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 .
Status of Application, Amendments and/or Claims
The amendment, filed 08 February 2024, has been entered in full.
The amendment, filed 13 September 2024, has been entered in full. Claims 2, 3, 6-9, 11-14, 19, 21-30, 33, 38-43 are canceled. Claims 1, 4, 5, 10, 15-17, 20, 32, 34-37, are amended. New claims 44 and 45 are added.
Applicant’s species election of sickle cell disease (for the species of diseases) and the species election of antibody eculizumab (for the species of complement C5 inhibitor), in the reply filed on 04 August 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)).
Claim 10 is withdrawn from further consideration pursuant to 37 CFR 1.142(b) as being drawn to a nonelected species, there being no allowable generic or linking claim. Election was made without traverse in the reply filed on 04 August 2026.
Claims 1, 4, 5, 15-18, 20, 31, 32, 34-37, 44 and 45 are under examination.
Information Disclosure Statement
The information disclosure statement(s) (IDS) (filed 20 September 2024) was received and complies with the provisions of 37 CFR §§1.97, 1.98 and MPEP § 609. It has been placed in the application file and the information referred to therein has been considered as to the merits.
Nucleotide and/or Amino Acid Sequence Disclosures
Summary of Requirements for Patent Applications Filed On Or After July 1, 2022, That Have Sequence Disclosures
37 CFR 1.831(a) requires that patent applications which contain disclosures of nucleotide and/or amino acid sequences that fall within the definitions of 37 CFR 1.831(b) must contain a “Sequence Listing XML”, as a separate part of the disclosure, which presents the nucleotide and/or amino acid sequences and associated information using the symbols and format in accordance with the requirements of 37 CFR 1.831-1.835. This “Sequence Listing XML” part of the disclosure may be submitted:
1. In accordance with 37 CFR 1.831(a) using the symbols and format requirements of 37 CFR 1.832 through 1.834 via the USPTO patent electronic filing system (see Section I.1 of the Legal Framework for Patent Electronic System (https://www.uspto.gov/PatentLegalFramework), hereinafter “Legal Framework”) in XML format, together with an incorporation by reference statement of the material in the XML file in a separate paragraph of the specification (an incorporation by reference paragraph) as required by 37 CFR 1.835(a)(2) or 1.835(b)(2) identifying:
a. the name of the XML file
b. the date of creation; and
c. the size of the XML file in bytes; or
2. In accordance with 37 CFR 1.831(a) using the symbols and format requirements of 37 CFR 1.832 through 1.834 on read-only optical disc(s) as permitted by 37 CFR 1.52(e)(1)(ii), labeled according to 37 CFR 1.52(e)(5), with an incorporation by reference statement of the material in the XML format according to 37 CFR 1.52(e)(8) and 37 CFR 1.835(a)(2) or 1.835(b)(2) in a separate paragraph of the specification identifying:
a. the name of the XML file;
b. the date of creation; and
c. the size of the XML file in bytes.
SPECIFIC DEFICIENCIES AND THE REQUIRED RESPONSE TO THIS NOTICE ARE AS FOLLOWS:
Specific deficiency - The incorporation by reference paragraph required by 37 CFR 1.834(c)(1), 1.835(a)(2), or 1.835(b)(2) is missing, defective or incomplete.
Required response - Applicant must:
• Provide a substitute specification in compliance with 37 CFR 1.52, 1.121(b)(3), and 1.125 inserting the required incorporation by reference paragraph, consisting of:
• A copy of the previously-submitted specification, with deletions shown with strikethrough or brackets and insertions shown with underlining (marked-up version);
• A copy of the amended specification without markings (clean version); and
• A statement that the substitute specification contains no new matter.
Drawings
The drawings are objected to under 37 CFR 1.83(a) because they fail to show "heme + anti-C5" as described in the specification. That is to say, part of the lettering of the word “heme” from the "heme + anti-C5" column, is missing in FIG. 13.
