PIE POST (APVRS 2021 Edition) - DAY 2

On Spotlight at APVRS 2021 Virtual

Real World Progress on AMD and PCV

by Joanna Lee

On Day 2 of the 14th Congress of the AsiaPacific Vitreo-retina Society (APVRS 2021 Virtual), all eyes were on the choroid.

Whether through the latest imaging technology or the ultra-widefield indocyanine green angiography (UW-ICGA), researchers are observing both its role in the development of neovascular age-related macular degeneration (nAMD) and its relationship with polypoidal choroidal vasculopathy (PCV). The availability of anti-VEGF treatment has been a major step forward for treating these conditions; however, real world data is vital in the effort to translate the success of clinical trial results into real world outcomes.

3D-visualization of Haller vessels in pachychoroid

With 3D-visualization of Haller vessels in pachychoroid, choroidal imaging may never be the same again. Different from retinal vessels, choroidal vessels are challenging to observe due to shadows, their complex arrangement, and difficult vessel course delineation.

Dr. Jay Chhablani and his team has, with the help of machine learning algorithms, developed

3D imaging to view choroidal vessels. “Now we can see specific individual vessels in more detail — you can see their wall, the diameter, changes in the diameter, and if there are any focal changes,” he said, while showing the vasculature of a patient’s eye with CSCR (central serous chorioretinopathy) in 3D.

Currently, Dr. Chhablani and colleagues are working on the machine’s ability to demonstrate any nicking, constrictions and narrowing, while also making an effort toward providing more quantitative and qualitative analyses.

Pachychoroid disease and polypoidal choroidal vasculopathy

Dr. Gemmy Cheung presented her latest study, which investigated how venous overload choroidopathy may be a unifying mechanism that could explain the characteristics of pachychoroid diseases. Among pachy vessel segments in the posterior or in the peripapillary region (outer-periphery), they observed how these vessels often connect adjacent to

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vortex systems and likely represent anastomoses rather than simple dilatation.

agents like faricimab are showing some promise.

Choroidal venous insufficiency and pachychoroid disease

Ultra-widefield indocyanine green angiography (UW-ICGA) has revealed patterns of choroidal venous insufficiency which could influence pachychoroid disease. Dr. K. Bailey Freund demonstrated UW-ICGA results showing how choroidal venous insufficiency in eyes with pachychoroid disease relates to an imbalance in choroidal venous drainage.

first and largest registries of nAMD — including more than 22,000 eyes — could help doctors identify the most effective treatment regimens in the management of this disease.

Dr. Jennifer Arnold shared how eyes with moderate disease “activity” (fluid) had the best long-term visual acuity outcomes. This suggests that some fluid might protect against the development of macular atrophy (MA).

“Pachychoroid may represent a new pathogenic mechanism that is particularly relevant to Asian AMD and polypoidal choroidal vasculopathy,” she said before showing how PCV is one of the neovascular complications of the pachychoroid spectrum. The pachychoroid disease spectrum, which divulges how PCV may arise from several clinical contexts, is a part of a schema taken from her latest upcoming publication.

Challenges of anti-VEGF therapy for nAMD

Optimal treatment for nAMD requires early, proactive and sustained therapy, according to Prof. Adrian Koh. The disease is expected to increase to 288 million cases by 2040, an exponential increase from 2020’s 196 million.

Thankfully, anti-VEGF therapy has set very high bars of success in reducing blindness due to nAMD. And now, real world evidence shows that by using the Treat & Extend (T&E) protocol proactively, patient outcomes improve.

Data from the Australian Fight Retinal Blindness Registry (FBR) showed that with T&E, it’s possible to maintain good vision in the long-term with a decreasing need for injections each successive year. However, the long-term maintenance of early visual gains is dependent on injection frequency.

Moving forward, there are still barriers that need to be addressed. These include treatment burden on patients, the need for carers, stressed clinical capacity and education. Meanwhile, new therapeutic

He noted that congestion of one or more vortex vein systems is associated with regional choroidal thickening, choroidal venous hyperpermeability, and a remodelling of venous drainage routes.

Vitelliform lesions

The pachychoroid may be involved in the development of vitelliform lesions in eyes. The accumulation of yellowish sub-retinal material can occur in a variety of macular disorders; these have been referred to generically as vitelliform lesions (AVL).

