UNLEASHING THE POWER OF
GENE THERAPY
About
The era of gene therapy has arrived
Our mission at Kriya Therapeutics is to expand the reach of gene therapy to address highly prevalent and serious diseases affecting millions of patients.
Kriya is a next-generation gene therapy company focused on designing and developing transformative new treatments for highly prevalent and severe chronic diseases. We target conditions where the underlying biology is well-understood, rationally designing one-time gene therapies to durably express therapeutic proteins within the appropriate human tissues. Our team has deep expertise in the field and includes former senior leadership from pioneering gene therapy companies and academic institutions. We use algorithmic tools, scalable infrastructure, and proprietary technology to optimize the efficacy and durability of our gene therapies – with a goal of transforming the treatment paradigm for some of the most serious and widespread diseases.
Team
Shankar Ramaswamy, MD
Co-Founder, Chairman, and Chief Executive Officer
Shankar Ramaswamy, MD
Co-Founder, Chairman, and Chief Executive Officer
Shankar Ramaswamy is a Co-Founder, Chairman, and Chief Executive Officer of Kriya. Dr. Ramaswamy was part of the early foundational team at Roivant Sciences, where he was responsible for helping to identify and evaluate assets across therapeutic areas and launch its first public subsidiary company. He went on to be the Chief Business Officer of Axovant Gene Therapies.
Dr. Ramaswamy holds a medical degree from Brown University and an undergraduate degree in economics from Harvard University.
Roger Jeffs, PhD
Co-Founder and Vice Chairman
Roger Jeffs, PhD
Co-Founder and Vice Chairman
Roger Jeffs is a Co-Founder and Vice Chairman at Kriya. Dr. Jeffs was the President and co-CEO of United Therapeutics Corporation, joining the company during its start-up phase in 1998. He helped lead the company’s IPO, oversaw the clinical development and regulatory approval of 6 products for rare diseases, and managed the commercial efforts that led to a $1.5B annual revenue run rate. He currently serves on the boards of Axsome Therapeutics and Albireo Pharma. He previously served on the boards of United Therapeutics, Sangamo Therapeutics, and Dova Pharmaceuticals.
Dr. Jeffs holds a PhD in pharmacology from the University of North Carolina School of Medicine and an undergraduate degree in chemistry from Duke University.
Fraser Wright, PhD
Scientific Co-Founder and Chief Scientific Advisor
Fraser Wright, PhD
Scientific Co-Founder and Chief Scientific Advisor
Fraser Wright is the Scientific Co-Founder and Chief Scientific Advisor at Kriya. Dr. Wright is a Professor of Pediatrics in the Center for Definitive and Curative Medicine at Stanford University. He was also the co-founder and Chief Technology Officer of Spark Therapeutics, where he helped oversee the product development and manufacturing of Luxturna (TM) and other pipeline programs. Prior to co-founding Spark Therapeutics, Dr. Wright was the founding Scientific Director of the Clinical Vector Core Laboratory at the Children’s Hospital of Philadelphia. He was also previously the director of development and clinical manufacturing at Avigen.
Dr. Wright holds a PhD in biochemistry and molecular immunology and an undergraduate degree in biochemistry and physiology from the University of Toronto.
Melissa Rhodes, PhD, DABT
Chief Development Officer
Melissa Rhodes, PhD, DABT
Chief Development Officer
Melissa Rhodes is the Chief Development Officer at Kriya. Dr. Rhodes was the Chief Development Officer at Aerami Therapeutics, a company focused on inhaled biologic therapies. Prior to Aerami, Dr. Rhodes was the Chief Development Officer at Altavant Sciences, a respiratory rare disease company. Dr. Rhodes also served as Senior Vice President at Roivant Sciences, where she built and led a team of scientists conducting nonclinical research for multiple Roivant subsidiary companies. She spent almost 10 years in R&D at GSK and started her career in drug development at an oncology-focused biotech university spin-out.
Dr. Rhodes completed a postdoctoral fellowship at the National Toxicology Program at the National Institute of Environmental Health Sciences, holds a PhD in pharmacology and toxicology from Duke University Medical Center, and is a Diplomate of the American Board of Toxicology.