Any structural detail that is essential for a proper understanding of the disclosed invention should be shown in the drawing. MPEP § 608.02(d). Corrected drawing sheets in compliance with 37 CFR 1.121(d) are required in reply to the Office action to avoid abandonment of the application. Any amended replacement drawing sheet should include all of the figures appearing on the immediate prior version of the sheet, even if only one figure is being amended. The figure or figure number of an amended drawing should not be labeled as “amended.” If a drawing figure is to be canceled, the appropriate figure must be removed from the replacement sheet, and where necessary, the remaining figures must be renumbered and appropriate changes made to the brief description of the several views of the drawings for consistency. Additional replacement sheets may be necessary to show the renumbering of the remaining figures. Each drawing sheet submitted after the filing date of an application must be labeled in the top margin as either “Replacement Sheet” or “New Sheet” pursuant to 37 CFR 1.121(d). If the changes are not accepted by the examiner, the applicant will be notified and informed of any required corrective action in the next Office action. The objection to the drawings will not be held in abeyance.
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 37 and 45 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 37 is indefinite because of the recitation “..treatment with a complement C5 inhibitor, provides comparable improvement in at least one outcome..” (see 37a).
The term “comparable” is a relative term which renders the claim indefinite. The term “comparable” is not defined by the claim, the specification does not provide a standard for ascertaining the requisite degree, and one of ordinary skill in the art would not be reasonably apprised of the scope of the invention.
Claims 37 and 45 are indefinite because of the recitations “..compared to the outcome with a standard treatment comprising hydroxyurea” (see 37a) and “compared to treatment with hydroxyurea”.
The instant claims do not recite that hydroxyurea is being administered to the subject. Therefore, it is unclear how treatment with a complement C5 inhibitor can be compared to treatment with hydroxyurea. The metes and bounds of these claims cannot be determined.
Claim Rejections - 35 USC § 102
In the event the determination of the status of the application as subject to AIA 35 U.S.C. 102 and 103 (or as subject to pre-AIA 35 U.S.C. 102 and 103) is incorrect, any correction of the statutory basis (i.e., changing from AIA to pre-AIA ) for the rejection will not be considered a new ground of rejection if the prior art relied upon, and the rationale supporting the rejection, would be the same under either status.
The following is a quotation of the appropriate paragraphs of 35 U.S.C. 102 that form the basis for the rejections under this section made in this Office action:
A person shall be entitled to a patent unless –
(a)(1) the claimed invention was patented, described in a printed publication, or in public use, on sale, or otherwise available to the public before the effective filing date of the claimed invention.
(a)(2) the claimed invention was described in a patent issued under section 151, or in an application for patent published or deemed published under section 122(b), in which the patent or application, as the case may be, names another inventor and was effectively filed before the effective filing date of the claimed invention.
Claims 1, 4, 5, 15-18, 20, 31, 32, 34-37, 44 and 45 are rejected under 35 U.S.C. 102(a1) and 35 U.S.C. 102(a2) as being anticipated by Rother et al. (Reference submitted by Applicant; ALEXION PHAMACEUTICALS, INC WO 2007/002571).
Rother et al. teach methods for treating patients suffering from sickle cell disease comprising intravenously administering a therapeutically effective amount of an anti-C5 antibody, wherein said treatment results in decreased lysis of red blood cells in said mammal. Rother et al. teach that in certain embodiments, said mammal is a human. Rother et al. teach those with the disease usually show some signs and symptoms after four months of age. These include: anemia because the sickle cells are fragile and break apart easily and die leaving a shortage of red blood cells to carry oxygen; periodic episodes of pain referred to as crises which develop when sickle-shaped red blood cells block blood flow through tiny blood vessels to the chest, abdomen and joints, with the pain sometimes occurring in the bones; hand-foot syndrome wherein swollen hands and feet occur when sickle-shaped red blood cells block blood flow out of the hands and feet; jaundice resulting from the liver being overwhelmed by the rapid breakdown of red blood cells; frequent infections because of damage to the spleen caused by the sickle cells; stunted growth because of a shortage of oxygen and nutrients due to a lack of healthy red blood cells; and vision problems which result from damage to blood vessels in the eye caused by sickle cells plugging those blood vessels. Other complications include stroke (caused by sickle cells blocking blood flow to the brain), acute chest syndrome (chest pain, fever and difficulty breathing caused by trapped sickle cells in the lungs); organ damage to not only the spleen and eyes but also the kidneys and liver due to chronic deprivation of oxygen- rich blood; and ulcers on the legs due to a lack of blood vessels to nourish the skin. Gallstones are a possible complication resulting from high levels of bilirubin due to the high level of breakdown of red blood cells. Persons with sickle cell disease may also experience fatigue, joint pain, breathlessness, rapid heart rate, abdominal pain, bloody urine (hematuria), excessive urination, and excessive thirst (paras 0001, 0003, 0024, 0026)(i.e. hemolytic anemia, intravascular hemolysis, vaso-occlusive crisis, acute painful hepatomegaly, lung/liver VOC, inflammation pain, applies to claims 1, 4, 15-18, 20, 31, 32, 34 and 35).