Dr. Won Ki Lee said that retinal pigment epithelium (RPE) stress response to insults caused by pachychoroid (both mechanical and ischemic), may impair photoreceptor outer segment turnover, playing some role in the development of vitelliform lesions.

In his study, the most frequent accompanying abnormality is pigment epithelial detachment (PED), which occured in 40% of the patients. Separation of RPE from the underlying choriocapillaris may lead to the formation of AVL.

There are cases in which the occurrence and regression of AVL depend on the activity of a pathologic lesion association with pachychoroid. In other cases, AVL is newly developed in the background of pachychoroid-related changes.

Insights from the Fight Retinal Blindness Program

Real world (RW) outcomes from the

“It’s important to distinguish between intraretinal and subretinal fluid when managing patients via a T&E or PRN (pro re nata). Contrary to previous assumptions, optimal re-treatment regimens may involve tolerating some degree of fluid, particularly subretinal fluid. Too much or too little fluid can result in poor outcomes via MA or subretinal fibrosis.

Neo-choriocapillaris predicting atrophy in nAMD

There have been some theorized benefits of macular neovascularization (MNV), particularly that it’s protective for macular atrophy development.

However, Dr. Srinivas Sadda inquired: Why is atrophy still developing, despite MNV? And are Type 1 MNV lesions developing a surrogate for the choriocapillaris?

Dr. Sadda explained that Dr. Freund’s imaging showed a type of capillarylike neovessels, resembling the choriocapillaris (CC), on the surface of the RPE. Dr. Sadda named these “neochoriocapillaris” or “neo-CC.”

So, can neo-CC predict the development of atrophy in neovascular AMD?

Dr. Sadda’s study showed that a proportion of the MNV lesions covered by a “neo-CC” at baseline was greater among eyes that did not progress to atrophy, versus those which progressed to macular atrophy. The choriocapillaris remote from the MNV lesion (peripheral macula) was similar in both groups and showed a similar progressive decrease during the follow-up in both groups, as one would expect with age.

In summary, the neo-CC may be a useful biomarker for a protective Type 1 MNV lesion.

“Pachychoroid may represent a new pathogenic mechanism that is particularly relevant to Asian AMD and polypoidal choroidal vasculopathy,”

Dr. Gemmy Cheung

A New International Classification of ROP

by Sam McCommon

To begin tackling any problem, the problem must first be defined. That’s especially true in the sciences, where different working definitions can lead to differing approaches, skewing understanding of said problem. That’s especially super double extra true in medicine, where protocols to treat medical conditions must be developed and standardized.

So, that leads us to today’s topic presented on Day 2 of the 14th Congress of the Asia-Pacific Vitreo-retina Society (APVRS 2021 Virtual): The third edition of the International Classification of Retinopathy of Prematurity (ICROP). Dr. R.V. Paul Chan, with the University of Illinois, led us through this discussion — including why a new classification was needed in the first place.

Toward a new international classification

First, let’s start with a bit of background,

as Dr. Chan did. The first International Classification of ROP was developed in 1984 to guide management of ROP. The first working definition included zone, stage and plus disease; the 1987 version added retinal detachment; and the 2005 edition included aggressive posterior ROP, pre-plus disease and the regression of ROP.

The goal here is to help doctors report their findings to other doctors who may handle that specific infant in the future. Accurate reporting is important to making informed choices, after all.

So, why update the ICROP now? As Dr. Chan put it, “I think we can all agree that the management and diagnosis of ROP over the last 10-15 years has evolved, especially with the advent of anti-VEGF treatment and advanced imaging techniques.”

Our understanding of plus disease has also evolved, so it’s now viewed more as a spectrum than a specific condition,

including the potentiality for a severity score.

So, a group got together: 34 retinal and pediatric ophthalmologists representing six continents. If this sounds like the beginning of an adventure story, it kind of is.

What’s new about the new classifications?

The panel discussed seven different factors in ROP. These included:

1. APROP (Aggressive posterior retinopathy of prematurity)

2. ROP regression

3. ROP reactivation

4. Stage 5 (a further categorization of stage 5 ROP)

5. Persistent avascular retina and vascular anomalies

6. Intermediate zone/Posterior zone II/Notch

7. Plus disease spectrum

APROP, for example, was originally described as typically affecting the smallest premature infants. Now we’re looking more at A-ROP. In Dr. Chan’s words, “Aggressive ROP is increasingly recognized to also occur in larger preterm infants and beyond the posterior retina, particularly in regions of the world with limited resources.” Despite the P for Posterior in APROP, aggressive ROP is not limited to the posterior segment. Instead, A-ROP is more distinguished by the tempo of disease and the appearance of vascular abnormalities than by its location.