Britt Petty
Chief Manufacturing Officer
Britt Petty
Chief Manufacturing Officer
Britt Petty is the Chief Manufacturing Officer at Kriya. Mr. Petty was the Chief Technical Officer (interim) and Head of Global Manufacturing at AveXis, a Novartis company, focused on the development and commercialization of gene therapies for neurological genetic diseases. At AveXis, he was responsible for manufacturing the company’s commercial and clinical gene therapy products at both internal and external sites across the United States and Europe. Britt oversaw all of supply chain, engineering, program management, and manufacturing science and technology functions.
Prior to AveXis, Mr. Petty held senior manufacturing roles at Biogen, including as the site head for the company’s North Carolina facility. He previously held manufacturing roles of increasing responsibility at Amgen and Eli Lily and Co.
Mr. Petty completed his undergraduate degree in mechanical engineering at Rose Hulman Institute of Technology and his master’s in business administration at the University of North Carolina-Chapel Hill.
Daniel Chen
Chief Financial Officer
Daniel Chen
Chief Financial Officer
Daniel Chen is the Chief Financial Officer at Kriya. Prior to joining Kriya, Mr. Chen worked at Portola Pharmaceuticals from 2009 to 2020, most recently as Vice President of Business Planning and Corporate Strategy. In that role, he was responsible for leading the financial planning and analysis (FP&A), treasury, purchasing and strategic planning functions for the company. Prior to Portola, Mr. Chen held various roles in finance and commercial operations at Johnson & Johnson and Gilead Sciences.
Mr. Chen received his undergraduate degree in molecular cell biology from the University of California, Berkeley and his master’s in business administration from the University of Southern California.
Nachi Gupta, MD, PhD
Chief of Staff
Nachi Gupta, MD, PhD
Chief of Staff
Nachi Gupta is the Chief of Staff at Kriya. Dr. Gupta is a board-certified emergency physician, who completed his residency at Mount Sinai Hospital in New York City. Prior to his career as a practicing physician, Dr. Gupta was a quantitative analyst at Brevan Howard Asset Management. He was also previously part of the ballistic missile defense team at MIT Lincoln Laboratories.
Dr. Gupta holds a medical degree from Brown University and a PhD in mathematics from Oxford University. He completed his master’s and undergraduate degrees in mathematics, computer science, economics, and telecommunications at the University of Pennsylvania.
Mitch Lower
Vice President of Technical Operations
Mitch Lower
Vice President of Technical Operations
Mitch Lower is the Vice President of Technical Operations at Kriya. Prior to joining Kriya, Mr. Lower was most recently the Vice President of Global Engineering and Facilities at AveXis, a Novartis company. He was responsible for the overall implementation and management of the company’s manufacturing capabilities, including the construction of two large gene therapy manufacturing facilities and the support of the AveXis engineering and facilities global network. Prior to AveXis, he held positions of increasing responsibility at Biogen for more than 20 years in roles across technical operations, engineering and facilities, manufacturing, supply chain, and network strategy.
Mr. Lower completed his undergraduate degree in Mechanical Engineering at North Carolina State University and his master’s in business administration at Duke University.
Zhu Pirot, PhD
Vice President of Translational Sciences
Zhu Pirot, PhD
Vice President of Translational Sciences
Zhu Pirot is the Vice President of Translational Sciences at Kriya. Prior to joining Kriya, Dr. Pirot was the head of analytical method development, CMC lead at Sangamo Therapeutics where she built and led the analytical function for AAV-mediated gene therapy, gene editing, and cell therapy programs. She has more than 20 years of experience in drug development and has held positions of increasing responsibility at Chiron, Avigen, and Geron.
Dr. Pirot holds a medical doctor degree from Inner Mongolia Medical College, a masters degree in cytogenetics from the Beijing Cancer Institute, and a Ph.D. in cellular and molecular biology from the University of Turin.
Brian Furmanski, PhD
Vice President of Regulatory Affairs
Brian Furmanski, PhD
Vice President of Regulatory Affairs
Brian Furmanski is the Vice President of Regulatory Affairs at Kriya. He was previously the Senior Director of Clinical Pharmacology and Regulatory Affairs at Nuventra Pharma Sciences. Prior to Nuventra, Dr. Furmanski was a Senior Clinical Pharmacology Reviewer at the FDA where he reviewed and provided regulatory and scientific advice on hundreds of IND, NDA and BLA submissions. Before his time at the FDA, Dr. Furmanski was an investigator in the muscle metabolism and repair group at GlaxoSmithKline. He started his career at Siga Technologies where he contributed to the development of novel antiviral therapies.
