Freeline to Present New Data on its Gaucher and Parkinson’s Disease Programs at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting
25 April 2024 - 6:00AM
Freeline Therapeutics today announced the acceptance of multiple
abstracts, including one for a late-breaking oral presentation on
new clinical data from its Gaucher disease program, at the upcoming
American Society of Gene and Cell Therapy (ASGCT) 27th Annual
Meeting taking place May 7-11, 2024 in Baltimore, Maryland.
“Gaucher disease is a severe and progressive disorder that leads
to a wide range of symptoms, including enlarged organs, bone pain
and fractures, low blood counts and chronic fatigue, and many
patients continue to experience debilitating symptoms despite
treatment with currently approved therapies,” said Michael Parini,
Freeline’s Chief Executive Officer. “FLT201, our investigational
gene therapy for Gaucher disease, has the potential to deliver
better efficacy with a one-time treatment, and we are excited to
present new clinical data from our ongoing Phase 1/2 trial in a
late-breaking oral presentation at ASGCT. We will also be sharing
new data from our Parkinson’s disease research program, which
builds on our work in Gaucher disease by leveraging the enhanced
stability of our rationally engineered GCase enzyme. Together,
these presentations highlight our approach of optimizing every
component of our product candidates to unlock the true potential of
gene therapy.”
Details of the presentations are below:
Oral Presentation Title: Results from
GALILEO-1, a First-In-Human Clinical Trial of FLT201 Gene Therapy
in Patients with Gaucher Disease Type 1Presenter:
Dr. Ozlem Goker-Alpan, Lysosomal and Rare Disorders Treatment
CenterSession Date and Time: Thursday, May 9, 2024
8:15-8:30 a.m. EDTSession Title: Late-Breaking
Abstracts ISession Room: Ballroom
3Late-Breaking Abstract Number: 2
Poster Presentation Title: GBA1-Linked
Parkinson’s Disease AAV Gene TherapyPresenter: Su
Kit Chew, PhD, Freeline TherapeuticsSession Date and
Time: Thursday, May 9, 2024 12:00-7:00 p.m.
EDTSession Title: Neurologic
DiseasesPresentation Room: Exhibit
HallFinal Abstract Number: 1117
Additional details on the meeting can be found at the ASGCT 27th
Annual Congress website. The presentation materials will be
available on the News & Events section of Freeline’s website
following presentation at the ASGCT Annual Meeting.
About FLT201FLT201 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 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 GBA1-linked Parkinson’s
DiseaseParkinson’s disease (PD) is a progressive
neurodegenerative disorder that results in tremors, muscle
rigidity, difficulty walking, anxiety, depression and cognitive
impairments. Approximately 5-15% of PD patients have mutations in
the GBA1 gene, which encodes for the glucocerebrosidase
(GCase) enzyme. The most common genetic risk factor for
PD, GBA1 mutations increase the risk of developing PD by
5- to 30-fold. GBA1 mutations are also associated with
earlier onset and more severe disease. There are no approved
disease-modifying therapies for PD, and current treatments, which
focus on managing symptoms, become less effective over time.
Freeline estimates GBA1-linked PD affects approximately 190,000
patients in the United States, United Kingdom, France, Germany,
Spain and Italy.
About Freeline
TherapeuticsFreeline is a clinical-stage biotechnology
company focused on developing transformative gene therapies for
chronic debilitating diseases. Freeline 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.
Media Contact:Naomi
Aokinaomi.aoki@freeline.life+ 1 617 283 4298
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