Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company
committed to delivering a new class of differentiated, one-time
curative genetic therapies, today reported new preclinical data
demonstrating the ability of Prime Editors to efficiently and
precisely correct the predominant mutations that cause rhodopsin
associated autosomal dominant retinitis pigmentosa (RHO adRP). The
data were presented today at the International Symposium on Retinal
Degeneration 2023 Congress (RD2023) in Costa del Sol, Spain.
RHO adRP is a rare inherited retinal disease that causes
progressive vision loss in early adolescence, leading to eventual
blindness in adulthood due to photoreceptor degeneration. It
results from mutations in the gene RHO, which encodes rhodopsin,
the light-sensitive G protein-coupled receptor involved in
phototransduction in rods, a type of photoreceptor, and leads to
the progressive loss of rods and, subsequently, cones in the
retina.
“The data presented today are the first proof-of-concept data
for Prime Editing’s application in treating ophthalmological
indications, and highlight the ability of Prime’s novel, dual AAV
delivery platform to efficiently deliver Prime Editors to the eye,”
said Jeremy Duffield, M.D., Ph.D., Chief Scientific Officer of
Prime Medicine. “Specifically, today’s data highlight the ability
of two Prime Editors to correct the predominant mutations causing
RHO adRP – one to correct p.P23H, the most common disease-causing
mutation in RHO in the U.S., and one to correct 18 different
mutations at a mutational hotspot in RHO, including p.V345L and
p.P347L, which are the most prevalent mutations in Europe. With no
detected off-target edits in human photoreceptors and no AAV
integrations observed in these studies, today’s results are a
positive step forward for those living with RHO adRP, for whom
there are currently no approved treatment options.”
To address the predominant mutations causing RHO adRP, Prime
Medicine conducted comprehensive high-throughput screening of more
than 1,000 Prime Editor guide RNAs (pegRNAs). Two potent Prime
Editors were identified – one that precisely corrected RHO p.P23H
located near the N-terminus of rhodopsin, and one that precisely
corrected the mutational hotspot located near the C-terminus, which
includes 18 pathogenic mutations including RHO p.V345L and p.P347L.
The Company then developed and optimized a proprietary dual AAV
system to deliver the Prime Editors via subretinal injection in
humanized mouse models. Prime Editor performance was assessed in a
suite of in vitro assays and demonstrated up to 45% correction at
RHO p.P23H, and more than 70% correction at RHO p.V345L and
p.P347L. According to scientific literature and Prime Medicine
research, 25% correction at both RHO p.P23H and the C-terminal
mutational hotspot may be sufficient to halt progression of RHO
adRP, and correcting these mutations has the potential to benefit
approximately 60% of patients living with this disease.
In today’s presentation at RD2023, Prime Medicine shared
findings from in vivo studies in humanized mice with its Prime
Editors targeting these prevalent RHO mutations. Key findings from
these studies showed:
- Up to 70% precise correction in photoreceptors at RHO p.P23H
and up to 65% at RHO p.V345L or p.P347L at a mutational hotspot
using Prime Editors delivered by a dual AAV system via subretinal
injection with less than 0.5% on-target unintended edits
detected.
- Efficient delivery of Prime Editors by dual AAV to human
(retinal explants) and murine (in vivo) photoreceptors.
- RHO correction well tolerated with no detectable changes in
retinal thickness or glial fibrillary acidic protein (GFAP) gene
expression.
- No measurable integration of the AAV vector at the edit site,
as measured by one-sided polymerase chain reaction (PCR).
- No detectable off-target edits observed in human photoreceptors
following a genome-wide off-target screening analysis.
These results demonstrated that Prime Medicine’s proprietary
dual AAV system effectively delivered Prime Editors to the eye with
high efficiency and precisely corrected pathogenic mutations
causing RHO adRP at high efficiencies well above the levels
believed to have the potential to halt disease progression.