Dr. Furmanski completed his undergraduate degree in chemistry and biology and his Ph.D. in biochemistry at the University of South Carolina. He completed his postdoctoral fellowship at St. Jude Children’s Research Hospital.
Stephen Soltys
Vice President of Process Development
Stephen Soltys
Vice President of Process Development
Stephen Soltys is the Vice President of Process Development at Kriya Therapeutics. Before joining Kriya, Stephen was Director of the Manufacturing Science and Technology (MSAT) labs at AveXis, a Novartis company, where he led a team of upstream, downstream, and analytical scientists. Prior to Avexis, Stephen designed the manufacturing processes for multiple gene therapy companies, including Precision Biosciences, and Bamboo Therapeutics (Pfizer), and was a process development scientist at the University of North Carolina Gene Therapy Center.
Stephen completed his undergraduate degree in Biology at Marietta College in Marietta, OH, and his master’s degree in Molecular Biology at Thomas Jefferson University in Philadelphia, PA.
Michele Stone, PhD
Vice President of Translational Development
Michele Stone, PhD
Vice President of Translational Development
Michele Stone is the Vice President of Translational Development at Kriya. She was previously the Vice President of Early Development and Head of Nonclinical and Bioanalytical at Axovant Gene Therapies. Prior to Axovant Gene Therapies, Dr. Stone was Executive Director of Vaccine Development and Bioanalytical at Liquidia Technologies. Dr. Stone was also the Director of R&D at Aeras where she was responsible for developing and leading partnerships to advance multiple vaccine platforms (viral vector, protein, and nucleic acid) into and through clinical development. In addition, Dr. Stone was a Senior Scientist at Canon US Life Sciences where she contributed to the development of diagnostics targeted towards personalized medicine.
Dr. Stone completed her undergraduate degree in Chemistry at Lynchburg College and her Ph.D. in Biochemistry and Molecular Biology at the University of Maryland, Baltimore. She completed her postdoctoral fellowship at the University of Maryland, Baltimore in Physiology and Neuroscience.
Lily Nguyen
Vice President of Project Management
Lily Nguyen
Vice President of Project Management
Lily Nguyen is the Vice President of Project Management at Kriya Therapeutics. Prior to joining Kriya, Ms. Nguyen was Director of Program and Alliance Management at SpringWorks Therapeutics, a company focused on advancing therapies in rare disease and oncology. She supported development teams to plan and execute on global clinical studies and managed development programs with alliance partners. Prior to SpringWorks, Ms. Nguyen was Senior Director of Program Management at Roivant Sciences where she led planning and organization of drug development projects through NDA preparation and coordination of due diligence review for potential in-licensed assets. She has over 15 years of drug development experience and has held project management roles of increasing responsibility at Aerie, Cempra and Syntheon Pharmaceuticals.
Ms. Nguyen completed her undergraduate degree in chemical engineering at North Carolina State University and her master’s in business administration at the University of North Carolina at Chapel Hill.
Jason Mallory, PhD
Vice President of Clinical Development
Jason Mallory, PhD
Vice President of Clinical Development
Jason Mallory is the Vice President of Clinical Development at Kriya Therapeutics. Prior to joining Kriya, Dr. Mallory led Clinical Development activities spanning pre-IND through Phase 3 development for AAV or LV-based gene therapies at Spark Therapeutics, GlaxoSmithKline, and Adverum Biotechnologies. Prior to his experience in rare disease gene therapy, he spent more than nine years working in Clinical Development for common diseases such as chronic pain and type 2 diabetes mellitus. While at GSK, Dr. Mallory contributed to the Phase 3 clinical development program, successful global licensing applications, and post-marketing studies for albiglutide, a once-weekly glucagon-like peptide 1 (GLP-1) receptor agonist for the management of patients with type 2 diabetes mellitus.
Dr. Mallory holds a PhD in Molecular Biology & Genetics from Baylor University. He completed post-doctoral work at the University of Texas Southwestern Medical Center and the Institute of Metabolic Disease at Baylor University Medical Center.