There’s much more to discuss here, but we’d like to point you to the real literature rather than gloss over it here. Since you’re reading this in an ebook or print format, a quick search for “International Classification of ROP, 3rd Edition” will yield the results you want: all of the new changes, explained in detail.

The work that has been poured into this is one of love, most certainly — for the most vulnerable among our species. Let’s keep that going.

Long-term Effects of COVID-19 on Retinal Vascular Dilation

by Sam McCommon

If you’re sick of hearing about COVID-19, well, tough. It’s still here, it’s likely an endemic disease by now, and you’re likely to be hearing about it for the rest of your life. So, we have to do the best we can to understand just how it affects all of the body — and yes, that includes the eyes. You’ve likely heard some about this, but there’s always new information coming out.

Italy’s Dr. Allessandro Invernizzi guided us through this discussion on the relationship between COVID-19 and the eyes so that we’ll all be better prepared to handle it.

Just for a rosy start, Dr. Invernizzi reminded us, “One of the most important problems with this disease is that it can induce a massive cytokine storm leading to an increased procoagulatory status, leading to the formation of blood clots and thrombotic complications including death.”

But hey, this is an ophthalmic publication covering a vitreoretinal society, so let’s cut to the chase.

COVID and the retina

As of this writing, there are more than 300 articles on PubMed linking COVID and the retina. One of the most common findings are signs of a microangiopathy, with the most common signs being cotton wool spots and intraretinal hemorrhage.

Additionally, with OCTA, you can detect an increased size of the foveal avascular zone and decreased vascular density in COVID patients. But on top of microangiopathy, there are also changes to larger blood vessels.

Most notably, major blood vessels, especially veins, were enlarged in COVID patients, explained Dr. Invernizzi. Interestingly, both arteries and veins around the optic nerve head were increased in size in COVID patients compared to controls, and the dilation of the vessels was directly correlated with the severity of the disease. Just for reference, this is after having taken into account all of the conditions that could have led

to increased severity for the disease. Simply put, COVID dilates blood vessels in the retina.

So, were these changes acute or were they long-lasting? That’s what Dr. Invernizzi and his team set out to learn.

The results were nuanced and eyebrowraising. Patients with mild COVID had blood vessels that had returned to their original size, whereas patients with severe COVID still had enlarged blood vessels six months after all other systemic inflammatory problems were back to normal. Again, the severity of the disease is directly correlated to the dilation of the blood vessels.

Effectively, the diameter only returns to normal in mild cases, suggesting longlasting structural alteration to retinal vessels in severe COVID-19.

So, what will this mean for the field of ophthalmology? Time will tell, but patients who have suffered from severe COVID will have to deal with the fallout of this structural vascular damage for a long time — though exactly how long is anyone’s guess. Doctors should be well aware of this and keep it in mind when treating patients even months after all other systems are back to normal. We’re still in the Wild West when it comes to the results of this one, folks. Thanks to Dr. Invernezzi for the discussion.

Ozurdex Wins for Early Treatment of DME and RVO

by Joanna Lee

The number of adults (aged 20-79) affected by blindness from diabetic macular edema (DME) is expected to double by 2050, according to the International Diabetes Federation. To best protect sight and prevent worsening visual acuity, early intervention is crucial. Sponsored by Allergan, an AbbVie company, a key symposium on Day 2 of the 14th Congress of the Asia-Pacific Vitreo-retina Society (APVRS 2021 Virtual), saw experts weighing in on management strategies and treatment decisions for these patients.

Practical management of DME from recent real-life studies

Using optical coherence tomography (OCT) to check the performance of patients’ eyes after receiving dexamethasone implants has changed how Prof. Matias Iglicki manages DME, he said.

To predict which DME patients would

most benefit from an early start, or an earlier treatment regimen switch, Dr. Iglicki and team conducted several real-life studies, ultimately finding four predictive biomarkers which tend to indicate good outcomes.

OCT showed the absence of disorganization of retinal inner layers (DRIL) resulted in better visual outcomes. Eyes with subretinal fluid and intact IS/ OS (inner segment/outer segment) fared better; eyes without hyperreflective foci also gained better vision outcomes.

