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Rocket Pharmaceuticals (RCKT)

Utility
PLAKOPHILIN-2 (PKP2) Gene Therapy Using Aav Vector
2 Jun 22
Provided herein is a gene therapy for PKP2 (Plakophilin-2), e.g. using an adeno-associated virus (AAV) vector.
Christopher Dean HERZOG, Chester Bittencort SACRAMENTO, Raj PRABHAKAR, David RICKS
Filed: 11 Feb 22
Utility
PLAKOPHILIN-2 (PKP2) Gene Therapy Using Aav Vector
2 Jun 22
Provided herein is a gene therapy for PKP2 (Plakophilin-2), e.g. using an adeno-associated virus (AAV) vector.
Christopher Dean HERZOG, Chester Bittencort SACRAMENTO, Raj PRABHAKAR, David RICKS
Filed: 11 Feb 22
Utility
Gene Therapy Vectors for Treatment of Danon Disease
12 May 22
The present disclosure provides gene therapy vectors comprising a polynucleotide sequence encoding a LAMP-2 polypeptide, methods of use thereof, pharmaceutical compositions, and more.
Annahita KERAVALA, Raj PRABHAKAR, Gaurav SHAH, Roderick WONG, Naveen YALAMANCHI, Piratip PRATUMSUWAN
Filed: 12 Feb 20
Utility
Gene Therapy Vectors for Treatment of Danon Disease
9 Dec 21
The disclosure relates to optimized polynucleotide sequences for LAMP-2B, expression cassettes, vectors, and methods of use thereof in treating disease, e.g.
Annahita KERAVALA, Simon MOORE, David RICKS
Filed: 8 Jan 21
Utility
Methods for Gene Modification of Hematopoietic Cells
23 Sep 21
The present invention relates generally to methods for genetic modification of hematopoietic cells.
Brian BEARD, Gaurav D. SHAH, Juan Antonio BUEREN RONCERO, Jose Carlos SEGOVIA SANZ, Paula RIO GALDO, Susana NAVARRO ORDONEZ, Elena ALMARZA NOVOA, Oscar QUINTANA BUSTAMANTE, Cristina MESA NUNEZ, Kenneth LAW, Kinnari PATEL
Filed: 30 Jul 19
Utility
Gene Therapy for CNS Degeneration
29 Jul 21
The present disclosure relates generally to compositions and methods for treating, preventing, inhibiting, or delaying central nervous system degeneration.
Annahita KERAVALA, Paven BATTIPROLU, Raj PRABHAKAR, Roderick WONG, Piratip PRATUMSUWAN, Naveen YALAMANCHI
Filed: 29 Apr 19
Utility
Production Methods for Viral Vectors
1 Jul 21
The present disclosure provides methods for manufacturing a recombinant lentiviral vectors in an adherent bioreactor, for example, by calcium-phosphate transfection of cells grown in adherent mode on low-compaction macrocarriers in an iCELLis® bioreactor system.
David RICKS, Brian BEARD, Kenneth LAW, Raj PRABHAKAR, Kinnari PATEL
Filed: 16 Aug 19
Utility
Compositions and Methods for Stem Cell Transplant
27 May 21
The present invention provides systems and methods for manufacturing and using gene corrected stem cells for gene therapy.
Brian BEARD, Julian Sevilla NAVARRO, Gaurav SHAH, Kinnari PATEL, Raj PRABHAKAR
Filed: 11 Apr 19
Utility
Gene therapy vectors for treatment of Danon disease
6 Jul 20
The disclosure relates to optimized polynucleotide sequences for LAMP-2B, expression cassettes, vectors, and methods of use thereof in treating disease, e.g.
Annahita Keravala, Simon Moore, David Ricks
Filed: 26 Dec 19
Utility
Gene Therapy Vectors for Treatment of Danon Disease
13 May 20
The disclosure relates to optimized polynucleotide sequences for LAMP-2B, expression cassettes, vectors, and methods of use thereof in treating disease, e.g.
Annahita KERAVALA, Simon MOORE, David RICKS
Filed: 26 Dec 19
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