FORMULATION OPTIMIZATION FOR VIRAL PARTICLES
The present disclosure provides formulations for maintaining viral particles in a stable form. In certain embodiments, the formulation comprises at least one basic amino acid and a buffer solution, wherein the viral particle is present at a concentration of about 1×10 11 to about 5×10 13 DNase resistant virus particles per mL.
1 . A formulation for maintaining a viral particle in a stable form, the formulation comprising at least one basic amino acid and a buffer solution, wherein the viral particle is present at a concentration of about 1×10 11 to about 5×10 13 DNase resistant virus particles per mL (DRP/mL).
2 . The formulation of claim 1 , wherein the basic amino acid is selected from the group consisting of: arginine, histidine and lysine.
3 . The formulation of claim 2 , wherein the arginine is present in the formulation in an amount of about 2 mM to about 10 mM.
4 . The formulation of claim 3 , wherein the arginine is present in the formulation in an amount of about 5 mM.
5 . The formulation of claim 2 , wherein the histidine is present in the formulation in an amount of about 2 mM to about 10 mM.
6 . The formulation of claim 5 , wherein the histidine is present in the formulation in an amount of about 5 mM.
7 . The formulation of claim 2 , wherein the lysine is present in the formulation in an amount of about 2 mM to about 10 mM.
8 . The formulation of claim 7 , wherein the lysine is present in the formulation in an amount of about 5 mM.
9 . The formulation of claim 1 , further comprising a non-ionic surfactant.
10 . The formulation of claim 1 , wherein the non-ionic surfactant is tween-20.
11 . The formulation of claim 10 , wherein the tween 20 is present in the formulation is an amount of about 0.005% to about 0.025% (v/v).
12 . The formulation of claim 11 , wherein the tween 20 is present in the formulation in an amount of about 0.014% (v/v).
13 . (canceled)
14 . The formulation of claim 1 , wherein the viral particle is present at a concentration of about 5×10 12 to about 5×10 13 DNase resistant virus particles per mL (DRP/mL).
15 . The formulation of claim 1 , wherein the pH of the formulation is about 7.0 to about 7.5.
16 . The formulation of claim 15 , wherein the pH of the formulation is about 7.5.
17 . The formulation of claim 1 , wherein the viral particle is an adeno-associated virus (AAV).
18 . The formulation of claim 17 , wherein the viral particle is a recombinant adeno-associated virus (rAAV).
19 . The formulation of claim 1 , wherein the formulation is stable after storage at −80 C or the formulation is stable after heating to 45 C.
20 . (canceled)
21 . (canceled)
22 . The formulation of claim 1 , wherein the formulation is used for the delivery of a therapeutic.
23 . The formulation of claim 22 , wherein the therapeutic is a gene therapy vector.