Rother et al. teach that sickle cell disease (also called sickle cell anemia) is an inherited form of anemia. The red blood cells are affected by a mutation in a single beta globin gene which affects hemoglobin structure (paras 0002 and 0004)(applies to claim 15). Rother et al. teach the mutant hemoglobin causes red blood cells, which normally are smooth and round, to become hard, sticky, and shaped like sickles or crescents. These red cells tend to get stuck and block the flow of blood in small blood vessels and to lyse which in turn leads to pain, damage and a low blood count or anemia (para 0002).
Rother et al. teach that the administered antibody used for treating sickle cell disease inhibits activation of the classical pathway, the alternative pathway or the lectin complement pathway. In certain embodiments, said antibody is an antibody against a member of the complement cascade components such as for example, C5, C5b, C6, C7, C8, and C9. In certain embodiments, said antibody is an inhibitor of C5 cleavage. In certain embodiments, said antibody decreases the availability of downstream complement components. In certain embodiments, said antibody is eculizumab (paras 0026-0029)(applies to claim 5).
Rother et al. teach treating an acute episode. In certain embodiments, the treatment inhibits or decreases the amount and/or extent of an undesirable physiological condition resulting from excess lysis of red blood cells. In certain embodiments, said undesirable physiological condition is selected from the group consisting of dystonia, a clotting disorder, pulmonary hypertension, systemic hypertension, gastrointestinal contractions, abdominal pain, sternal pain, erectile dysfunction, priapism, inflammation, esophageal spasm, dysphagia, thrombosis, decreased organ perfusion, platelet activation and death. Rother et al. teach that treatment of a sickle cell disease patient with a complement inhibitor will decrease lysis of red blood cells and at least some of the symptoms seen in sickle cell patients. Many of the symptoms are a direct result of the lysis of red blood cells. Rother et al. teach one aspect of the lysis of red blood cells is the consequent excess free hemoglobin in the bloodstream. This can lead to smooth muscle dystonias involving the gastrointestinal, cardiovascular, pulmonary, and urogenital systems, as well as clotting disorders. These symptoms include gastrointestinal contractions, erectile dysfunction such as priapism, and pulmonary hypertension as well as to clot formation. Rother et al. teach that approximately one-third of sickled red blood cells are lysed via intravascular hemolysis and that it is proposed that inhibition of complement activation will decrease the amount of lysis and thereby decrease the amount and/or extent of symptoms related to the lysis of red blood cells in sickle cell disease patients. Inhibitors of members of the complement cascade including for example antibodies to components such as C5, C5b, C6, C7, C8, and C9 (paras 0026-0030)(applies to claims 36, 37, 44, 45).
Conclusion
No claims are allowed.
Any inquiry concerning this communication or earlier communications from the examiner should be directed to REGINA M DEBERRY whose telephone number is (571)272-0882. The examiner can normally be reached M-F 9:00-6:30 pm (alt Fri).
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/R.M.D/Examiner, Art Unit 1647 9/8/2026
/BRIDGET E BUNNER/Primary Examiner, Art Unit 1647
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