Notice of Pre-AIA or AIA Status
The present application, filed on or after March 16, 2013, is being examined under the first inventor to file provisions of the AIA .
Applicant’s amendments and remarks filed December 2, 2025 have been received and reviewed. Claims 1, 3-7, 9-13 and 15 are now pending in this application.
Election/Restrictions
Claims 1, 3-7, 13 and 15 are under consideration.
Claims 9-12 remain withdrawn as being drawn to non-elected subject matter.
Note that compounds, corresponding compositions, a method of use and a process of making that are of the same scope are considered to form a single inventive concept under PCT Rule 13.1, 37 CFR 1.475(d). The claims are not so linked as to form a single inventive concept.
Claim Rejections - 35 USC § 112
Claims 1, 3-7, 13 and 15 are again rejected under 35 U.S.C. 112(a) or 35 U.S.C. 112 (pre-AIA ), first paragraph, as failing to comply with the enablement requirement. The claims contain subject matter which was not described in the specification in such a way as to enable one skilled in the art to which it pertains, or with which it is most nearly connected, to use the invention.
The basis of this rejection is the same as given in the previous office action and is incorporated herein fully by reference.
The claims have been amended to be directed to a method of treating a genetic disease in a subject caused by nonsense mutation selected from the group consisting of;
Cystic fibrosis due to a nonsense mutation G542X in the cystic fibrosis
transmembrane conductance regulator gene,
Duchenne muscular dystrophy due to a nonsense mutation in dystrophin,
Beta thalassemia due to nonsense mutation in ß-globin,
Niemann-Pick disease type A, B or C due to nonsense L261X mutation in acid sphingomyelinase,
Hurler syndrome,
Dravet syndrome,
Spinal muscular atrophy,
Myoadenylate deaminase deficiency,
Antithrombin III deficiency,
Alpha-1 antitrypsin deficiency,
Apolipoprotein deficiency of apolipoprotein AI, B, CII or E,
adenine phosphoribosyltransferase (APRT) deficiency,
Haemophilia A due to nonsense mutation in Factor VIII),
Haemophilia B due to nonsense mutation in Factor IX),
Von Willebrand disease due to a nonsense mutation in Von Willebrand factor,
Fanconi anemia-group C,
Marfan syndrome,
Gaucher disease,
Donohue syndrome,
Oculocerebrorenal syndrome of Lowe, and
Xeroderma pigmentosum.
Applicant’s arguments have been fully considered but not found persuasive. Gentamicin is not known to treat any of the diseases recited. In fact, no compound has ever been found that treat the diseases recited generally.
Applicants have not demonstrated nor have they alleged there is any correlation between the in vitro assays they are relying on and clinical efficacy against any disease. Case law is clear on this point. In an unpredictable art, such as genetic disease therapy, in vitro assays may be used for enablement only if there is a well-established correlation between the assay and clinical efficacy. Applicants own research articles do not go as far as the claim’s conclusion that the treatment of these diseases is enabled.
No compound has ever been found that can treat the diseases recited generally even though massive efforts have been directed towards this end. Since this assertion is contrary to what is known in medicine, proof must be provided that this revolutionary assertion has merits. Applicant is asserting that he succeeded where others have failed. Where extensive efforts have all failed, it is reasonable for the Patent and Trademark Office to require proof that the claimed invention actually works for this specific utility. It is well established that a utility rejection is proper when scope of enablement is not reasonably correlated to the scope of the claims. (In re Vaeck 20 USPQ2d 1439, 1444, In re Ferens 163 USPQ 609).
THIS ACTION IS MADE FINAL. Applicant is reminded of the extension of time policy as set forth in 37 CFR 1.136(a).
A shortened statutory period for reply to this final action is set to expire THREE MONTHS from the mailing date of this action. In the event a first reply is filed within TWO MONTHS of the mailing date of this final action and the advisory action is not mailed until after the end of the THREE-MONTH shortened statutory period, then the shortened statutory period will expire on the date the advisory action is mailed, and any nonprovisional extension fee (37 CFR 1.17(a)) pursuant to 37 CFR 1.136(a) will be calculated from the mailing date of the advisory action. In no event, however, will the statutory period for reply expire later than SIX MONTHS from the mailing date of this final action.
Any inquiry concerning this communication or earlier communications from the examiner should be directed to BRUCK KIFLE whose telephone number is (571)272-0668. The examiner can normally be reached 8 AM - 6 PM, M-F.
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If attempts to reach the examiner by telephone are unsuccessful, the examiner’s supervisor, Jeffrey H. Murray can be reached at 571-272-9023. The fax phone number for the organization where this application or proceeding is assigned is 571-273-8300.
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March 5, 2026
/BRUCK KIFLE/Primary Examiner, Art Unit 1624