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SEPTEMBER 2020 | EYEWORLD | 31
replace" strategy to treat autosomal dominant
retinitis pigmentosa.
Gene therapy to treat more commonly
occurring wet AMD is in development as well.
Dr. Ciulla said REGENXBIO is developing
a ranibizumab-like anti-VEGF protein for
subretinal delivery, and Adverum is creating
an aflibercept-like anti-VEGF for intravitreal
delivery. For dry AMD, Hemera Biosciences
is developing HMR59 to produce the protein
CD59 that blocks the final step of the comple-
ment cascade, Dr. Ciulla explained.
What's in the pipeline?
There are several companies working on gene
therapies for recessive disorders. Dr. Ciulla
listed achromatopsia (AGTC, MeiraGTx),
Stargardt disease (Oxford Biomedica/Sano-
fi), and Usher syndrome (Oxford Biomedica/
Sanofi) as under clinical investigation. He also
said gene augmentation is being studied for
X-linked recessive disorders, such as choroider-
emia (Biogen, Spark Therapeutics) and retinitis
pigmentosa (AGTC, Biogen, MeiraGTx), and
mitochondrially inherited disorders such as
Leber hereditary optic neuropathy (GenSight).
Iveric Bio is working on a "knockdown and
continued on page 32
Relevant disclosures
Ciulla: Clearside Biomedical
Sohn: Oxford Biomedica
In some gene therapies, an adeno-associated virus (AAV) vector is used to transfer normal, correct genetic material
into patients' cells. In ophthalmology, Luxturna is the only FDA-approved gene therapy, and it uses an AAV vector to
deliver a functional RPE65 gene to retinal cells.
Source: George Church, Creative Commons Attribution-Share Alike 3.0
Unported License, commons.wikimedia.org/wiki/File:AAV_Gene_Therapy.jpg
