Freeline Therapeutics Holdings plc (FRLN)
First Day Turnover31.52%
We are a clinical-stage, fully integrated, next generation, systemic AAV-based gene therapy company with the ambition of transforming the lives of patients suffering from inherited systemic debilitating diseases. We aim to deliver one-time gene therapy treatments that provide functional cures through permanently sustained physiological protein levels, leveraging the high expression enabled by our proprietary gene therapy platform. Our initial focus is on developing treatments for monogenic diseases with high unmet need.
We are a fully-integrated biotechnology company with internal expertise and capability across the value chain in gene therapy, including expression platform, research, manufacturing, clinical development and commercialization. Our pipeline includes two programs in the clinic and two late-stage preclinical product candidates, as well as research programs targeting novel applications for systemic gene therapy, for which we have, through owned and in-licensed intellectual property rights, development and worldwide commercial rights.
Our differentiated platform uses our proprietary, rationally designed adeno-associated virus, or AAV, capsid, which we refer to as AAVS3. We have optimized our capsid to preferentially deliver a therapeutic gene of interest to the human liver, and thereby to cause the expression of the necessary protein to address a targeted disease. AAVS3 was developed at University College London, or UCL, by a team led by our founder, Prof. Amit Nathwani. In preclinical settings, the AAVS3 capsid has been observed to have significantly higher transduction efficiency in human liver cells as compared to wild-type AAV serotypes used in many other gene therapy programs, including AAV5, AAV6, AAV8 and others.
We are able to further enhance the potency of our AAVS3 vector platform through our advanced chemistry, manufacturing and controls, or CMC, capabilities, which include proprietary manufacturing processes and analytic methods. We believe the combination of our proprietary capsid and our powerful CMC platform allows us to produce gene therapy product candidates that have the potential to drive higher levels of protein expression than comparable programs have demonstrated in the clinic to date.
We have invested a significant amount of time and resources in developing proprietary and optimized assays and CMC processes that we believe help us to produce high quality product candidates. Today, we have a scaled manufacturing process which we believe has the ability to consistently and efficiently produce gene therapy product candidates at commercial scale and competitive cost. As a result of the differentiated potency of our platform, we are able to administer our product candidates at lower doses for a given level of protein expression, with potential benefits in safety and cost of goods. This advantage also gives us the opportunity to address conditions that are not currently amenable to gene therapy because of the high levels of protein expression required to treat such diseases. Our current pipeline programs address hemophilia A and hemophilia B as well as the lysosomal storage disorders, or LSDs, Fabry disease and Gaucher disease.
Our most advanced product candidate, FLT180a for the treatment of hemophilia B, is being evaluated in a Phase 1/2 dose-finding clinical trial in adult males, and preliminary data to date has shown its potential to provide patients with sustained normal Factor IX, or FIX, activity levels following a one-off treatment. We recently reported updated data as of June 15, 2020 from the Phase 1/2 B-AMAZE clinical trial of FLT180a for the treatment of hemophilia B at the 2020 International Society on Thrombosis and Hemostasis Congress, or ISTH 2020. We plan to report further data from this trial in due course, and to commence a planned pivotal clinical trial in 2021, subject to agreement with regulatory authorities.
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