Harold Alterson
Vice President of Quality
Harold Alterson
Vice President of Quality
Harold Alterson is the Vice President of Quality at Kriya. Prior to joining Kriya, Mr. Alterson was most recently Senior Director of Quality Control and CMC Lead at Bluebird Bio. He was part of the team involved in the construction and start-up of Bluebird’s first internal manufacturing facility, and was responsible for building out the Quality Control team at the site to support facility qualification and routine operations. Prior to Bluebird, Mr. Alterson held positions of increasing responsibility in Quality Assurance at Biogen, overseeing Quality Engineering, Quality Systems, and Quality Operations. He spent the early part of his career in research and development focused on alphavirus vectors for vaccine use at AlphaVax.
Mr. Alterson holds a B.S. in Microbiology from Montana State University.
Shankar Ramaswamy, MD
Co-Founder, Chairman, and Chief Executive Officer
Shankar Ramaswamy, MD
Co-Founder, Chairman, and Chief Executive Officer
Shankar Ramaswamy is a Co-Founder, Chairman, and Chief Executive Officer of Kriya. Dr. Ramaswamy was part of the early foundational team at Roivant Sciences, where he was responsible for helping to identify and evaluate assets across therapeutic areas and launch its first public subsidiary company. He went on to be the Chief Business Officer of Axovant Gene Therapies.
Dr. Ramaswamy holds a medical degree from Brown University and an undergraduate degree in economics from Harvard University.
Roger Jeffs, PhD
Co-Founder and Vice Chairman
Roger Jeffs, PhD
Co-Founder and Vice Chairman
Roger Jeffs is a Co-Founder and Vice Chairman at Kriya. Dr. Jeffs was the President and co-CEO of United Therapeutics Corporation, joining the company during its start-up phase in 1998. He helped lead the company’s IPO, oversaw the clinical development and regulatory approval of 6 products for rare diseases, and managed the commercial efforts that led to a $1.5B annual revenue run rate. He currently serves on the boards of Axsome Therapeutics and Albireo Pharma. He previously served on the boards of United Therapeutics, Sangamo Therapeutics, and Dova Pharmaceuticals.
Dr. Jeffs holds a PhD in pharmacology from the University of North Carolina School of Medicine and an undergraduate degree in chemistry from Duke University.
Fraser Wright, PhD
Scientific Co-Founder and Chief Scientific Advisor
Fraser Wright, PhD
Scientific Co-Founder and Chief Scientific Advisor
Fraser Wright is the Scientific Co-Founder and Chief Scientific Advisor at Kriya. Dr. Wright is a Professor of Pediatrics in the Center for Definitive and Curative Medicine at Stanford University. He was also the co-founder and Chief Technology Officer of Spark Therapeutics, where he helped oversee the product development and manufacturing of Luxturna (TM) and other pipeline programs. Prior to co-founding Spark Therapeutics, Dr. Wright was the founding Scientific Director of the Clinical Vector Core Laboratory at the Children’s Hospital of Philadelphia. He was also previously the director of development and clinical manufacturing at Avigen.
Dr. Wright holds a PhD in biochemistry and molecular immunology and an undergraduate degree in biochemistry and physiology from the University of Toronto.
Melissa Rhodes, PhD, DABT
Chief Development Officer
Melissa Rhodes, PhD, DABT
Chief Development Officer
Melissa Rhodes is the Chief Development Officer at Kriya. Dr. Rhodes was the Chief Development Officer at Aerami Therapeutics, a company focused on inhaled biologic therapies. Prior to Aerami, Dr. Rhodes was the Chief Development Officer at Altavant Sciences, a respiratory rare disease company. Dr. Rhodes also served as Senior Vice President at Roivant Sciences, where she built and led a team of scientists conducting nonclinical research for multiple Roivant subsidiary companies. She spent almost 10 years in R&D at GSK and started her career in drug development at an oncology-focused biotech university spin-out.
Dr. Rhodes completed a postdoctoral fellowship at the National Toxicology Program at the National Institute of Environmental Health Sciences, holds a PhD in pharmacology and toxicology from Duke University Medical Center, and is a Diplomate of the American Board of Toxicology.
Britt Petty
Chief Manufacturing Officer
Britt Petty
Chief Manufacturing Officer
Britt Petty is the Chief Manufacturing Officer at Kriya. Mr. Petty was the Chief Technical Officer (interim) and Head of Global Manufacturing at AveXis, a Novartis company, focused on the development and commercialization of gene therapies for neurological genetic diseases. At AveXis, he was responsible for manufacturing the company’s commercial and clinical gene therapy products at both internal and external sites across the United States and Europe. Britt oversaw all of supply chain, engineering, program management, and manufacturing science and technology functions.
