Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company
committed to delivering a new class of differentiated one-time
curative genetic therapies, today announced the presentation of new
preclinical data that further demonstrated the potential of Prime
Editing to correct the causative mutation of chronic granulomatous
disease (CGD) and preclinical data that showcased the potential
application of the Prime Editing Assisted Site-Specific Integrase
Gene Editing (PASSIGE™) platform to generate multiplex-edited CAR-T
cells for the treatment of certain cancers and immune diseases. The
data are being presented today during the American Society of Gene
and Cell Therapy (ASGCT) 26th Annual Meeting, being held May 16-20,
2023, in Los Angeles, California.
“We are very pleased to present these new data for our CGD
program and PASSIGE platform today at ASGCT, which underscore our
belief in the breadth and potential of Prime Editing to offer
curative treatments for many diseases,” said Jeremy Duffield, M.D.,
Ph.D., Chief Scientific Officer of Prime Medicine. “Our CGD program
is progressing well, and with today’s data demonstrating the
reproducibility of PM359 to correct the disease-causing mutation in
CD34+ cells ex vivo with no off-target editing detected in the
comprehensive set of studies done to date, we look forward to the
PM359 program’s advancement through Investigational New
Drug-enabling studies. Further, while the benefits of autologous
CAR-T therapies are well established, their full potential is often
hindered by manufacturing and delivery challenges. With our PASSIGE
platform, we believe we can create allogeneic products that may
overcome these challenges with a one-step, non-viral approach that
could expand the applicability of T cell therapies for the
potential treatment of tumors and immune diseases.”
CGD Presentation Highlights CGD is a rare
inherited disease that leads to recurrent, debilitating and often
life-threatening infections. It is caused by mutations in genes,
including NCF1, that encode proteins that form NADPH oxidase, an
enzyme that kills bacteria and fungi to control infection. Prime
Medicine is advancing an ex vivo Prime Editing program that aims to
correct the disease-causing mutation in NCF1 in CGD patient CD34+
hematopoietic stem cells (HSCs) and restore NADPH oxidase function.
Prime Medicine has previously shared data from the CGD program that
demonstrated the ability of Prime Editing to correct a CGD
causative mutation in CD34+ cells ex vivo. The Prime Edited CD34+
cells engrafted long-term in vivo with editing levels greater than
92%. Today’s findings added to that, showing:
- Prime Editing was highly reproducible, demonstrating greater
than 90% Prime Editing in CD34+ cells from each of four donors
- 16-week engrafted Prime Edited CD34+ cells repopulated the bone
marrow, reconstituted production of human blood cells, and
biodistributed to the spleen and peripheral blood
- Comprehensive off-target analyses demonstrated no detected
off-target activity, large deletions or translocations in Prime
Edited CD34+ cells
- These findings provide further support for the advancement of
the company’s first development candidate, PM359, as a potential
treatment for CGD
PASSIGE Presentation HighlightsPrime Medicine
is advancing a platform technology known as PASSIGE, which combines
Prime Editing with an integrase or site-specific recombinase enzyme
to enable the introduction of large-sized cargo into the genome as
a potential one-time therapy. This approach is designed to expand
the versatility of Prime Editing with the intent to broaden the
range of permanent genomic edits that Prime Editing can make to
potentially treat disease, including the ability to insert, delete
or invert gene-sized pieces of DNA. In today’s presentation, Prime
Medicine highlighted expanded work using PASSIGE technology and a
non-viral approach to generate CD19 CAR-T cells, as well as robust
disruption of relevant target genes (TRAC and B2M) using Prime
Editing in primary human T cells. Results showed:
- Single-step PASSIGE-mediated insertion of a CD19-CAR at the
TRAC genetic locus in primary human T cells led to greater than 90%
loss of T cell receptor expression and 60% targeted integration of
a 3.5 kb CD19 CAR transgene, with no observed impact on T cell
viability or T cell expansion
- PASSIGE-generated CD19 CAR-T cells exhibited potent anti-tumor
activity in vitro and in vivo
- Prime Editing of the B2M gene in primary human T cells led to
greater than 90% knock-out of B2M protein expression
- Efficient multiplex Prime Editing at three genomic target sites
in primary human T cells
- These results support the potential of PASSIGE and Prime
Editing to provide a modular, one-step system to create
best-in-class, potent and targeted, allogeneic CAR-T cell
therapies
Presentation Details Abstract
Title: (101) Prime Editing of Human CD34+ Long-Term
Hematopoietic Stem Cells Precisely Corrects the Causative Mutation
of p47phox Chronic Granulomatous Disease and Restores NADPH Oxidase
Activity in Myeloid ProgenyDate & Time:
Wednesday, May 17, 2023, 5:15 – 5:30 p.m. PTRoom:
Room 515 ABSession Title: Genome Editing Therapies
& Safety IPresenter: Jennifer Gori
Abstract Title: (602) An All-Prime Editing
One-Step Approach for Non-Viral Generation of a Multiplex-Edited
Allogeneic CAR-T Cell ProductDate & Time:
Wednesday, May 17, 2023, 12:00 p.m. PTSession
Title: Wednesday Poster SessionPresenter:
Emily Pomeroy
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.
Cautionary Note Regarding 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, including the initiation of IND-enabling studies for
PM359, the potential of PM359 to correct the causative mutation of
CGD, the capacity of its PASSIGE technology to edit CAR-T cells for
the treatment of certain cancers and immune 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 other development requirements for
potential product candidates, including uncertainties related to
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 Quarterly Report on Form 10-Q, 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.
Investor ContactHannah DeresiewiczStern
Investor Relations,
Inc.212-362-1200hannah.deresiewicz@sternir.com
Media ContactDan Budwick,
1ABdan@1ABmedia.com
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