A61K31/4515

GENE THERAPY FOR AADC DEFICIENCY
20210236653 · 2021-08-05 ·

The present invention is directed to compositions and methods for treating aromatic L-amino acid decarboxylase (AADC) deficiency. This invention includes a method of treating AADC deficiency in a pediatric subject, comprising the steps of: (a) providing a pharmaceutical formulation comprising an rAAV2-hAADC vector, (b) stereotactically delivering the pharmaceutical formulation to at least one target site in the brain of the subject in a dose of an amount at least about 1.8×10.sup.11 vg; wherein delivering the pharmaceutical formulation to the brain is optionally by frameless stereotaxy, and optionally wherein the dose is an amount of at least about 2.4×10.sup.11 vg and in some embodiments wherein the pharmaceutical formulation comprises a rAAV2-hAADC vector concentration of about 5.7×10.sup.11 vg/mL. This invention is also directed to methods for treating aromatic L-amino acid decarboxylase (AADC) deficiency, wherein the method optionally further comprises the step of administering a therapeutically effective dose of dopamine-antagonist to the subject such as risperidone. This invention is also directed to methods for treating aromatic L-amino acid decarboxylase (AADC) deficiency, wherein the method optionally comprises providing a pharmaceutical formulation comprising an rAAV2-hAADC vector, and empty capsids.

GENE THERAPY FOR AADC DEFICIENCY
20210236653 · 2021-08-05 ·

The present invention is directed to compositions and methods for treating aromatic L-amino acid decarboxylase (AADC) deficiency. This invention includes a method of treating AADC deficiency in a pediatric subject, comprising the steps of: (a) providing a pharmaceutical formulation comprising an rAAV2-hAADC vector, (b) stereotactically delivering the pharmaceutical formulation to at least one target site in the brain of the subject in a dose of an amount at least about 1.8×10.sup.11 vg; wherein delivering the pharmaceutical formulation to the brain is optionally by frameless stereotaxy, and optionally wherein the dose is an amount of at least about 2.4×10.sup.11 vg and in some embodiments wherein the pharmaceutical formulation comprises a rAAV2-hAADC vector concentration of about 5.7×10.sup.11 vg/mL. This invention is also directed to methods for treating aromatic L-amino acid decarboxylase (AADC) deficiency, wherein the method optionally further comprises the step of administering a therapeutically effective dose of dopamine-antagonist to the subject such as risperidone. This invention is also directed to methods for treating aromatic L-amino acid decarboxylase (AADC) deficiency, wherein the method optionally comprises providing a pharmaceutical formulation comprising an rAAV2-hAADC vector, and empty capsids.

RADIATION SENSITIVITY ENHANCING COMPOSITION CONTAINING ARIPIPRAZOLE AS ACTIVE INGREDIENT

The present invention relates to a composition for enhancing radiation sensitivity comprising aripiprazole as an active ingredient, and more specifically, to a composition for enhancing radiation sensitivity capable of treating cancer by acting as a radiation sensitizer when aripiprazole is combined with radiation. By administering an effective amount of aripiprazole according to the present invention in combination with radiation irradiation, it has excellent radiation sensitivity enhancing effects such as reducing cancer cell viability and inducing cancer cell death, so it can be usefully used as a radiation sensitivity enhancer.

RADIATION SENSITIVITY ENHANCING COMPOSITION CONTAINING ARIPIPRAZOLE AS ACTIVE INGREDIENT

The present invention relates to a composition for enhancing radiation sensitivity comprising aripiprazole as an active ingredient, and more specifically, to a composition for enhancing radiation sensitivity capable of treating cancer by acting as a radiation sensitizer when aripiprazole is combined with radiation. By administering an effective amount of aripiprazole according to the present invention in combination with radiation irradiation, it has excellent radiation sensitivity enhancing effects such as reducing cancer cell viability and inducing cancer cell death, so it can be usefully used as a radiation sensitivity enhancer.

RADIATION SENSITIVITY ENHANCING COMPOSITION CONTAINING ARIPIPRAZOLE AS ACTIVE INGREDIENT

The present invention relates to a composition for enhancing radiation sensitivity comprising aripiprazole as an active ingredient, and more specifically, to a composition for enhancing radiation sensitivity capable of treating cancer by acting as a radiation sensitizer when aripiprazole is combined with radiation. By administering an effective amount of aripiprazole according to the present invention in combination with radiation irradiation, it has excellent radiation sensitivity enhancing effects such as reducing cancer cell viability and inducing cancer cell death, so it can be usefully used as a radiation sensitivity enhancer.

COMPOSITIONS AND METHODS FOR OPIOID OVERDOSE RESCUE
20210228570 · 2021-07-29 ·

Disclosed herein are various drug delivery systems, pharmaceutical compositions, dosage forms, and kits for providing opioid overdose rescue to a patient as well as a method for providing opioid overdose rescue to a patient. The drug delivery systems, pharmaceutical compositions, dosage forms, and kits may comprise an opioid antagonist and an antipsychotic agent.

COMPOSITIONS AND METHODS FOR OPIOID OVERDOSE RESCUE
20210228570 · 2021-07-29 ·

Disclosed herein are various drug delivery systems, pharmaceutical compositions, dosage forms, and kits for providing opioid overdose rescue to a patient as well as a method for providing opioid overdose rescue to a patient. The drug delivery systems, pharmaceutical compositions, dosage forms, and kits may comprise an opioid antagonist and an antipsychotic agent.

COMPOSITIONS AND METHODS FOR OPIOID OVERDOSE RESCUE
20210228570 · 2021-07-29 ·

Disclosed herein are various drug delivery systems, pharmaceutical compositions, dosage forms, and kits for providing opioid overdose rescue to a patient as well as a method for providing opioid overdose rescue to a patient. The drug delivery systems, pharmaceutical compositions, dosage forms, and kits may comprise an opioid antagonist and an antipsychotic agent.

NEUROMODULATING COMPOSITIONS AND RELATED THERAPEUTIC METHODS FOR THE TREATMENT OF CANCER

Described herein are methods for treating a subject having or at risk of developing cancer by administering a neuromodulating agent.

NEUROMODULATING COMPOSITIONS AND RELATED THERAPEUTIC METHODS FOR THE TREATMENT OF CANCER

Described herein are methods for treating a subject having or at risk of developing cancer by administering a neuromodulating agent.