DETAILED ACTION
Notice of Pre-AIA or AIA Status
1. The present application, filed on or after March 16, 2013, is being examined under the first inventor to file provisions of the AIA .
2. The Preliminary Amendment filed on June 9, 2023, has been received and entered.
3. Applicant’s election without traverse of Group I (with species) on October 10, 2025, is acknowledged.
Claim Disposition
4. Claims 1-18 have been cancelled. Claims 19-40 have been added and are pending. Claims 19-30 are under examination. Claims 31-40 are withdrawn from consideration as directed to a non-elected invention.
Information Disclosure Statement
5. The Information Disclosure Statement filed on June 9, 2023, has been received and entered. The references cited on the PTO-1449 Form have been considered by the examiner and a copy is attached to the instant Office action.
Abstract
6. The abstract is objected to for the following informalities:
“The present invention relates to nucleic acid molecules encoding variants of lysosomal acid lipase (LAL) and uses thereof”.
Correction is required.
Specification Objection
7. The specification is objected to for the following informalities:
The title of the invention is not descriptive. A new title is required that is clearly indicative of the invention to which the claims are directed. The following is suggested: " nucleic acid molecules encoding lysosomal acid lipase variants and uses thereof ".
The specification is objected to because it contains an embedded hyperlink and/or other form of browser-executable code. Applicant is required to delete the embedded hyperlink and/or other form of browser-executable code. See MPEP § 608.01. See page 16, for example. It is suggested that http:// is deleted.
The specification is objected to because trademarks are disclosed and they are not capitalized. The use of the trademark such as Triton X-100, has been noted in this application (see page 36, for example). It should be capitalized wherever it appears and be accompanied by the generic terminology. Although the use of trademarks is permissible in patent applications, the proprietary nature of the marks should be respected and every effort made to prevent their use in any manner, which might adversely affect their validity as trademarks”.
Appropriate correction is required.
Claim objection
8. Claims 19-30 are objected to for the following informalities:
For clarity and precision of claim language it is suggested that claim 19 is amended to recite an actual function for the chimeric LAL protein especially since modified in structure and the assertion of “functional” does not endow a function, thus should be deleted. The dependent claims hereto are also included.
For clarity and precision of claim language it is suggested that claim 29 is amended read, “A genetically modified hematopoietic stem cell, wherein the cell is modified with the insertion of the nucleic acid of claim 19…”.
For clarity it is suggested that claim 30 is amended to read, “The cell of claim 29, further comprising at least one globin locus with the nucleic acid molecule…..”.
Appropriate correction is required.
Claim Rejections - 35 USC § 112
The following is a quotation of the first paragraph of 35 U.S.C. 112(a):
(a) IN GENERAL.—The specification shall contain a written description of the invention, and of the manner and process of making and using it, in such full, clear, concise, and exact terms as to enable any person skilled in the art to which it pertains, or with which it is most nearly connected, to make and use the same, and shall set forth the best mode contemplated by the inventor or joint inventor of carrying out the invention.
The following is a quotation of the first paragraph of pre-AIA 35 U.S.C. 112:
The specification shall contain a written description of the invention, and of the manner and process of making and using it, in such full, clear, concise, and exact terms as to enable any person skilled in the art to which it pertains, or with which it is most nearly connected, to make and use the same, and shall set forth the best mode contemplated by the inventor of carrying out his invention.
9. Claims 19-30 are rejected under 35 U.S.C. 112(a) or 35 U.S.C. 112 (pre-AlA), first paragraph, as failing to comply with the written description requirement. The claim(s) contains subject matter which was not described in the specification in such a way as to reasonably convey to one skilled in the relevant art that the inventor or
a joint inventor, or for applications subject to pre-AIA 35 U.S.C. 112, the inventor(s), at the time the application was filed, had possession of the claimed invention.
The claimed invention is directed to “a nucleic acid molecule encoding a functional chimeric LAL protein comprising a signal peptide moiety and a functional LAL moiety, wherein the signal peptide moiety has an amino acid sequence selected from the group consisting of SEQ ID NOs:3, 4, and 5…. “ (see claim 19 in its entirety).
