FRLN Freeline Therapeutics Holdings PLC

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