Suitable naive versus pretreated DME patients

Understanding the pathophysiology of the disease and how intravitreal drugs play a role in the pathway of the disease is vital, according to Dr. Javier Zarranz Ventura.

Anti-VEGF drugs work very well, but about one-third of patients respond insufficiently, according to the Protocol I and T studies. For these patients,

dexamethasone may provide different reactions.

After studying 203 eyes treated with Ozurdex (Allergan, an AbbVie company, Dublin, Ireland), Dr. Ventura said that treatment naive eyes’ visual acuity (VA) fared better at 24-month follow-up. “The data also tells us that if we start the treatment of these eyes with Ozurdex earlier, we could perhaps [get a] better response and need a lower number of injections,” he said.

While assessing the suitability of patients for treatment, it is important to take into account the patients’ phakic status, intraocular pressure, cardiovascular risk factors, social factors and OCT patterns. “During the pandemic, Ozurdex can also lower the number of visits for treatment, an important point that must be considered,” added Dr. Ventura.

Switching early in DME

A study by Dr. Jorge Ruiz Medrano

and his team has uncovered better functional results in patients with insufficient response to anti-VEGF who switched at 3 injections, compared with those who switched after more than 3 anti-VEGF injections (as measured by best corrected visual acuity (BCVA) at 12 months). Both groups showed a reduction in central retinal thickness (CRT) of more than 30%.

The results seem to be confirmed in a study by Martinez et. al., where patients who were switched early to Ozurdex benefited in terms of BCVA, compared to patients who did not switch early.

Just how safe is Ozurdex?

Prof. Laurent Kodjikian said safety data related to Ozurdex do indicate side effects like ocular hypertension (OHT), cataract, cardiovascular (CV) risk and endophthalmitis. However, these side effects are highly manageable.

In the SAFODEX study, among the 120 OHT cases, topical treatment alone

INDUSTRY UPDATE

was sufficient (97%). This result was also similar in the SAFODEX 2 study and the pivotal MEAD study, with 99% of patients’ IOP brought under control. SAFODEX 2 analysis also showed that repeat injections of Ozurdex neither increased nor decreased the risk of OHT. SAFODEX 1 revealed that it is safe to reinject a patient who had presented with OHT after Ozurdex. For cataract eyes, the MEAD study indicated it is safe to be injected at 6-month intervals.

According to many pivotal studies, there is no increase of CV risk in Ozurdex, said Prof. Kodjikian. In patients with DME and history of macular edema, Ozurdex is an appropriate option. And although data from studies have demonstrated a certain risk of endophthalmitis, the numbers are not strong representatives. “What is important is that the burden of DME is lower with steroids than with anti-VEGF based on injection numbers,” he said, adding that there are three times fewer average injections with steroids compared with anti-VEGFs.

Ozurdex in RVO

Finally, in retinal vein occlusion (RVO), venous obstruction followed by retinal ischemia will trigger excessive VEGF production. The inflammation occurs early on in the disease.

Drawing from his 12 years of specializing in retinal and ocular inflammation in Lausanne, Switzerland, Prof. Marc de Smet said we should consider the use of steroids as first-line therapy for RVO patients.

He demonstrated evidence from the GENEVA and China Registration studies (both RCTs) and COBALT study, as well as real-world data in the Canadian CHROME study, support the early use of Ozurdex in RVO for BCVA improvement. Prof. de Smet specified that patients “with a recent history of a major cardiovascular event, patients with pseudophakic eyes, and those who are unwilling to come for monthly injections and monitoring in the first six months” are suitable candidates.

Part B of Phase 2 KALAHARI Study Underway

40-50% of DME patients who respond suboptimally to anti-VEGF therapy.

The company shared that the primary objective of Part B of the study is to “assess the difference in treatment effect between the selected dose from Part A of THR-149 (0.13mg) and aflibercept from baseline to month 3, in terms of increase in best-corrected visual acuity (BCVA), the primary endpoint.”

Oxurion NV (Leuven, Belgium) recently announced that the first patient has been dosed in Part B of its two-part Phase 2 KALAHARI study, which is evaluating multiple administrations of THR-149 versus

aflibercept for treating DME. THR-149 acts through inhibition of the plasma kallikrein-kinin (PKaI-Kinin) system, a validated VEGF-independent target for DME treatment. The drug is being developed to treat the approximately

“The success of this study would pave the way to a pivotal trial, that if successful, could position THR-149 to capture an important share of and expand the $4.5 billion DME market, which at the moment is almost entirely served by anti-VEGF therapies,” said Tom Graney, CFA, CEO of Oxurion, in a press release.