The claimed invention also encompasses a LAL moiety that is at least 75% identical to SEQ ID NO:9 and a functional signal peptide that can have deletion, insertion or substitutions of 1-5 amino acids. The recitation of the language “functional” does not actually endow activity, thus the claimed invention is devoid of an activity for the expression product. The claimed invention encompasses a lot of variability with no asserted/recited activity and encompassed unlimited variability with the open language, thus goes beyond the recited 1 to 5 amino acid change. The recitation of the language “from 1 to 5 deletion, insertion or substitution” does not actually limit the claim embodiment and could mean deletion of substantial amount of residues. The claimed invention also encompasses a large variable genus of cells, that are not adequately described. Further, the invention does not define the chimeric LAL in terms of origin and appears to retain natural parts, thus not adequately described (especially since disclosure in the specification is more geared towards foreign portions making up the chimeric LAL protein.
The claimed invention is not commensurate in scope with the disclosure and no correlation is made between structure and function (as a large variable genus of products with modifications are encompassed and not adequately described). The specification fails to provide a representative number of species for the claimed genus to show that applicant was in possession of the claimed genus. A representative number of species means that the species, which are adequately described, are representative of the entire genus.
The written description requirement for a claimed genus may be satisfied through sufficient description of a representative number of species by actual reduction to practice, disclosure of drawings, or by disclosure of relevant identifying characteristics, for example, structure or other physical and/or chemical properties, by
functional characteristics coupled with a known or disclosed correlation between function and structure, or by a combination of such identifying characteristics, sufficient to show the applicant was in possession of the claimed genus. Vas-Cath Inc. v. Mahurkar, 935 F.2d 1555, 1563-64, 19 USPQ2d 1111, 1117 (Fed. Cir. 1991), states that "applicant must convey with reasonable clarity to those skilled in the art that, as of the filing date sought, he or she was in possession of the invention. The invention is, for purposes of the ‘written description’ inquiry, whatever is now claimed" (See page 1117). The specification does not "clearly allow persons of ordinary skill in the art to recognize that [he or she] invented what is claimed" (See Vas-Cath at page 1116). The skilled artisan cannot envision the detailed chemical structure of the encompassed genus, and therefore, conception is not achieved until reduction to practice has occurred, regardless of the complexity or simplicity of the method of isolation. Adequate written description requires more than a mere statement that it is part of the invention and reference to a potential method of isolating it. The compound itself is required. See Fiers v. Revel, 25 USPQ2d 1601 at 1606 (CAFC 1993).
Therefore, for all these reasons the specification lacks adequate written description, and one of skill in the art cannot reasonably conclude that the applicant had possession of the claimed invention at the time the instant application was filed.
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.
10. Claims 19-30 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.
Claim 19 is directed to “a functional chimeric LAL protein comprising a signal peptide moiety and a functional LAL moiety….”. The term "chimeric" means that the LAL protein or the signal peptide (or both) have been engineered from different sources or modified to enhance expression, secretion efficiency, or function. The claim does not provide a source for the chimeric LAL, however, dependent claim 20 recites that it is a human LAL moiety that is optionally devoid of its natural signal peptide, therefore ambiguous since the chimeric protein is not decisively from different sources and appears to have a native signal peptide. In addition, claim 19 lacks clarity for the recitation of the word ‘functional three times in the claim, however no function was asserted. The dependent claims hereto are also included.
Claim 30 is indefinite because claim 29 from which it depends does not recite that the cell is engineered.
Conclusion
11. No claims are presently allowable, however, SEQ ID NO:21 is free of the art.
Any inquiry concerning this communication or earlier communications from the examiner should be directed to HOPE A ROBINSON whose telephone number is (571) 272-0957. The examiner can normally be reached 9-5pm on Monday to Friday.
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/HOPE A ROBINSON/Primary Examiner, Art Unit 1652