We are a clinical-stage biotechnology company engineering purpose-built in vivo
CRISPR technologies designed to extend healthy lifespan through disease
prevention and durable therapeutic intervention. While current genetic medicines
are largely limited to rare disorders, we are engineering our technologies for
use in common diseases affecting millions. By targeting prevalent diseases with
significant unmet need and high clinical burden, we aim to usher in a new era of
broadly scalable, transformative, and preventative genetic medicines. Our focus
is on cardiovascular and metabolic diseases with initial programs to address the
key drivers of atherosclerotic cardiovascular disease, or ASCVD. We are
designing CRISPR-based genetic medicines to be well-tolerated, effective,
durable, and scalable enough to shift the treatment paradigm from symptom-driven
intervention and chronic care to population-level prevention, with the goal of
broadly democratizing access to the cardioprotective effects of known genetic
variants. Our lead product candidate, STX-1150, utilizes ELXR, our highly
engineered epigenetic silencing technology, and is designed to deliver
persistent and potent LDL-C reductions without permanent genetic changes.
STX-1150 is a demonstration of moving towards our goal to transform practical
therapeutic adherence and real-world medical outcomes for patients in the
multibillion-dollar LDL-C lowering landscape. We have secured regulatory
clearance from the Australian Therapeutic Goods Administration, or TGA, for and
initiated a first-in-human clinical trial of STX-1150 in Australia in up to 64
adults with elevated LDL-C and increased risk of ASCVD. We anticipate reporting
initial data from this trial, including safety, tolerability, and LDL-C-lowering
activity, in the first half of 2027.
Every 40 seconds, someone in the United States suffers a heart attack, and each
year, heart disease costs the nation more than $400 billion, according to the
American Heart Association. Despite major advances in our understanding of the
pathology of heart disease and ASCVD, and the development of new classes of
pharmaceuticals, we believe todayβs standard of care for ASCVD is insufficient.
Existing treatments fail to demonstrate broad real-world impact due to
limitations in efficacy, well-documented side effects, and the burden of chronic
polypharmacy, which all contribute to poor uptake, low adherence, and limited
persistence. Similarly, patients often discontinue therapy due to the lifelong
burden of taking medication, loss of insurance, high out-of-pocket costs, or the
logistical burden of ongoing clinic visits. Moreover, treatment is often
initiated only after substantial cumulative arterial injury or an acute
cardiovascular event. These limitations of the current treatment paradigms
underscore the importance of developing effective and durable therapies that can
be administered safely early in the course of disease, reduce the barriers to
treatment continuation for patients, and provide compelling health-economic
value to payors.
We believe that a fundamentally different approach is required to meaningfully
treat or prevent ASCVD. We must create practical, optimized therapies that
remain effective even when used early in disease progression. To realize this
vision, we are executing a differentiated strategy that applies a full-stack
engineering approach coupling generative artificial intelligence and machine
learning, with massively parallel experimental validation to optimize and tailor
CRISPR technologies for the precise demands of each therapeutic application.
Through this proprietary approach, which we refer to as CRISPR by Design, we
systematically evaluate disease biology, target genetics, and commercial
opportunities against product requirements to engineer the most appropriate and
scalable solution for each therapeutic application. This disciplined,
data-driven strategy has led us to engineer two proprietary technologies based
on a novel CRISPR-CasX enzyme: the Epigenetic Long-Term X-Repressor, or ELXR,
designed for precise, durable epigenetic silencing without altering the
underlying DNA sequence; and the X-Editor, or XE, designed for specific and
efficient gene editing. When developing a program, rather than committing to a
single technology upfront, we advance multiple approaches in parallel and
prioritize the modality that demonstrates the most favorable safety, durability,
and efficacy profile for each product candidate based on empirical data. We
believe this engineering-led, evidence-based strategy, combined with a
technology stack purpose-built for broad patient populations, positions us to
develop competitive and scalable genetic medicines across a wide range of
cardiovascular indications. We are advancing three in vivo product candidates
targeting three key lipid drivers of ASCVD: elevated low-density lipoprotein
cholesterol, or LDL-C, elevated lipoprotein(a), or Lp(a), and elevated
triglycerides carried by triglyceride-rich lipoproteins, or TRLs.
