Broadening the Scope of the Claims in Gene Therapy Applications

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Broadening the Scope of the Claims in Gene
Therapy Applications

• Deborah Reynolds
• Detailee, TCPS1600
• 571-272-0734
• Deborah.Reynolds_at_uspto.Gov

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Topics

• Gene Therapy, a little background
• Claims Analysis
• Analysis of the Disclosure
• Correlation of Evidence
• How to overcome the rejection

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Gene Therapy

• Ex-vivo gene therapy
• Cells transfected with gene in vitro and then
  administered to the patient
• In-vivo gene therapy
• Polynucleotide is administered directly into the
  patient

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Gene Therapy

• Traditionally
• Replacement of a defective gene
• Currently
• Replacement of a defective gene
• Vaccine immunization
• DNA immunization
• Anti-sense therapy
• Supplying any therapeutic expression product

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Obstacles for Gene Therapy

• Stable expression of encoded gene
• Host immune responses to vectors
• Targeting vectors to specific cells
• Specificity of vector expression
• Representative animal models
• Recognition of Immunogenic Epitopes which provide
  a therapeutic benefit

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Scope of Enablement

• the specification 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, 27 USPQ2d 1510, 1513 (Fed. Cir. 1993).

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Claim Interpretation

• The claims are to be given their broadest
  reasonable interpretation that is consistent with
  the specification
• Limitations and examples appearing in the
  specification do not generally limit what is
  covered by the claims

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Intended Use Limitation

• When a compound or composition is limited by a
  particular use, enablement of that claim should
  be evaluated based on that limitation. MPEP
  2164.01(c)
• Prior art evaluation may or may not turn based
  upon an intended use. The language used and
  where it occurs in the claim must be considered.
  See Eaton Corp. v. Rockwell International Corp.,
  66 USPQ2d 1271 (CA FC 2003).

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Types of Claims

• Products
• A product, in order to be enabled must be
  disclosed such that there is at least one enabled
  method of making, and at least one enabled method
  of using the claimed product

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Types of Claims

• Methods
• In order for a method claim to be enabled, each
  step must be enabled including all materials used
  therein

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Foundation for Examiners Analysis

• Determine what each claim as a whole covers
• Broadest reasonable interpretation in light of
  and consistent with written description - In re
  Morris, 127 F.3d 1048, 44 USPQ2d 1023 (Fed. Cir.
  1997)
• Preamble and transition phrases are treated under
  common usage

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Gene Therapy Claims

• Examination considerations
• Sufficient administration
• Sufficient expression
• Art recognition of animal model
• Phenotypic change correlated to the disease

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Examiner Analysis of Gene Therapy Claims

• Evaluate claims on
• Scope of the vector
• (Adenoviral, retroviral, naked DNA, liposomes)
• Scope of delivery
• (IM, IV, sub Q, ID, oral, tissue specific target)
• Scope of treatment
• (Cancer, vaccine, viruses, hereditary, etc.)
• Scope of molecule(s) delivered
• (Related to disease being treated?)
• Potential for ineffective in vivo responses
• (Against vector, against cells, against host)

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MPEP 2164.05

• "In making the determination of enablement, the
  examiner shall consider the original disclosure
  and all evidence in the record, weighing evidence
  that supports enablement against evidence that
  the specification is not enabling."

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MPEP 2164.03

• "The scope of the required enablement varies
  inversely with the degree of predictability
  involved, but even in unpredictable arts, a
  disclosure of every operable species is not
  required. A single embodiment may provide broad
  enablement in cases involving predictable
  factors, such as mechanical or electrical
  elements...However, in applications directed to
  inventions in arts where the results are
  unpredictable, the disclosure of a single species
  usually does not provide an adequate basis to
  support generic claims.

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Working Examples

• Working examples.
• Compliance with enablement requirement does not
  turn on whether an example is disclosed.
• Specification need not contain an example if the
  invention is otherwise disclosed in such manner
  that one skilled in the art will be able to
  practice it without undue amount of
  experimentation, in re Borkowski, 164 USPQ 642
  (CCPA 1970).
• A single working example in the specification for
  a claimed invention is enough to preclude a
  rejection which states that nothing is enabled
  since at least one embodiment is enabled.

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Ex parte Maas, 9 Uspq2d 1746

• "Substantiating evidence may be in the form of
  animal tests which constitute recognized
  screening procedures with clear relevance to
  utility in humans. See Ex parte Krepelka, 231
  USPQ 746 (Board of Patent Appeals and
  Interferences 1986) and cases cited therein."

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Ex parte Maas, 9 Uspq2d 1746

• "First, although appellants' specification
  describes certain in vitro experiments, there is
  no correlation on this record between in vitro
  experiments and a practical utility in currently
  available form for humans or animals. It is not
  enough to rely on in vitro studies where, as
  here, a person having ordinary skill in the art
  has no basis for perceiving those studies as
  constituting recognized screening procedures with
  clear relevance to utility in humans or animals"
  (emphasis added)

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Ex parte Balzarini, 21 Uspq2d 1892

• "There is no evidence of record that experimental
  animal models have been developed in this area
  which would be predictive of human efficacy."

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Cross v. Iizuka, 224 USPQ 739

• "Cross is arguing that there must be a rigorous
  correlation of pharmacological activity between
  the disclosed in vitro utility and an in vivo
  utility to establish practical utility. We,
  however, find ourselves in agreement with the
  Board that, based upon the relevant evidence as a
  whole, there is a reasonable correlation between
  the disclosed in vitro utility and an in vivo
  activity, and therefore a rigorous correlation is
  not necessary where the disclosure of
  pharmacological activity is reasonable based upon
  the probative evidence."

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Means to Obviate the Enablement Rejection

• Has a reasonable basis to question the
  enablement been established? See MPEP 2164.04
• Applicants may submit argument and/or evidence
  that the disclosure as filed is enabled. See
  MPEP 2161.05
• Help us draw the correlations between your data
  and the real world utility

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