Prior to AveXis, Mr. Petty held senior manufacturing roles at Biogen, including as the site head for the company’s North Carolina facility. He previously held manufacturing roles of increasing responsibility at Amgen and Eli Lily and Co.
Mr. Petty completed his undergraduate degree in mechanical engineering at Rose Hulman Institute of Technology and his master’s in business administration at the University of North Carolina-Chapel Hill.
Daniel Chen
Chief Financial Officer
Daniel Chen
Chief Financial Officer
Daniel Chen is the Chief Financial Officer at Kriya. Prior to joining Kriya, Mr. Chen worked at Portola Pharmaceuticals from 2009 to 2020, most recently as Vice President of Business Planning and Corporate Strategy. In that role, he was responsible for leading the financial planning and analysis (FP&A), treasury, purchasing and strategic planning functions for the company. Prior to Portola, Mr. Chen held various roles in finance and commercial operations at Johnson & Johnson and Gilead Sciences.
Mr. Chen received his undergraduate degree in molecular cell biology from the University of California, Berkeley and his master’s in business administration from the University of Southern California.
Nachi Gupta, MD, PhD
Chief of Staff
Nachi Gupta, MD, PhD
Chief of Staff
Nachi Gupta is the Chief of Staff at Kriya. Dr. Gupta is a board-certified emergency physician, who completed his residency at Mount Sinai Hospital in New York City. Prior to his career as a practicing physician, Dr. Gupta was a quantitative analyst at Brevan Howard Asset Management. He was also previously part of the ballistic missile defense team at MIT Lincoln Laboratories.
Dr. Gupta holds a medical degree from Brown University and a PhD in mathematics from Oxford University. He completed his master’s and undergraduate degrees in mathematics, computer science, economics, and telecommunications at the University of Pennsylvania.
Mitch Lower
Vice President of Technical Operations
Mitch Lower
Vice President of Technical Operations
Mitch Lower is the Vice President of Technical Operations at Kriya. Prior to joining Kriya, Mr. Lower was most recently the Vice President of Global Engineering and Facilities at AveXis, a Novartis company. He was responsible for the overall implementation and management of the company’s manufacturing capabilities, including the construction of two large gene therapy manufacturing facilities and the support of the AveXis engineering and facilities global network. Prior to AveXis, he held positions of increasing responsibility at Biogen for more than 20 years in roles across technical operations, engineering and facilities, manufacturing, supply chain, and network strategy.
Mr. Lower completed his undergraduate degree in Mechanical Engineering at North Carolina State University and his master’s in business administration at Duke University.
Zhu Pirot, PhD
Vice President of Translational Sciences
Zhu Pirot, PhD
Vice President of Translational Sciences
Zhu Pirot is the Vice President of Translational Sciences at Kriya. Prior to joining Kriya, Dr. Pirot was the head of analytical method development, CMC lead at Sangamo Therapeutics where she built and led the analytical function for AAV-mediated gene therapy, gene editing, and cell therapy programs. She has more than 20 years of experience in drug development and has held positions of increasing responsibility at Chiron, Avigen, and Geron.
Dr. Pirot holds a medical doctor degree from Inner Mongolia Medical College, a masters degree in cytogenetics from the Beijing Cancer Institute, and a Ph.D. in cellular and molecular biology from the University of Turin.
Brian Furmanski, PhD
Vice President of Regulatory Affairs
Brian Furmanski, PhD
Vice President of Regulatory Affairs
Brian Furmanski is the Vice President of Regulatory Affairs at Kriya. He was previously the Senior Director of Clinical Pharmacology and Regulatory Affairs at Nuventra Pharma Sciences. Prior to Nuventra, Dr. Furmanski was a Senior Clinical Pharmacology Reviewer at the FDA where he reviewed and provided regulatory and scientific advice on hundreds of IND, NDA and BLA submissions. Before his time at the FDA, Dr. Furmanski was an investigator in the muscle metabolism and repair group at GlaxoSmithKline. He started his career at Siga Technologies where he contributed to the development of novel antiviral therapies.
Dr. Furmanski completed his undergraduate degree in chemistry and biology and his Ph.D. in biochemistry at the University of South Carolina. He completed his postdoctoral fellowship at St. Jude Children’s Research Hospital.