Our lead product candidate, STX-1150, is an epigenetic silencing therapy that is
based on our ELXR technology and is designed to durably lower LDL-C by
repressing the expression of PCSK9, a genetically and clinically validated
target. Inhibition of PCSK9 is among the most effective known mechanisms to
reduce LDL-C, complementing or outperforming other existing therapies. Unlike
CRISPR gene editing, base editing, or prime editing approaches, STX-1150 is
designed to achieve long-lasting therapeutic benefit without permanently
altering underlying DNA sequence. STX-1150 aims to improve on the real-world
efficacy of small-molecule, antibody, and siRNA therapies by eliminating the
need for years to decades of chronic medication. In a study conducted with
non-human primates, or NHPs, a single dose of a prototype STX-1150 was generally
well-tolerated and produced therapeutically meaningful, durable LDL-C reduction
of greater than 50% sustained for two years. Durable and early LDL-C lowering of
this magnitude has been demonstrated in human genetic studies to reduce ASCVD
risk by up to 88%, underscoring the potential to transform the current treatment
paradigms from late-stage intervention to effective prevention. The large and
expanding LDL-C lowering market is characterized by a persistent gap between
efficacy observed in controlled settings and real-world outcomes. We believe
STX-1150βslong-acting epigenetic mechanism could increase current adherence
rates that are typically only 40β50%, which could drive superior clinical
outcomes for patients through increased adherence-adjusted efficacy and
compelling health-economic value for payors while avoiding the risks of
permanent genetic modification. Given that ASCVD is a chronic condition, we
believe providing patients with improvements in practical adherence could
compound over time to drive better clinical outcomes, enabling more efficient
payor management and broadening patient access. As a result, we believe STX-1150
has the potential to expand the growing market for PCSK9-based LDL-Cβlowering
medicines, which currently exceeds $5 billion annually yet remains significantly
underpenetrated, with fewer than 1% of eligible patients treated. We have
secured regulatory clearance from the Australian TGA for and initiated a
first-in-human clinical trial of STX-1150 in Australia in up to 64 adults with
elevated LDL-C and increased risk of ASCVD. We anticipate reporting initial data
from this trial, including safety, tolerability, and LDL-C-lowering activity, in
the first half of 2027.
Our next programs, STX-1200 and STX-1400, target two additional lipid drivers of
ASCVD, elevated Lp(a) and severely high triglycerides, by editing the LPA and
APOC3 genes, respectively. Elevated Lp(a) and high triglycerides represent key
risk factors for ASCVD affecting large populations. Both programs are based on
our XE technology and aim to provide curative solutions for genetic diseases
associated with the modification of these targets. In mouse models of disease,
prototype versions of both STX-1200 and STX-1400 programs have achieved greater
than 90% reduction in target gene expression with durable effect consistent with
a one-time treatment profile. In NHPs, surrogates demonstrated greater than 95%
Lp(a) reduction and greater than 75% APOC3 on-target editing. In an off-target
analysis of primary human hepatocyte donor cells, no detectable off-target
editing was observed even at supersaturating doses. We have been awarded grant
funding from the California Institute for Regenerative Medicine, or CIRM, of up
to approximately $25.7 million to support the development and manufacturing
activities for our STX-1200 and STX-1400 programs. With CIRM funding support, we
anticipate continuing to advance both the STX-1200 and STX-1400 programs through
preclinical development to enable the initiation of a Phase 1 clinical trial for
one of these programs as early as 2027 and one in 2028.
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Our disciplined operating model, complemented by upfront and milestone payments
from our strategic collaborations and grants, has allowed us to operate with a
high degree of capital efficiency. We believe our CRISPR by Design approach
enables us to advance target concepts to development candidates quickly, with
the potential to materially reduce discovery timelines and associated costs. Our
strategic collaborations β including with Sanofi in rare genetic diseases, such
as sickle cell disease, and Lilly in neurological and neuromuscular disorders β
leverage the commercial and delivery expertise of leading biopharmaceutical
companies while demonstrating the versatility of our technologies. Since our
founding in 2017, we have raised approximately $150.0 million in equity
financing, with only one dilutive financing in the past four years, while
maintaining significant ownership and optionality across both our internal
program portfolio and our collaboration programs.
Since the groundbreaking discovery of CRISPR-based genome editing by our
co-founder, Nobel Laureate Dr. Jennifer Doudna, the extraordinary potential for
genetic medicine has become increasingly clear. We were founded on the thesis
that comprehensive CRISPR system engineering and optimization are required to
address the activity, specificity, and delivery limitations of earlier
technologies. We believe our engineering-first philosophy has enabled us to
become one of the only companies that has created two distinct, novel and
therapeutically relevant CRISPR technologies derived from a unique CRISPR enzyme
foundation. This foundation, together with our enabling engineering, supports a
robust intellectual property portfolio. Our deliberate focus on cardiovascular
and metabolic diseases further differentiates us by driving the creation of
technologies designed to address some of the most prevalent and debilitating
conditions worldwide.
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We were incorporated under the laws of the State of Delaware on June 30, 2017.
Our principal executive offices are located at 1150 Marina Village Parkway,
Alameda, California 94501, and our telephone number is (510) 626-8587. Our
website address is https://www.scribetx.com.
