Freeline to Present New Clinical Data for FLT201 in Gaucher Disease in Oral Presentation at Upcoming ESGCT 30th Annual Congress
October 16 2023 - 7:00AM
Freeline Therapeutics Holdings plc (Nasdaq: FRLN) today
announced that it will present new clinical data from its ongoing
Phase 1/2 GALILEO-1 trial of FLT201, its adeno-associated virus
(AAV) gene therapy candidate, in patients with Gaucher disease Type
1 at the European Society of Gene & Cell Therapy (ESGCT) 30th
Annual Congress being held October 24-27, 2023 in Brussels,
Belgium.
The oral presentation will include data on safety, tolerability,
GCase enzyme activity, substrate, and hemoglobin and platelet
levels from the first two patients treated with FLT201.
Details of the oral presentation are below:
Presentation Title: Results from GALILEO-1, a
First-In-Human Clinical Trial of FLT201 Gene Therapy in Patients
with Gaucher Disease Type 1Presenter: Ozlem
Goker-Alpan, MD, Lysosomal and Rare Disorders Research and
Treatment CenterDate and Time: Wednesday, October
25 from 14:30-16:30 CEST (8:30-10:30 a.m.
ET)Session: 5B – Gene Therapy for Metabolic
Diseases at Clinical Stage
Additional details on the meeting can be found at the ESGCT 30th
Annual Congress website. The presentation materials will be
available on the Investors section of Freeline’s website following
presentation at the ESGCT Congress.
About Gaucher
DiseaseGaucher disease is caused by a mutation in the GBA1
gene that results in abnormally low levels of glucocerebrosidase
(GCase), an enzyme needed to metabolize a certain type of lipid. As
a result, harmful substrates glucosylceramide (Gb-1) and
glucosylsphingosine (lyso-Gb1) build up in cells that then
accumulate in various organs, causing inflammation and dysfunction.
Gaucher disease is hereditary and presents in various subtypes.
Freeline is currently focused on Gaucher disease Type 1, the most
common form of the disease, which affects the health of the spleen,
liver, bone and lung. Despite treatment with existing therapies,
many people with Gaucher disease continue to experience symptoms
and disease progression. Gaucher disease affects approximately
18,000 people in the United States, United Kingdom, France,
Germany, Spain, Italy and Israel.
About FLT201 FLT201
is an adeno-associated virus (AAV) gene therapy candidate that is
currently being investigated in the Phase 1/2 GALILEO-1 clinical
trial in adults with Gaucher disease Type 1. FLT201 is designed to
generate durable increases in glucocerebrosidase (GCase) and reduce
the accumulation of harmful substrates, with the aim of providing a
one-time treatment that can stop disease progression, improve
outcomes, and free people from lifelong treatment. FLT201 uses
Freeline’s proprietary AAVS3 capsid to introduce a novel transgene
into liver cells to produce a rationally engineered GCase variant.
In preclinical studies, the GCase variant has demonstrated a
greater than 20-fold increase in half-life at lysosomal pH
conditions compared to wildtype human GCase. Preclinically, FLT201
has shown robust GCase expression, leading to significant GCase
uptake and substrate reduction in key tissues. For more information
about the GALILEO-1 trial, please visit clinicaltrials.gov
(NCT05324943).
About Freeline
TherapeuticsFreeline is a clinical-stage biotechnology
company focused on developing transformative gene therapies for
chronic debilitating diseases. Freeline uses its proprietary,
rationally designed AAV vector and capsid (AAVS3), along with novel
promoters and transgenes, to deliver a functional copy of a
therapeutic gene into human liver cells, thereby expressing a
persistent functional level of the missing or dysfunctional protein
into a patient’s bloodstream. The company is currently advancing
FLT201, a highly differentiated gene therapy candidate that
delivers a novel transgene, in a Phase 1/2 clinical trial in people
with Gaucher disease type 1. Freeline has additional programs in
research, including one focused on GBA1-linked Parkinson’s disease
that leverages the same novel transgene as FLT201. Freeline is
headquartered in the UK and has operations in the United States.
For more information, visit www.freeline.life/or connect with
Freeline on LinkedIn and X.
Forward-Looking
StatementsThis press release contains statements that
constitute “forward-looking statements” as that term is defined in
the United States Private Securities Litigation Reform Act of 1995,
including statements that express the opinions, expectations,
beliefs, plans, objectives, assumptions or projections of Freeline
Therapeutics Holdings plc (the “Company”) regarding future events
or future results, in contrast with statements that reflect
historical facts. In some cases, you can identify such
forward-looking statements by terminology such as “anticipate,”
“intend,” “believe,” “estimate,” “plan,” “seek,” “project,”
“expect,” “may,” “will,” “would,” “could” or “should,” the negative
of these terms or similar expressions. Forward-looking statements
are based on management’s current beliefs and assumptions and on
information currently available to the Company, and you should not
place undue reliance on such statements. Forward-looking statements
are subject to many risks and uncertainties. Such risks and
uncertainties may cause the statements to be inaccurate and readers
are cautioned not to place undue reliance on such statements. The
Company cannot guarantee that any forward-looking statement will be
realized. Should known or unknown risks or uncertainties
materialize or should underlying assumptions prove inaccurate,
actual results could vary materially from past results and those
anticipated, estimated, or projected. Investors are cautioned not
to put undue reliance on forward-looking statements. A further list
and description of risks, uncertainties, and other matters can be
found in the Company’s Annual Report on Form 20-F for the fiscal
year ended December 31, 2022, and in subsequent reports on Form
6-K, in each case including in the sections thereof captioned
“Cautionary Statement Regarding Forward-Looking Statements” and
“Item 3.D. Risk factors.” Many of these risks are outside of the
Company’s control and could cause its actual results to differ
materially from those it thought would occur. The forward-looking
statements included in this press release are made only as of the
date hereof. The Company does not undertake, and specifically
declines, any obligation to update any such statements or to
publicly announce the results of any revisions to any such
statements to reflect future events or developments, except as
required by law. For further information, please reference the
Company’s reports and documents filed with the U.S. Securities and
Exchange Commission (the “SEC”). You may review these documents by
visiting EDGAR on the SEC website at www.sec.gov.Media and
Investor Contact:Naomi Aokinaomi.aoki@freeline.lifeSenior
Vice President, Head of Investor Relations & Communications+ 1
617 283 4298
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