Editas Medicine Presents Preclinical Data on EDIT-103 for Rhodopsin-associated Autosomal Dominant Retinitis Pigmentosa at the Association for Research in Vison and Ophthalmology Conference
May 04 2022 - 1:30PM
Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing
company, today announced ex vivo and in vivo preclinical data for
its experimental medicine EDIT-103 for the treatment of
rhodopsin-associated autosomal dominant retinitis pigmentosa
(RHO-adRP). The Company reported these data in an oral presentation
today at the Association for Research in Vison and Ophthalmology
(ARVO) Annual Meeting in Denver.
EDIT-103 is a mutation-independent CRISPR/Cas9-based, dual AAV5
vectors “knockout and replace” (KO&R) therapy to treat
RHO-adRP. This approach has the potential to treat any of over 150
dominant gain-of-function rhodopsin mutations that cause RHO-adRP
with a one-time subretinal administration.
“These promising data in non-human primates and mice demonstrate
the potential of EDIT-103 to efficiently remove the defective RHO
gene responsible for RHO-adRP, and, critically, replace it with a
sufficient level of RHO to preserve photoreceptor structure and
functions,” said Mark S. Shearman, Ph.D., Executive Vice President
and Chief Scientific Officer, Editas Medicine. “The program is
progressing toward the clinic, and we expect to start IND-enabling
studies by the end of 2022. EDIT-103, which uses a dual AAV gene
editing approach, also provides initial proof of concept for the
treatment of other autosomal dominant disease indications where a
gain of negative function needs to be corrected, and we are
evaluating the development of additional programs.”
Key findings include:
- In human retina explants, EDIT-103 demonstrated highly specific
editing with no off-target editing observed after
transduction.
- In a mRhohRHO/+ mouse model, EDIT-103 achieved rapid gene
editing, with maximal levels at six (6) weeks and sustained, stable
editing until end of study (13 weeks).
- In non-human primates (NHPs), EDIT-103 demonstrated nearly 100%
knockout of endogenous RHO, and the replacement RHO gene produced
over 30% of normal RHO protein levels in the treated area of
subretinal injection.
- The EDIT-103 (KO&R)-injected eyes of NHPs showed
restoration of RHO expression in the outer segments and retention
of normal photoreceptor structure and function compared to the
KO-injected eye.
Presentation Session Information: |
|
Presentation Title: A Mutation-Independent
CRISPR/Cas9-based ‘Knockout and Replace’ Strategy to Treat
Rhodopsin-Associated Autosomal Dominant Retinitis Pigmentosa |
|
Session
Title: Gene therapy and other novel therapeutics in
ophthalmic diseases 2 |
|
Session Date and
Time: May 4, 2022, from 10:00 a.m. to 12:00 p.m. MDT |
|
Presentation
Time: May 4, 2022, 11:08 a.m. MDT (1:08 p.m. EDT) |
|
Presenter:
Zoe (Chi-Hsiu) Liu, Senior Scientist I, In Vivo Gene Editing,
Editas Medicine |
|
Location: Denver Convention Center, 4AB Mile
High Ballroom |
Full details of the Editas Medicine presentations can be
accessed in the Posters & Presentations
section on the Company’s website.
About EDIT-103EDIT-103 is a CRISPR/Cas9-based
experimental medicine in preclinical development for the treatment
of rhodopsin-associated autosomal dominant retinitis pigmentosa
(RHO-adRP), a progressive form of retinal degeneration. EDIT-103 is
administered via subretinal injection and uses two adeno-associated
virus (AAV) vectors to knockout and replace mutations in the
rhodopsin gene to preserve photoreceptor function. This approach
can potentially address more than 150 gene mutations that cause
RHO-adRP.
About Editas MedicineAs a leading genome
editing company, Editas Medicine is focused on translating the
power and potential of the CRISPR/Cas9 and CRISPR/Cas12a genome
editing systems into a robust pipeline of treatments for people
living with serious diseases around the world. Editas Medicine aims
to discover, develop, manufacture, and commercialize
transformative, durable, precision genomic medicines for a broad
class of diseases. For the latest information and scientific
presentations, please visit www.editasmedicine.com.
Forward-Looking StatementsThis press release
contains forward-looking statements and information within the
meaning of The Private Securities Litigation Reform Act of 1995.
The words "anticipate," "believe," "continue," "could," "estimate,"
"expect," "intend," "may," "plan," "potential," "predict,"
"project," "target," "should," "would," and similar expressions are
intended to identify forward-looking statements, although not all
forward-looking statements contain these identifying words.
Forward-looking statements in this press release include statements
regarding the Company’s expectation to start IND-enabling studies
for EDIT-103 by the end of 2022. The Company may not actually
achieve the plans, intentions, or expectations disclosed in these
forward-looking statements, and you should not place undue reliance
on these forward-looking statements. Actual results or events could
differ materially from the plans, intentions and expectations
disclosed in these forward-looking statements as a result of
various factors, including: uncertainties inherent in the
initiation and completion of preclinical studies and clinical
trials and clinical development of the Company’s product
candidates; availability and timing of results from preclinical
studies and clinical trials; whether interim results from a
clinical trial will be predictive of the final results of the trial
or the results of future trials; expectations for regulatory
approvals to conduct trials or to market products and availability
of funding sufficient for the Company’s foreseeable and
unforeseeable operating expenses and capital expenditure
requirements. These and other risks are described in greater detail
under the caption “Risk Factors” included in the Company’s most
recent Annual Report on Form 10-K, which is on file with
the Securities and Exchange Commission, and in other filings
that the Company may make with the Securities and Exchange
Commission in the future. Any forward-looking statements
contained in this press release speak only as of the date hereof,
and the Company expressly disclaims any obligation to update any
forward-looking statements, whether because of new information,
future events or otherwise.
Contacts:
Media
Cristi Barnett
(617) 401-0113
cristi.barnett@editasmed.com
Investors
Ron Moldaver
(617) 401-9052
ir@editasmed.com
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