Presentation Details
- Title: Prime Editors efficiently and precisely
correct pathological mutations causing rhodopsin associated
autosomal dominant retinitis pigmentosa (adRP)
- Date: October 24, 2023
- Location: Costa del Sol, Spain
About Prime MedicinePrime Medicine is a leading
biotechnology company dedicated to creating and delivering the next
generation of gene editing therapies to patients. The Company is
leveraging its proprietary Prime Editing platform, a versatile,
precise and efficient gene editing technology, to develop a new
class of differentiated, one-time, potentially curative genetic
therapies. Designed to make only the right edit at the right
position within a gene while minimizing unwanted DNA modifications,
Prime Editors have the potential to repair almost all types of
genetic mutations and work in many different tissues, organs and
cell types.
Prime Medicine is currently progressing a diversified portfolio
of eighteen programs initially focused on genetic diseases with a
fast, direct path to treating patients or with a high unmet need
because they cannot be treated using other gene-editing approaches.
Over time, the Company intends to maximize Prime Editing’s
therapeutic potential and advance potentially curative therapeutic
options to patients for a broad spectrum of diseases. For more
information, please visit www.primemedicine.com.
Forward Looking StatementsThis press release
contains forward-looking statements within the meaning of the
Private Securities Litigation Reform Act of 1995, as amended,
including, without limitation, implied and express statements about
Prime Medicine’s beliefs and expectations regarding: the
initiation, timing, progress, and results of its research and
development programs, preclinical studies and future clinical
trials, and the release of data related thereto, the ability of
Prime Editors to correct the causative mutation of RHO adRP, its
ability to expand preclinical proof-of-concept in vivo data in
humanized mouse models, its ability to demonstrate superior
off-target profiles for Prime Editing programs for the treatment of
ophthalmological diseases, and the potential for Prime Editors to
repair genetic mutations. The words “may,” “might,” “will,”
“could,” “would,” “should,” “expect,” “plan,” “anticipate,”
“intend,” “believe,” “expect,” “estimate,” “seek,” “predict,”
“future,” “project,” “potential,” “continue,” “target” and similar
words or expressions are intended to identify forward-looking
statements, although not all forward-looking statements contain
these identifying words.
Any forward-looking statements in this press release are based
on management’s current expectations and beliefs and are subject to
a number of risks, uncertainties and important factors that may
cause actual events or results to differ materially from those
expressed or implied by any forward-looking statements contained in
this press release, including, without limitation, risks associated
with: uncertainties related to the authorization, initiation, and
conduct of preclinical and IND-enabling studies and other
development requirements for potential product candidates,
including uncertainties related to opening INDs and obtaining
regulatory approvals; risks related to the development and
optimization of new technologies, the results of preclinical
studies, or clinical studies not being predictive of future results
in connection with future studies; the scope of protection Prime
Medicine is able to establish and maintain for intellectual
property rights covering its Prime Editing technology; Prime
Medicine’s ability to identify and enter into future license
agreements and collaborations; and general economic, industry and
market conditions, including rising interest rates, inflation, and
adverse developments affecting the financial services industry.
These and other risks and uncertainties are described in greater
detail in the section entitled “Risk Factors” in Prime Medicine’s
most recent Annual Report on Form 10-K, as well as any subsequent
filings with the Securities and Exchange Commission. In addition,
any forward-looking statements represent Prime Medicine’s views
only as of today and should not be relied upon as representing its
views as of any subsequent date. Prime Medicine explicitly
disclaims any obligation to update any forward-looking statements
subject to any obligations under applicable law. No representations
or warranties (expressed or implied) are made about the accuracy of
any such forward-looking statements.
© 2023 Prime Medicine, Inc. All rights reserved.
PRIME MEDICINE, the Prime Medicine logos, and PASSIGE are
trademarks of Prime Medicine, Inc. All other trademarks referred to
herein are the property of their respective owners.
Investor ContactHannah DeresiewiczStern
Investor Relations,
Inc.212-362-1200hannah.deresiewicz@sternir.com
Media ContactDan Budwick,
1ABdan@1ABmedia.com
Prime Medicine (NASDAQ:PRME)
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