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A Bet on a Unique Set of Vectors

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Content provided by RARECast. All podcast content including episodes, graphics, and podcast descriptions are uploaded and provided directly by RARECast or their podcast platform partner. If you believe someone is using your copyrighted work without your permission, you can follow the process outlined here https://player.fm/legal.

Homology Medicines is developing a range of genetic therapies based on a unique set of adeno-associated virus vectors derived from human hematopoietic stem cells that allow it to target a wide range of tissues. It is developing both gene therapies and gene editors simultaneously using these vectors. It’s lead program is an experimental gene therapy for phenylketonuria or PKU, a rare, genetic metabolic condition that causes an enzyme deficiency that results in an inability to breakdown the amino acid phenylalanine, which is common in protein containing foods. We spoke to Arthur Tzianabos, CEO of Homology Medicines, about the company’s genetic therapies, its program in PKU, and how it pairs its vectors and approach to meet the needs of a given condition.

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488 episodes

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A Bet on a Unique Set of Vectors

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Manage episode 304671532 series 60790
Content provided by RARECast. All podcast content including episodes, graphics, and podcast descriptions are uploaded and provided directly by RARECast or their podcast platform partner. If you believe someone is using your copyrighted work without your permission, you can follow the process outlined here https://player.fm/legal.

Homology Medicines is developing a range of genetic therapies based on a unique set of adeno-associated virus vectors derived from human hematopoietic stem cells that allow it to target a wide range of tissues. It is developing both gene therapies and gene editors simultaneously using these vectors. It’s lead program is an experimental gene therapy for phenylketonuria or PKU, a rare, genetic metabolic condition that causes an enzyme deficiency that results in an inability to breakdown the amino acid phenylalanine, which is common in protein containing foods. We spoke to Arthur Tzianabos, CEO of Homology Medicines, about the company’s genetic therapies, its program in PKU, and how it pairs its vectors and approach to meet the needs of a given condition.

  continue reading

488 episodes

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