Results from Part B of this study are expected mid 2023.

Trauma Symposium

Great Name, Greater Content

by Andrew Sweeney

“What’s in a name?” you might ask. It’s a fair question, as naming customs are remarkably important, whether that be personally or professionally, and they all tell a story. The writer of this article’s name, for example, is as common as muck where he’s from and quickly identifies him as being of a Celtic background (and likely a Celtic supporter). When it comes to ophthalmology however, we’re less interested in ethnicity and soccer, and more in cold hard medical facts.

Which makes the name of one particular symposium at the 14th Asia-Pacific Vitreo-retina Society Congress (APVRS 2021 Virtual) really stand out. Rapid Fire Eye Trauma, Emergencies & Infections, Ocular Oncology & Pathology, Intraocular Inflammation, Uveitis & Scleritis, Translational Medicine, Ocular Imaging Session is certainly a mouthful — but it tells the story about an online session that was jam-packed full of ophthalmic information. When you have a name or title that’s so comprehensive, it’s hard

not to get curious about the content you know is coming.

Uveal melanoma: It’s in the presentation

With a symposium name that long, it won’t come as much surprise that this particular symposium had a large number of speakers and presentations. The first, and one of the highlights, was Patients Presenting with Metastases: Stage IV Uveal Melanoma, an International Study by Dr. Gaurav Garg, New York Eye Cancer Center, USA. He reported on his work to analyze ocular and systemic findings of patients with both uveal and metastatic melanoma at first presentation.

Dr. Garg studied 3,610 patients worldwide who he split into two groups: Group M with stage IV disease at presentation; and Group N at stages I-III, with no metastasizes. He found that in Group M, 80% had choroidal melanoma, 16% had ciliary body melanoma, and 4% had iris

melanoma. In Group N, 87% had choroidal melanoma, 8% had ciliary body melanoma, and 45% had iris melanoma.

Using deep learning for AMD screening

Age-related macular degeneration (AMD) is one of the most common diseases an ophthalmologist can encounter, so we recommend you check out Validation of an Automated, Offline Deep Learning-Based Tool to Screen for Age-Related Macular Degeneration by Dr. Divya Rao, medical director of Remidio Innovative Solutions (Bangalore, India). She reported on how improving offline screening for AMD will help improve access to vital treatment in remote areas. Her study involved boosting the limited data collected by one fundus camera device (deployable via a smartphone) by incorporating a larger dataset captured from a different device.

Dr. Rao’s study found that it was possible to achieve reasonable accuracy on the target device by incorporating data from the supplementary device. This also applied to image sensitivity, reporting that “acceptable sensitivity and specificity” was observed via the clinical support system. In conclusion, she stated that offline deployment of a portable fundus camera can be used to improve access to screening and that smartphone deployment can provide AMD screening to scale.

Improving access to healthcare in rural areas is an awesome aim.

Gene Therapy Showing Promise in Pediatric Patients

by Nick Eustice

Gene therapy has been a hot topic at the 14th Congress of the Asia-Pacific Vitreo-retina Society (APVRS 2021 Virtual), and a lot of exciting news has been revealed about the first application of new treatments for genetic diseases.

During Pediatric Retina: Epidemiology, Screening, Diagnosis and Treatment symposium on the Day 2 of the Congress, Dr. Audina Berrocal reported some very encouraging results from treating her pediatric patients with Luxturna (Spark Therapeutics, Philadelphia, Pennsylvania, USA) at the Bascom Palmer Eye Institute in Miami, Florida.

Luxturna (voretigene neparvovec) made major news in December 2017 when it was the first gene therapy approved by the FDA in ophthalmology. It is used to treat a genetic mutation in the RPE65 gene, commonly known as Leber Congenital Amaurosis. This rare condition’s onset is in infancy, and occurs in 2 to 3 out of every 100,000 babies. It causes autosomal recessive (AR) retinal degeneration, leading to nystagmus, amaurotic pupils, nyctalopia and severe visual loss.

