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 the Application
This Office Action is in response to Applicant's arguments filed on June 4, 2026. Claim(s) 35 and 36 are pending and examined herein.
Response to Arguments
Applicant amendments/arguments and Declaration of Dr. Farhan with respect to the 112(a) or 35 U.S.C. 112 (pre-AIA ), first paragraph rejection over claim 35 as failing to comply with the enablement requirement have been fully considered.
Applicant argues:
… in the present application at pp. 101-102, paragraphs [000199]-[000201], in some embodiments, a Pyrazolopyridazine compound, such as Compound 85, binds to a molecular chaperone that is a member of the Hsp 60 or Hsp 90 family.
Further, Dr. Farhan’s Declaration presents Figure A to show:
… that Compound 85 significantly promotes expression of CryAB, HSF1 I and CNX relative to a DMSO control, collectively aiding in the stabilization and proper cellular targeting of misfolded proteins.
Additionally, Dr. Farhan’s concludes:
Compound 85 has been demonstrated to upregulate CryAB. HSF1 and CNX (Figure A) and inhibit HSP60 and HSP90, which collectively aid in the stabilization and proper cellular targeting of misfolded proteins.
In Figure A, applicant provides mRNA expression levels for HSF1, however this study does not show specifically which HSP protein (family) is being inhibited. The data presented is not commensurate in scope of applicant’s arguments in which it is suggested that compound 85 binds preferentially to the Hsp60 or Hsp90 family. Furthermore, there is no evidence of administration to any patients with any amount. One of ordinary skill cannot discern based on the transcript abundance what concentration of compound 85 is sufficient to treat a proteopathy as claimed. mRNA upregulation doesn’t necessarily mean treatment success. Moreover, mRNA expression promotion of hsf1 as compared to CryAB and CNX is not given weight because the latter two molecular chaperones are not embraced within the scope of the instant application.
Based on the foregoing reasons, the rejection of record is hereby maintained.
The maintained/modified rejections are made in the Final Office action below as necessitated by amendment.
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.
Claims 35-36 are 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 claim(s) contains 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 make and/or use the invention.
To be enabling, the specification of the patent must teach those skilled in the art how to make and use the full scope of the claimed invention without undue experimentation. In re Wright, 999 F.2d 1557, 1561 (Fed. Cir. 1993). Explaining what is meant by "undue experimentation," the Federal Circuit has stated: The test is not merely quantitative, since a considerable amount of experimentation is permissible, if it is merely routine, or if the specification in question provides a reasonable amount of guidance with respect to the direction in which the experimentation should proceed to enable the determination of how to practice a desired embodiment of the claimed invention. PPG v. Guardian, 75 F.3d 1558, 1564 (Fed. Cir. 1996).1 The factors that may be considered in determining whether a disclosure would require undue experimentation are set forth by In re Wands, 8 USPQ2d 1400 (CAFC 1988) at 1404 where the court set forth the eight factors to consider when assessing if a disclosure would have required undue experimentation. Citing Ex parte Forman, 230 USPQ 546 (BdApls 1986) at 547 the court recited eight factors: 1) the nature of the invention 2) the state of the prior art 3) the relative skill of those in the art 4) the breadth of the claims 5) predictability of the art 6) the amount of direction or guidance provided 7) the presence or absence of working examples and 8) the quantity of experimentation necessary. These factors are always applied against the background understanding that scope of enablement varies inversely with the degree of unpredictability involved. In re Fisher, 57 CCPA 1099, 1108, 427 F.2d 833, 839, 166 USPQ 18, 24 (1970). Keeping that in mind, the Wands factors are relevant to the instant fact situation for the following reasons:
(1), (6), (7): The nature of the invention, the amount of direction or guidance provided, the presence or absence of working examples:
The claim is directed to a method of treating and preventing multiple distinct diseases using the pyrazolopyridazine compound recited in the instant claim. The specification does not demonstrate the inventors were in possession of the claimed therapeutic methods at the time of filing. Although the specification identifies the chemical structure of said pyrazolopyridazine compound and the synthesis thereof, the specification does not include any experimental data, in vitro studies, animal models, or clinical evidence demonstrating the effectiveness of the compound to treat or prevent any of the recited diseases. The specification contains only prophetic statements asserting that the pyrazolopyridazine compound “can” be administered [000227] and recitation of numerous illustrative proteopathies (table 3-5, by way of example).
The Cleveland Clinic (Sickle Cell Anemia: Symptoms, Causes & Treatment) establishes that sickle cell anemia is an inherited blood disorder (i.e., it’s genetic), so administering the recited compounds is not expected to prevent this disease. Moreover, regarding treatment the recited compounds are not among the few recognized treatments of sickle cell disease. Thus, there remains a great level of unpredictability of treating sickle cell disease.
Additionally, the reference by Basak (Cellular Molecular Life Science, 2021) establishes that even in 2021 there was no cure or approved treatment for CLN5 Batten disease, so by inference there was also no cure or approved treatment of this disease on Applicant’s earliest effective filing date of June 11, 2015. Consequently, there is great unpredictability in treating or preventing Finnish variant late infantile CLN5/Batten disease and it would impose an undue burden on the ordinary skilled artisan to practice the claimed method.
Further, Vermilion (https://www.medlink.com/articles/batten-disease) teaches that Batten disease, or neuronal ceroid lipofuscinosis, comprises 13 genetically distinct disorders that differ by causative gene, gene product, and biological process. This further corroborates the notion that there is great unpredictability in treating or preventing Batten disease and would impose an undue burden on the ordinary skilled artisan to practice the claimed method.
Applicant has not provided sufficient written description that would enable the skilled artisan to make and/or use the invention.
Conclusion
Claims 35-36 are not allowed.
Applicant's amendment necessitated the new ground(s) of rejection presented in this Office action. Accordingly, THIS ACTION IS MADE FINAL. See MPEP § 706.07(a). 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 extension fee 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.
Information regarding the status of an application may be obtained from the Patent Application Information Retrieval (PAIR) system. Status information for published applications may be obtained from either Private PAIR or Public PAIR. Status information for unpublished applications is available through Private PAIR only. For more information about the PAIR system, see http://pair-direct.uspto.gov. Should you have questions on access to the Private PAIR system, contact the Electronic Business Center (EBC) at 866-217-9197 (toll-free). If you would like assistance from a USPTO Customer Service Representative or access to the automated information system, call 800-786-9199 (IN USA OR CANADA) or 571-272-1000.
Any inquiry concerning this communication or earlier communications from the examiner should be directed to Sahar Javanmard whose telephone number is (571)270-3280. The examiner can normally be reached on Monday-Friday, 9:00-5:00 EST.
If attempts to reach the examiner by telephone are unsuccessful, the examiner’s supervisor, James Alstrum-Acevedo can be reached on 571-272-5548. The fax phone number for the organization where this application or proceeding is assigned is 571-273-8300.
/SAHAR JAVANMARD/Primary Examiner, Art Unit 1622
Prosecution Insights