Emerging Gene Therapies Signal Transformative Shift in Retinal Disorder Treatment, According to Spherix Global Insights
July 24 2024 - 9:56AM
The American Society of Retinal Specialists (ASRS) annual meeting
held last week in Stockholm, Sweden, showcased groundbreaking
advancements in gene therapy for retinal disorders. The promising
data presented during the event has generated excitement among
ophthalmologists and suggests a significant transformation in the
treatment landscape for retinal diseases. Among the notable
presentations, 4D Molecular Therapeutics’ 4D-150 and Adverum
Biotechnologies’ Ixo-vec stood out with compelling phase 2 data.
These therapies demonstrated efficacy, safety and a reduction in
treatment burden, setting a new precedent for gene therapies that
maintain or improve visual acuity while significantly reducing
treatment burden along with a favorable safety profile.
These advancements signal a new era in retinal treatments,
moving away from traditional therapies. Spherix Global Insights
surveyed 77 US retinal specialists in the Special Topix™: Gene
Therapy (US) study on the evolution of the retinal landscape.
As one member of Spherix Global Insights’ Ophthalmology Community
remarked:
“I think that in five years from now, we're going to be having a
completely different conversation. I think that all these genetic
agents that are coming out, these intraocular factory agents, these
Adverum and RegenxBio agents, this is going to be the way of the
future. The eye will become its own factory for aflibercept, and we
won't be using Lucentis, Eylea, and Vabysmo anymore. They'll all
fall by the wayside.”
Furthermore, retinal specialists identified 4D-150, Ixo-vec, and
Johnson & Johnson’s JNJ-1887 as the most likely candidates for
prescription. Meanwhile, AbbVie/RegenxBio’s ABBV-RGX-314 generated
less interest due to its invasive route of administration
(subretinal or suprachoroidal injections).
The study analysis also reveals that the highest interest in
gene therapy uptake will be for retinitis pigmentosa (RP),
Stargardt disease, and geographic atrophy (GA)—conditions with
significant unmet needs for effective treatments. With regards to
age-related and diabetic retinal disease, 84% of ophthalmologists
overwhelmingly agree that a gene therapy with a durability of two
years would improve treatment outcomes, compared to a 49% agreement
for therapies with a six-month durability. Moreover, specialists
show a preference for therapies that improve visual acuity over
those that maintain it.
Despite the enthusiasm, safety concerns remain a primary barrier
to adoption. Retinal specialists are cautiously optimistic, closely
monitoring trial results for long-term consequences, administration
methods, and costs. “I’m open to gene therapy but cautious about
long-term consequences,” noted one specialist, while another added,
“I am excited about gene therapy, but I am very nervous about
potential side effects and blindness.” These statements illustrate
that while gene therapies hold revolutionary potential, patient
safety remains the top priority for ophthalmologists.
Spherix will continue to closely monitor the market to help
navigate this evolving landscape and support informed
decision-making in retinal disorder treatments.
Spherix's recent gene therapy study answers key business
questions regarding the future potential of gene therapies,
including indications and endpoints of utmost interest to retinal
specialists, their attitudes regarding the application timing and
location for gene therapy, and the educational requirements they
foresee needing from manufacturers.
Special Topix™ is an independent service that includes
access to a report or series of reports based on current events or
topics of interest in specialty markets covered by Spherix.
About Spherix Global Insights
Spherix is a leading independent market intelligence and
advisory firm that delivers commercial value to the global life
sciences industry, across the brand lifecycle.
The seasoned team of Spherix experts provides an unbiased and
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nephrology, neurology, ophthalmology, and hematology. Spherix
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extensive Spherix Physician Community.
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Spherix Global Insights Contacts
Blaine Cloud, Ophthalmology Franchise Head
blaine.cloud@spherixglobalinsights.com
NOTICE: All company, brand or product names in this press
release are trademarks of their respective holders. The findings
and opinions expressed within are based on Spherix Global Insight’s
analysis and do not imply a relationship with or endorse.
Blaine Cloud, Ophthalmology Franchise Head
Spherix Global Insights
4848794284
Blaine.cloud@spherixglobalinsights.com