Dr. Berrocal’s presentation, entitled Gene Therapy for Pediatric RPE 65

LCA: The Bascom Palmer Experience, detailed some of her observations from

the Institute’s first administrations of Luxturna in young patients. Her lecture spanned the course of these treatments thus far, beginning with her reflections on administration of the new therapy, including improvements made upon the methods used during clinical trials, and leading to some video clips showing just how effective this treatment can be in partially restoring the sight of several young patients.

Surgical improvements

The primary evolution of the Luxturna administration procedure, which Dr. Berrocal has come to recommend, is the use of the MicroDose Injection Kit. In clinical trials, the retinal injection was done manually, using a syringe. Over the course of her experience at Bascom Palmer, Dr. Berrocal has come to prefer the precision offered by this device. Attached directly to the vitrectomy machine, it can be controlled with the machine’s foot pedal with a greater degree of precision and stability.

Use of the MicroDose injector in this context requires the creation of a prebleb prior to the administration of the gene therapy. On the topic of blebs, Dr. Berrocal reminded us that the bleb should not be too high, and that it is far better to create two blebs rather than one very

high one. She recommends the use of intraoperative OCT to evaluate the height of the bleb. “Blebs have a mind of their own,” she said, “and will go wherever they want to go.”

In terms of the locus of treatment, Dr. Berrocal has learned that it is beneficial to treat the macula — while not necessarily detaching the fovea — despite the trials’ recommendation to do so. She has also found that the treatment should be done nasally. In all cases of treatment, this has been found to enlarge the visual field of the patients.

Striking results

At Bascom Palmer, 11 children (aged 1219) have been treated with Luxturna. Dr. Berrocal says that the best improvements have been seen in the younger patients. Though some improvements are stronger than others, she has received positive reports of results from parents within 24 hours following treatment in every one of these cases.

“They always text me,” Dr. Berrocal remarked, “and they tell me, ‘I think they’re needing less light’ — something almost unbelievable, as it’s been consistently true.” In all cases, there has been noticeable improvement in one area or another. Many have reported

subjective improvement in nystagmus and autofluorescence. Though only two patients were given electroretinogram (ERG) examinations, both showed significant improvements in voltage. Patients also all showed a substantial improvement in color vision.

To demonstrate these improvements, Dr. Berrocal closed with several striking video examples documenting patients’ visual improvements. The first showed two panels of a young boy, attempting to navigate a playground. The first of these showed him walking around while covering his recently operated eye. With the use of only his untreated eye, he proceeded slowly and carefully, often bumping into obstacles. In the second video, with both eyes open, the boy was seen running around the playground at a

INDUSTRY UPDATE

comfortable speed, with full confidence in his ability to avoid obstacles.

In the next video, a young girl was shown accurately shooting a target with an air rifle at a considerable distance. The parents had reported that merely seeing the target through a scope was beyond the realm of possibility for her previously, but the video showed that she had hit a well-grouped number of shots on target.

Dr. Berrocal closed by describing her experience with these results: “I will tell you honestly that as a physician and as a surgeon, this has been one of the most wonderful experiences I have had, dealing with these patients and these families. Honestly, what gene therapy is doing is almost a miracle, and this is only the beginning. We have much to learn!”

Rayner acquires Omidria, expanding surgical offerings in USA and Europe

Rayner Surgical Group (Worthing, U.K.) recently announced that it has received ophthalmology assets from Omeros Corporation, including Omidria™ — a drug that prevents miosis in cataract surgery and reduces postoperative pain. Rayner expects this transfer to expand its commercial, regulatory and marketing infrastructure in the U.S., with a broad range of products, including intraocular

lenses (IOLs), for cataract surgeons.

Approved in 2015 by the FDA, Omidria has become a key product in surgeons’ arsenals. It has also received EMA approval for marketing in Europe.

“We work closely with Rayner on their advanced technology IOLs and I also worked on the original clinical studies of Omidria. It’s great to see this product

coming to Europe and is consistent with Rayner’s vision to provide superior visual outcomes,” said Dr. Allon Barsam, an ophthalmologist in London, U.K., in a press release.

The transaction is expected to complete by Dec. 31, 2021.

“I will tell you honestly that as a physician and as a surgeon, this has been one of the most wonderful experiences I have had, dealing with these patients and these families. Honestly, what gene therapy is doing is almost a miracle, and this is only the beginning. We have much to learn!”

Dr. Audina Berrocal