Stephen Soltys
Vice President of Process Development
Stephen Soltys
Vice President of Process Development
Stephen Soltys is the Vice President of Process Development at Kriya Therapeutics. Before joining Kriya, Stephen was Director of the Manufacturing Science and Technology (MSAT) labs at AveXis, a Novartis company, where he led a team of upstream, downstream, and analytical scientists. Prior to Avexis, Stephen designed the manufacturing processes for multiple gene therapy companies, including Precision Biosciences, and Bamboo Therapeutics (Pfizer), and was a process development scientist at the University of North Carolina Gene Therapy Center.
Stephen completed his undergraduate degree in Biology at Marietta College in Marietta, OH, and his master’s degree in Molecular Biology at Thomas Jefferson University in Philadelphia, PA.
Michele Stone, PhD
Vice President of Translational Development
Michele Stone, PhD
Vice President of Translational Development
Michele Stone is the Vice President of Translational Development at Kriya. She was previously the Vice President of Early Development and Head of Nonclinical and Bioanalytical at Axovant Gene Therapies. Prior to Axovant Gene Therapies, Dr. Stone was Executive Director of Vaccine Development and Bioanalytical at Liquidia Technologies. Dr. Stone was also the Director of R&D at Aeras where she was responsible for developing and leading partnerships to advance multiple vaccine platforms (viral vector, protein, and nucleic acid) into and through clinical development. In addition, Dr. Stone was a Senior Scientist at Canon US Life Sciences where she contributed to the development of diagnostics targeted towards personalized medicine.
Dr. Stone completed her undergraduate degree in Chemistry at Lynchburg College and her Ph.D. in Biochemistry and Molecular Biology at the University of Maryland, Baltimore. She completed her postdoctoral fellowship at the University of Maryland, Baltimore in Physiology and Neuroscience.
Lily Nguyen
Vice President of Project Management
Lily Nguyen
Vice President of Project Management
Lily Nguyen is the Vice President of Project Management at Kriya Therapeutics. Prior to joining Kriya, Ms. Nguyen was Director of Program and Alliance Management at SpringWorks Therapeutics, a company focused on advancing therapies in rare disease and oncology. She supported development teams to plan and execute on global clinical studies and managed development programs with alliance partners. Prior to SpringWorks, Ms. Nguyen was Senior Director of Program Management at Roivant Sciences where she led planning and organization of drug development projects through NDA preparation and coordination of due diligence review for potential in-licensed assets. She has over 15 years of drug development experience and has held project management roles of increasing responsibility at Aerie, Cempra and Syntheon Pharmaceuticals.
Ms. Nguyen completed her undergraduate degree in chemical engineering at North Carolina State University and her master’s in business administration at the University of North Carolina at Chapel Hill.
Jason Mallory, PhD
Vice President of Clinical Development
Jason Mallory, PhD
Vice President of Clinical Development
Jason Mallory is the Vice President of Clinical Development at Kriya Therapeutics. Prior to joining Kriya, Dr. Mallory led Clinical Development activities spanning pre-IND through Phase 3 development for AAV or LV-based gene therapies at Spark Therapeutics, GlaxoSmithKline, and Adverum Biotechnologies. Prior to his experience in rare disease gene therapy, he spent more than nine years working in Clinical Development for common diseases such as chronic pain and type 2 diabetes mellitus. While at GSK, Dr. Mallory contributed to the Phase 3 clinical development program, successful global licensing applications, and post-marketing studies for albiglutide, a once-weekly glucagon-like peptide 1 (GLP-1) receptor agonist for the management of patients with type 2 diabetes mellitus.
Dr. Mallory holds a PhD in Molecular Biology & Genetics from Baylor University. He completed post-doctoral work at the University of Texas Southwestern Medical Center and the Institute of Metabolic Disease at Baylor University Medical Center.
Harold Alterson
Vice President of Quality
Harold Alterson
Vice President of Quality
Harold Alterson is the Vice President of Quality at Kriya. Prior to joining Kriya, Mr. Alterson was most recently Senior Director of Quality Control and CMC Lead at Bluebird Bio. He was part of the team involved in the construction and start-up of Bluebird’s first internal manufacturing facility, and was responsible for building out the Quality Control team at the site to support facility qualification and routine operations. Prior to Bluebird, Mr. Alterson held positions of increasing responsibility in Quality Assurance at Biogen, overseeing Quality Engineering, Quality Systems, and Quality Operations. He spent the early part of his career in research and development focused on alphavirus vectors for vaccine use at AlphaVax.
