending dosage format. It is a prospective, open-label study, designed to determine the safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation into patients with Stargardt's macular dystrophy at 12 months, the study's primary endpoint. The patient, a 47-year-old male, was treated with the smallest dose of 50,000 cells.

A third Phase1/2 trial for Stargardt's macular dystrophy was recently initiated at the Moorfields Eye Hospital in London, treating the first patient on Jan. 20.

Jan. 30, 2012 – ACT Announces Aberdeen Royal Infirmary in Scotland as Additional Site for Phase 1/2 Clinical Trial Using hESC-Derived RPE Cells for Macular Degeneration

Advanced Cell Technology announced that the Aberdeen Royal Infirmary, the largest of the Grampian University Hospitals in Scotland, has been confirmed as a site for its Phase 1/2 human clinical trial for Stargardt's Macular Dystrophy (SMD) using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs). The Phase 1/2 trial is a prospective, open-label study designed to determine the safety and tolerability of the RPE cells following sub-retinal transplantation into patients with SMD.

"A leading medical institution in the United Kingdom, Aberdeen Royal Infirmary is an ideal partner for our European clinical trial for SMD," said Gary Rabin, chairman and CEO of ACT. "Moreover, we are particularly pleased that the lead investigator is Dr. Noemi Lois, a leading expert in SMD. We continue to forge ties with some of the best eye surgeons and hospitals in the world and work towards bringing this cutting-edge therapy closer to fruition. Our preliminary results to date keep us optimistic that we are on the right path both in terms of our science and the clinical team we are working with, particularly eye surgeons such as Dr. Lois."

This approved SMD clinical trial that Dr. Lois and her team will participate in is 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 is similar in design to the FDA-cleared US trial initiated in July 2011.

"It is an honor to have been designated as a site for this path-breaking clinical trial," said Noemi Lois, M.D., Ph.D. "We could not be more pleased to be a part of this trial for a promising potential new treatment for SMD, using hESC-derived RPE cells." Dr. Lois is a is a member of the Department of Ophthalmology, NHS Grampian, and associated to the University of Aberdeen, Scotland, United Kingdom. Dr. Lois practices at the Aberdeen Royal Infirmary; she is an Ophthalmologist with special interest in Medical retina and Retinal surgery.

Editor’s Note: I am in the process of ascertaining whether Aberdeen Royal Infirmary will be operating under the European NCT that Moorfields is operating under, or the U.S. NCT which UCLA and Wills Eye Institute operate under. I expect it is the former.

Feb. 13, 2012 – ACT Announces Third Patient with Stargardt's Disease Treated in U.S. Clinical Trial with RPE Cells Derived from Embryonic Stem Cells

Final Patient from First Cohort in Phase I/II Trial Using hESC-Derived RPE Cells

Advanced Cell announced the dosing of a third patient in its Phase I/II trial for Stargardt's macular dystrophy (SMD) using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs). The patient was treated on Monday (Feb. 6) by Steven Schwartz, M.D., Ahmanson Professor of Ophthalmology at the David Geffen School of Medicine at the University of California, Los Angeles (UCLA) and retina division chief at UCLA's Jules Stein Eye Institute. The outpatient transplantation surgery was performed successfully and the patient is recovering uneventfully.

"With the treatment of this third Stargardt's patient at Jules Stein Eye Institute, we have now completed the treatment of the first cohort of patients under our clinical protocol for phase I/II of our U.S. SMD trial," said Gary Rabin, chairman and chief executive officer of ACT. "We will continue to regularly monitor the three SMD patients in this trial, and by early spring anticipate review of their progress and safety-related data by the Data and Safety Monitoring Board (DSMB). With approval of the DSMB, we would then advance to the next cohort of patients and administer a higher dosage of RPE cells. In the context of all three trials we have running, this patient is the fifth person worldwide to be treated with our hESC-derived RPE cells. To date, there have been no complications or side effects due to the RPE cells, and we remain cautiously optimistic that our ongoing clinical programs will demonstrate the safety and tolerability of ACT's stem cell-derived RPE cells."

Feb. 15m 2012 – ACT Announces Approval of Wills Eye Institute as an Additional Site for Stem Cell Clinical Trial for Stargardt's Disease

Leading Eye Institute Will Participate as Site for Both of ACT's Phase I/II Clinical Trials Using Human Embryonic Stem Cell-Derived RPE Cells for Macular Degeneration

Advanced Cell announced that the Wills Eye Institute in Philadelphia has received institutional review board (IRB) approval as a site for the company's Phase I/II clinical trial for Stargardt's Macular Dystrophy (SMD), a form of juvenile macular degeneration, using human embryonic stem cell (hESC)-derived retinal pigment epithelial (RPE) cells. Earlier this year, the Company also announced that the IRB at Wills Eye Institute had approved the participation of the institution as a site for ACT's clinical trial for dry age-related macular degeneration (dry AMD).

"We thank Wills Eye Institute once more for providing their IRB and their invaluable contribution to our macular degeneration studies," said Gary Rabin, ACT's chairman and CEO. "We are very happy that we can now report that Wills Eye Institute has been approved as a clinical trial site for both our SMD and dry AMD clinical trials. Ranked as one of the best ophthalmology hospitals in the country by U.S. News & World Report, the Wills Eye Institute is a truly world-class institution. Our team is eagerly anticipating working with Dr. Carl Regillo, a renowned retinal surgeon and director of clinical retina research at Wills Eye Institute, as well as a professor of ophthalmology at Thomas Jefferson University, along with the rest of his team as we move forward with these ground-breaking trials."

Specific patient enrollment for both trials at the Wills Eye Institute will be determined in the near future. Further information about patient eligibility for the SMD study and the concurrent study on dry AMD is also available on; Identifiers: NCT01345006 and NCT01344993.