DETAILED ACTION
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 .
Priority
The instant application is a national stage entry of PCT application PCT/US2022/025208, filed 10/25/2023 under 35 USC 371. Acknowledgement is made of the applicant’s claim for benefit to prior-filed U.S. provisional patent applications 63/179,684, which was filed 04/26/2021.
Election/Restrictions
Applicant’s election of Group II, claims 54, 55, 58-60, 68, 69 and 73-75, drawn to a recombinant adeno-associated virus (rAAV), in the reply filed on 03/27/2026 is acknowledged. Because applicant did not distinctly and specifically point out the supposed errors in the restriction requirement, the election has been treated as an election without traverse (MPEP § 818.01(a)).
Accordingly, claims 54, 55, 58-60, 68, 69 and 73-75 have been considered on the merits. Claims 1, 3, 5, 7, 9, 11, 13, 15 and 18-19 are withdrawn from consideration pursuant 37 CFR 1.142(b).
Claim Objections
Claims 55, 58-60 and 74-75 are objected to because of the following informalities: Claims 55 and 74-75 recite “GM3S Ia Type 2 isoforms”, claims 58-59 recite “GM3S protein isoforms”, claim 60 recites “GM3 synthase protein isoforms”, they are recommended to amended to “GM3S Ia Type 2 protein isoforms” to be consistent with the claim language of claim 54.
Appropriate correction is required.
Claim Rejections - 35 USC § 112
The following is a quotation of 35 U.S.C. 112(b):
(b) CONCLUSION.—The specification shall conclude with one or more claims particularly pointing out and distinctly claiming the subject matter which the inventor or a joint inventor regards as the invention.
The following is a quotation of 35 U.S.C. 112 (pre-AIA ), second paragraph:
The specification shall conclude with one or more claims particularly pointing out and distinctly claiming the subject matter which the applicant regards as his invention.
Claims 58 and 75 are rejected under 35 U.S.C. 112(b) or 35 U.S.C. 112 (pre-AIA ), second paragraph, as being indefinite for failing to particularly point out and distinctly claim the subject matter which the inventor or a joint inventor (or for applications subject to pre-AIA 35 U.S.C. 112, the applicant), regards as the invention.
Claims 58 and 75 recite “a Kozak sequence (GCCACC)”, the use of parentheses renders instant claims indefinite. Since there is not only one specific nucleotide sequence for a Kozak sequence, it is not clear whether the sequence in parentheses is required or only an exemplary Kozak sequence. Therefore the scope of instant claims is not clear.
Related Prior arts
Instant claims are directed to a recombinant adeno associated virus (rAAV) comprising (i) an AAV Cap-Mac capsid protein; and (ii) an isolated nucleic acid comprising a transgene having a nucleic acid sequence encoding one or more monosialodihexosylganglioside synthase (GM3S) Ia Type 2 protein isoforms, and wherein the transgene is flanked by adeno-associated virus (AAV) inverted terminal repeats (ITRs). The closet prior arts are Naso et al. (BioDrugs. 2017 Aug;31(4):317-334) and Bowser et al. (Mol Genet Metab. 2019 Apr;126(4):475-488). Naso et al. teach the use of AAV as a vector for gene therapy (Abstract). Naso et al. teach the basic components of a gene insert packaged inside recombinant AAV gene transfer vector (p319, figure 1), which comprises two ITRs, a promoter and a gene of interest (transgene). Naso et al. also teach AAV capsid selection and optimization, states that capsid can influence cell-type transduction (see p319, right column). Naso et al. do not teach the limitations of “the transgene having a nucleic acid sequence encoding one or more monosialodihexosylganglioside synthase (GM3S) protein isoforms” as well as the capsid is “an AAV Cap-Mac capsid protein”. Bowser et al. teach GM3 synthase is encoded by ST3GAL5 and sialylateslactosylceramide (LacCer) to form GM3 (p475, right column). Mutation of ST3GAL5 cause undetectable GM3 protein in plasma (Abstract). Bowser et al. teach ST3GAL5 gene replacement holds promise as a way to reconstitute the Golgi system's endogenous biosynthetic and sorting machinery. Both lentivirus- and adeno-associated virus (AAV)-based gene vectors, delivered either systemically or intrathecally, have proven safe and relatively effective in numerous murine, large animal, and human neurogenetic disorders (p486, right column). However, there is no prior arts regarding the limitation “an AAV Cap-Mac capsid protein” in the rAAV. The earliest reference regarding AAV CAP-Mac capsid protein on record is Chuapoco et al. (BioRxiv, online on January 09, 2022, as cited in IDS), which is later than the priority date of instant invention (04/26/2021). Therefore PHOSITA would not have been taught or suggested to use the AAV CAP-Mac capsid protein in the rAAV as recited in instant claims. Instant claims are therefore considered novel and art-free.
Conclusion
Claims 54, 68-69 and 73 are allowed.
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/Q.G./Examiner, Art Unit 1633
/FEREYDOUN G SAJJADI/Supervisory Patent Examiner, Art Unit 1699
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