Mr. Alterson holds a B.S. in Microbiology from Montana State University.
PIPELINE
Vector
Protein
Indication
KT-A112
AAV
Insulin + Glucokinase
Type 1 Diabetes Mellitus
Type 2 Diabetes Mellitus
Learn More
KT-A112 (AAV-Ins+Gck)
KT-A112 is an intramuscularly delivered gene therapy for the treatment of Type 1 and Type 2 Diabetes. The approach involves a one-time codelivery of the genetic material to produce insulin and glucokinase, packaged within an AAV vector and delivered directly to skeletal muscle. In the streptozotocin (STZ) induced diabetic mouse model, treated mice exhibited a dose-dependent improvement in glycemic control throughout the observation period. Furthermore, in an induced diabetic dog model, treated dogs followed for up to 8 years demonstrated dramatic and durable improvements in fasting blood glucose, insulinemia, fructosamine (a marker of blood glycosylated proteins), weight, and responsiveness to bolus glucose administration without the need for treatment with exogenous insulin.
[1] Jaén, Maria Luisa, et al. “Long-term efficacy and safety of insulin and glucokinase gene therapy for diabetes: 8-year follow-up in dogs.” Molecular Therapy-Methods & Clinical Development 6 (2017): 1-7.
[2] Callejas, David, et al. “Treatment of diabetes and long-term survival after insulin and glucokinase gene therapy.” Diabetes 62.5 (2013): 1718-1729.
[3] Mas, Alex, et al. “Reversal of type 1 diabetes by engineering a glucose sensor in skeletal muscle.” Diabetes 55.6 (2006): 1546-1553.
KT-A522
AAV
Glucagon Like Peptide
(GLP-1) receptor agonist
Type 2 Diabetes Mellitus
Obesity
Learn More
KT-A522 (AAV-GLP1-RA)
KT-A522 is a gene therapy administered directly to the salivary gland for the treatment of Type 2 Diabetes and Obesity. The approach involves a one-time delivery of the genetic material to produce a glucagon-like peptide 1 (GLP-1) receptor agonist, packaged within an AAV vector for continuous and tonic production of a GLP-1 receptor agonist to promote sustained and physiologic glucose control. In the Zucker fa/fa rat and high fed diet mouse models, treated animals achieved therapeutic circulating levels of the GLP-1 receptor agonist and experienced significant improvements in glycemic control and decreased weight gain.
[1] Di Pasquale, Giovanni, et al. “Sustained exendin-4 secretion through gene therapy targeting salivary glands in two different rodent models of obesity/type 2 diabetes.” PLoS One 7.7 (2012).
KT-A832
AAV
Insulin Growth Factor 1
Type 1 Diabetes Mellitus
Learn More
KT-A832 (AAV-IGF1)
KT-A832 is a gene therapy for the treatment of Type 1 Diabetes, designed for direct intrapancreatic delivery. The approach involves a one-time delivery of the genetic material to produce modified insulin growth factor 1 (IGF-1) packaged within an AAV vector to preserve pancreatic beta cell mass in the face of the autoimmune destruction that occurs in Type 1 Diabetes. In the non-obese diabetic (NOD) mouse model, treated mice demonstrated protection against the development of diabetes, together with preservation of pancreatic beta cell mass and insulin production.
[1] Mallol, Cristina, et al. “AAV-mediated pancreatic overexpression of Igf1 counteracts progression to autoimmune diabetes in mice.” Molecular metabolism 6.7 (2017): 664-680.
[2] Casellas, Alba, et al. “Expression of IGF-I in pancreatic islets prevents lymphocytic infiltration and protects mice from type 1 diabetes.” Diabetes 55.12 (2006): 3246-3255.
[3] George, Mónica, et al. “β cell expression of IGF-I leads to recovery from type 1 diabetes.” The Journal of clinical investigation 109.9 (2002): 1153-1163.
