Aceragen has filed a patent for a method of treating Farber disease using specific doses and pharmacokinetic profiles. The method involves administering purified recombinant human acid ceramidase (rhAC) in activated form at a dose ranging from about 1 mg/kg to about 10 mg/kg. GlobalData’s report on Aceragen gives a 360-degree view of the company including its patenting strategy. Buy the report here.
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Treatment of farber disease using purified recombinant human acid ceramidase
A recently filed patent (Publication Number: US20230123505A1) describes a method for treating Farber disease in humans. Farber disease is a rare genetic disorder characterized by the accumulation of fatty substances called ceramides in various tissues and organs. The method involves administering purified recombinant human acid ceramidase (rhAC) in activated form to the patient at a dose ranging from about 1 mg/kg to about 10 mg/kg.
The patent claims specify different dosage ranges within this range, including about 1 mg/kg to about 5 mg/kg, about 1 mg/kg, about 2 mg/kg, about 2.5 mg/kg, about 5 mg/kg, and about 10 mg/kg. The purified rhAC used in the method is referred to as RVT-801 and is produced through a process that includes chromatography steps and viral inactivation steps. The purity of the rhAC is at least 95% by weight.
The patent also mentions that the method can be used in both adult and child patients, with a dose of about 0.8 mg/kg for adults and about 1.2 mg/kg for children. Additionally, the method allows for the administration of antipyretic, antihistamine, corticosteroid, or a combination of these medications before, during, or after the administration of rhAC.
The purified rhAC can be administered in a therapeutic composition through various routes, including oral, inhalation, intranasal instillation, topical, transdermal, parenteral, subcutaneous, intravenous injection, intra-arterial injection, intramuscular injection, intrapleurally, intraperitoneally, intrathecally, or application to a mucous membrane. The method also allows for repeat administrations of the therapeutic composition.
Furthermore, the patent mentions the possibility of administering additional agents that reduce ceramide levels along with the rhAC treatment.
In summary, the patent describes a method for treating Farber disease using purified recombinant human acid ceramidase (rhAC) in activated form. The method involves administering different doses of rhAC to patients, depending on their age and weight. The purified rhAC is produced through a specific process and can be administered through various routes. The method also allows for the use of additional medications and repeat administrations of the therapeutic composition.
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GlobalData Patent Analytics tracks bibliographic data, legal events data, point in time patent ownerships, and backward and forward citations from global patenting offices. Textual analysis and official patent classifications are used to group patents into key thematic areas and link them to specific companies
