Freeline Granted ILAP Designation in United Kingdom by MHRA for FLT201, an Investigational Gene Therapy for the Treatment of Gaucher Disease
22 5월 2023 - 9:00PM
Freeline Therapeutics Holdings plc (Nasdaq: FRLN) today announced
that FLT201 has been awarded the Innovation Passport for the
treatment of Gaucher disease type 1 under the Innovative Licensing
and Access Pathway (ILAP) process by the U.K. Medicines and
Healthcare products Regulatory Agency (MHRA). The Innovation
Passport is the first step in the ILAP process, which in
collaboration with MHRA and partner agencies, aims to accelerate
time to market and facilitate patient access to medicines in the UK
for life-threatening or seriously debilitating conditions, or
conditions for which there is a significant patient or public
health need.
“We believe FLT201 has life-changing potential for
people with Gaucher disease type 1, the most common type of the
disease,” said Pamela Foulds, MD, Chief Medical Officer of
Freeline. “There remains a significant unmet need in Gaucher
disease. Existing therapies come with a heavy life-long treatment
burden, and even with treatment, many patients continue to
experience serious symptoms. We believe that FLT201 may improve
outcomes for patients with a one-time gene therapy. We look forward
to working with MHRA to bring FLT201 to patients in the UK as
efficiently as possible.”
The Innovation Passport is granted by the UK's ILAP
Steering Group, which consists of representatives from MHRA, the
National Institute for Health and Care Excellence (NICE), the
Scottish Medicines Consortium (SMC), the All Wales Therapeutics and
Toxicology Centre (AWTTC), and the National Health Service (NHS)
England. Benefits of ILAP include opportunities for enhanced
regulatory interactions through the development and approval
process, as well as access to a range of development tools, such as
the potential for a 150-day accelerated Marketing Authorization
Application (MAA) assessment, rolling review and a continuous
benefit-risk assessment.
About Gaucher DiseaseGaucher
disease is caused by a mutation in the GBA 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
lungs. Despite treatment with existing treatments, 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, European Union and Israel.
About FLT201 FLT201 is an
adeno-associated virus (AAV) gene therapy candidate that is
currently being investigated in the GALILEO-1 Phase 1/2 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 designed GCase variant. In
preclinical studies, the GCase variant has demonstrated a 20-fold
increase in half-life at lysosomal pH conditions compared to
wild-type human GCase. Preclinically, FLT201 has shown robust GCase
expression, as well as significant GCase uptake and substrate
reduction in key tissues. FLT201 has been granted Orphan Drug
Designation for the treatment of Gaucher disease by US and EU
regulatory authorities.
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 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
Twitter.
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 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. These
statements relate to future events, including the potential of
FLT201 to be a first-in-class or life-changing gene therapy and
improve outcomes for patients with a one-time gene therapy, and
involve known and unknown risks, uncertainties and other factors
that may cause the actual results, levels of activity, performance
or achievements of the Company or its industry to be materially
different from those expressed or implied by any forward-looking
statements. 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
Freeline Therapeutics (NASDAQ:FRLN)
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