KT-A112
Vector
AAV
Protein
Insulin + Glucokinase
Indication
Type 1 Diabetes Mellitus
Type 2 Diabetes Mellitus
Learn More
KT-A112 is an intramuscularly delivered gene therapy for the treatment of Type 1 and Type 2 Diabetes. The approach involves a one-time codelivery of the genetic material to produce insulin and glucokinase, packaged within an AAV vector and delivered directly to skeletal muscle. In the streptozotocin (STZ) induced diabetic mouse model, treated mice exhibited a dose-dependent improvement in glycemic control throughout the observation period. Furthermore, in an induced diabetic dog model, treated dogs followed for up to 8 years demonstrated dramatic and durable improvements in fasting blood glucose, insulinemia, fructosamine (a marker of blood glycosylated proteins), weight, and responsiveness to bolus glucose administration without the need for treatment with exogenous insulin.
[1] Jaén, Maria Luisa, et al. “Long-term efficacy and safety of insulin and glucokinase gene therapy for diabetes: 8-year follow-up in dogs.” Molecular Therapy-Methods & Clinical Development 6 (2017): 1-7.
[2] Callejas, David, et al. “Treatment of diabetes and long-term survival after insulin and glucokinase gene therapy.” Diabetes 62.5 (2013): 1718-1729.
[3] Mas, Alex, et al. “Reversal of type 1 diabetes by engineering a glucose sensor in skeletal muscle.” Diabetes 55.6 (2006): 1546-1553.
KT-A522
Vector
AAV
Protein
Glucagon Like Peptide (GLP-1) receptor agonist
Indication
Type 2 Diabetes Mellitus
Obesity
Learn More
KT-A522 (AAV-GLP1-RA)
KT-A522 is a gene therapy administered directly to the salivary gland for the treatment of Type 2 Diabetes and Obesity. The approach involves a one-time delivery of the genetic material to produce a glucagon-like peptide 1 (GLP-1) receptor agonist, packaged within an AAV vector for continuous and tonic production of a GLP-1 receptor agonist to promote sustained and physiologic glucose control. In the Zucker fa/fa rat and high fed diet mouse models, treated animals achieved therapeutic circulating levels of the GLP-1 receptor agonist and experienced significant improvements in glycemic control and decreased weight gain.
[1] Di Pasquale, Giovanni, et al. “Sustained exendin-4 secretion through gene therapy targeting salivary glands in two different rodent models of obesity/type 2 diabetes.” PLoS One 7.7 (2012).
KT-A832
Vector
AAV
Protein
Insulin Growth Factor 1
Indication
Type 1 Diabetes Mellitus
Learn More
KT-A832 (AAV-IGF1)
KT-A832 is a gene therapy for the treatment of Type 1 Diabetes, designed for direct intrapancreatic delivery. The approach involves a one-time delivery of the genetic material to produce modified insulin growth factor 1 (IGF-1) packaged within an AAV vector to preserve pancreatic beta cell mass in the face of the autoimmune destruction that occurs in Type 1 Diabetes. In the non-obese diabetic (NOD) mouse model, treated mice demonstrated protection against the development of diabetes, together with preservation of pancreatic beta cell mass and insulin production.
[1] Mallol, Cristina, et al. “AAV-mediated pancreatic overexpression of Igf1 counteracts progression to autoimmune diabetes in mice.” Molecular metabolism 6.7 (2017): 664-680.
[2] Casellas, Alba, et al. “Expression of IGF-I in pancreatic islets prevents lymphocytic infiltration and protects mice from type 1 diabetes.” Diabetes 55.12 (2006): 3246-3255.
[3] George, Mónica, et al. “β cell expression of IGF-I leads to recovery from type 1 diabetes.” The Journal of clinical investigation 109.9 (2002): 1153-1163.
MANUFACTURING
Our 51,350 square foot manufacturing facility is located in Research Triangle Park, North Carolina, one of the global centers in the manufacturing of biological therapies. The facility is designed to have its own fully integrated process development lab, pilot production suite, and current good manufacturing practice (cGMP) production capability – and will support the manufacturing of material using our scalable suspension cell culture manufacturing process at up to 2,000-liter bioreactor scale. The facility’s pilot production suite and full cGMP manufacturing infrastructure are expected to be online in the first and second half of 2021, respectively.
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CAREERS
Interested in joining the team?
We are actively building our team and seeking high-quality candidates for research, manufacturing, and operational roles. If you are a motivated, ambitious person who is looking to make a difference in the lives of patients, please contact us.
