RARE Daily

Syncona Bids to Acquire Freeline

October 18, 2023

Rare Daily Staff

Syncona has made a non-binding proposal to acquire gene therapy developer Freeline Therapeutics for $5.00 per American Depository Share, according to a filing with the U.S. Securities and Exchange Commission.

Syncona, founded by Wellcome Trust, is building a portfolio of companies based on UK and EU-based research. It is the majority shareholder of Freeline with about a 58 percent stake. The deal would value the Freeline at around $21.8 million according to data on Koyfin. It’s bid represented a 20 percent premium over the volume weighted average price of $4.16 between October 4 and October 16, according to MarketWatch.

In the filing, Freeline said its board of directors has formed a special committee of independent directors of the company to explore and evaluate all strategic and financing opportunities available to the company, including Syncona’s proposal.

The company said it does not intend to comment further publicly with regards to a potential transaction unless a specific transaction is approved by the committee.

Earlier this month Freeline reported positive initial clinical data from a phase 1/2 study of its experimental gene therapy FLT201 for Gaucher disease, a lysosomal storage disorder. The data from the first two patients in the study showed that a single infusion of the gene therapy led to a several hundred-fold increase in glucocerebrosidase activity in plasma and normalization of glucocerebrosidase activity in leukocytes. Glucocerebrosidase is an enzyme needed to metabolize a certain type of lipid.

Gaucher disease is caused by a mutation in the GBA1 gene that results in abnormally low levels of glucocerebrosidase (GCase). As a result, harmful substrates glucosylceramide and glucosylsphingosine 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.

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 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. Preclinically, FLT201 has shown robust GCase expression, leading to significant GCase uptake and substrate reduction in key tissues.

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