Eladocagene exuparvovec

Global Cell and Gene Therapy Markets Report 2024-2028: Focus on Zolgensma, Yescarta, Kymriah, Abecma, Maci, Tecartus, Epicel - ResearchAndMarkets.com

Retrieved on: 
Wednesday, April 3, 2024
Health, Genetics, Growth, PTC Therapeutics, Prevalence, Gene editing, ESG, CSL Behring, Roche, Etranacogene dezaparvovec, Gene, Gilead Sciences, Cancer, Eladocagene exuparvovec, Therapy, CRISPR, Mallinckrodt, PTC, Probability, BLA, Pharmaceutical industry

The "Global Cell and Gene Therapy Market" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Global Cell and Gene Therapy Market" report has been added to ResearchAndMarkets.com's offering.
  • The Global Cell and Gene Therapy Market is expected to grow at a compound annual growth rate (CAGR) of 26.40% from 2023 through 2028.
  • The scope of this study encompasses an investigation of the cell and gene therapy market.
  • The report analyzes cell and gene therapy market based on therapy type, product, application, and region.

PTC Therapeutics Provides Key Regulatory Updates

Retrieved on: 
Tuesday, March 19, 2024
PTC Therapeutics, MAA, Ataluren, Patient, PKU, NDA, EMA, Eladocagene exuparvovec, Duchenne muscular dystrophy, Therapy, PTC, FDA, AADC, BLA, Pharmaceutical industry

SOUTH PLAINFIELD, N.J., March 19, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the submission of a BLA to the U.S. FDA for Upstaza™ (eladocagene exuparvovec), a gene therapy for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency. In addition, based on recent feedback from the FDA, PTC plans to re-submit an NDA for Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). PTC expects to make the NDA resubmission by mid-year.

Key Points: 
  • SOUTH PLAINFIELD, N.J., March 19, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the submission of a BLA to the U.S. FDA for Upstaza™ (eladocagene exuparvovec), a gene therapy for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency.
  • In addition, based on recent feedback from the FDA, PTC plans to re-submit an NDA for Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD).
  • PTC expects to make the NDA resubmission by mid-year.
  • The data collected to date continue to demonstrate the transformative clinical benefits the gene therapy provides to patients," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics.

PTC Therapeutics Provides an Update on Commercial Progress and R&D Pipeline at 42nd Annual J.P. Morgan Healthcare Conference

Retrieved on: 
Monday, January 8, 2024
Nikolaus Friedreich, Regulation, Conference, Eladocagene exuparvovec, Economics, PTC, EMA, FDA, MAA, Friedreich's ataxia, Duchenne muscular dystrophy, FA, NDA, Deflazacort, Prescription, HD, Civil service commission, Therapy, Bank statement, PTC Therapeutics, Cardinal, DMD, PKU, European, Duchenne, AADC, Patient, ALS, GAAP, CHMP, BLA, Pharmaceutical industry

SOUTH PLAINFIELD, N.J., Jan. 8, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) will present an update on its commercial progress and R&D pipeline at the 42nd Annual J.P. Morgan Healthcare Conference today, Monday, January 8, at 10:30am EST/7:30am PST. Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics, will provide an update on 2023 accomplishments and highlight potential 2024 value-creating milestones. Preliminary 2023 unaudited financial results and 2024 financial guidance will also be provided. The presentation is being webcast live on the Events and Presentations page of the Investors section of PTC Therapeutics website at www.ptcbio.com.

Key Points: 
  • Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics, will provide an update on 2023 accomplishments and highlight potential 2024 value-creating milestones.
  • The presentation is being webcast live on the Events and Presentations page of the Investors section of PTC Therapeutics website at www.ptcbio.com .
  • PTC announced strategic portfolio prioritizations, which resulted in reductions in both operating expenses and headcount of approximately 25% and 30%, respectively.
  • PTC maintains all economics associated with up to $250 million in the remaining commercial sales milestones associated with Evrysdi global net sales.

PTC Therapeutics Announces Strategic Pipeline Prioritization

Retrieved on: 
Tuesday, May 23, 2023
Workforce, PTC Therapeutics, Friedreich's ataxia, PTC, Research, Brain, Angelman syndrome, EDT, CNS, DSM-IV codes, Patient, OPEX, Growth, Therapy, News, Conference, Pharmaceutical industry, Eladocagene exuparvovec

SOUTH PLAINFIELD, N.J., May 23, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the discontinuation of preclinical and early research programs in gene therapy as part of a strategic portfolio prioritization. PTC will continue its development and global commercialization of Upstaza, the first-ever approved gene therapy directly administered to the brain. As a result of the prioritization, estimated reductions of approximately fifteen percent in residual 2023 operating expenses (OPEX) are expected. Additional 2023 OPEX guidance will be provided as part of second quarter earnings.

Key Points: 
  • SOUTH PLAINFIELD, N.J., May 23, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the discontinuation of preclinical and early research programs in gene therapy as part of a strategic portfolio prioritization.
  • PTC will continue its development and global commercialization of Upstaza, the first-ever approved gene therapy directly administered to the brain.
  • As a result of the prioritization, estimated reductions of approximately fifteen percent in residual 2023 operating expenses (OPEX) are expected.
  • In addition, PTC Therapeutics also announced today that PTC's Chief Financial Officer Emily Hill has been relieved of her responsibilities and will be leaving the organization.

Stuart Peltz, Ph.D., Rare Disease Pioneer, to Retire as Chief Executive Officer of PTC Therapeutics

Retrieved on: 
Friday, March 24, 2023
Yale University, Mitochondrion, PTC Therapeutics, Drug development, University, Gene, The Conference Board, Conference call, PTC, Cancer, Eladocagene exuparvovec, Coronavirus Scientific Advisory Board, European Research Advisory Board, Brain, CMO, RNA, History, CNS, DSM-IV codes, EMA, Bachelor of Arts in Clinical Psychology, Biosensors and Bioelectronics, Shriners Hospitals for Children, ET, Retirement, Oxidative stress, Consultant, Patient, University of Washington Department of Global Health, Stuart, Growth, Mitochondrial disease, Rare, Therapy, Scientific Advisory Group for Emergencies, Conference, Stu, Management, Pharmaceutical industry, Vaccine, Nursing

SOUTH PLAINFIELD, N.J., March 24, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT). Stuart Peltz, Ph.D., founding Chief Executive Officer (CEO) of PTC Therapeutics announced today that he is stepping down after 25 years. Dr. Peltz has led PTC Therapeutics as its CEO since its founding in 1998 and pioneered the field of RNA-directed drug development. Under his leadership, PTC has grown from a research organization with expertise in the control of RNA processes to a publicly traded, integrated, global biopharmaceutical company focused on the discovery, development and commercialization of innovative therapies for the treatment of neurological and neuromuscular disorders, metabolic disorders, and cancer.

Key Points: 
  • Stuart Peltz, Ph.D., founding Chief Executive Officer (CEO) of PTC Therapeutics announced today that he is stepping down after 25 years.
  • Dr. Peltz has led PTC Therapeutics as its CEO since its founding in 1998 and pioneered the field of RNA-directed drug development.
  • What Stuart and the team have achieved in 25 years is extraordinary," said Michael Schmertzler, Chairman of the Board of Directors.
  • Matthew Klein, M.D., M.S., F.A.C.S., current Chief Operating Officer, has been named the company's CEO and will join the Board of Directors.