Phenylalanine hydroxylase

Orphan designation: Adeno-associated virus serotype HSC15, containing human homology arms, expressing human phenylalanine hydroxylase Treatment of phenylalanine hydroxylase deficiency, 16/03/2022 Withdrawn

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星期二, 四月 9, 2024
Diamond, Community, MC1R, Civil service commission, Narcolepsy, Diagnosis, Pigment, Erythropoietic protoporphyria, Prevalence, Disease, EPP, Patient, Committee, EMA, IRIS, Agency, European, Skin, COMP, Sponsor, European Commission, Pain, Phototoxicity, PAH, Marketing, Phenylalanine hydroxylase, Medicine, Pharmaceutical industry

Orphan designation: Adeno-associated virus serotype HSC15, containing human homology arms, expressing human phenylalanine hydroxylase Treatment of phenylalanine hydroxylase deficiency, 16/03/2022 Withdrawn

Key Points: 


Orphan designation: Adeno-associated virus serotype HSC15, containing human homology arms, expressing human phenylalanine hydroxylase Treatment of phenylalanine hydroxylase deficiency, 16/03/2022 Withdrawn

Agios Reports Business Highlights and Third Quarter 2023 Financial Results

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星期四, 十一月 2, 2023
Anti-transglutaminase antibodies, Research, RISE, PKU, Research and development, IND, AGIO, Rare, Sickle cell disease, MDS, Public expenditure, Enrollment, SG, Pyruvate kinase deficiency, R, Society, Phenylketonuria, ASH, Thalassemia, PK, Security, Abstract, Phenylalanine hydroxylase, TMPRSS6, Agios Pharmaceuticals, PAH, Agios, Patient, Administration, Nursing, Cryptocurrency, Pharmaceutical industry, Medical device, Video game, Alnylam Pharmaceuticals

CAMBRIDGE, Mass., Nov. 02, 2023 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, today reported business highlights and financial results for the third quarter ended September 30, 2023.

Key Points: 
  • Launch: Generated $7.4 million in U.S. net revenue for the third quarter of 2023, a 10 percent increase over the second quarter of 2023.
  • A total of 160 unique patients have completed prescription enrollment forms, representing an increase of 9 percent over the second quarter of 2023.
  • Net Loss: Net loss was $91.3 million for the third quarter of 2023 compared to $81.7 million for the third quarter of 2022.
  • ET to discuss third quarter 2023 financial results and recent business activities.

MediciNova’s Collaborator Initiates Clinical Development of a Gene Therapy Product for the Treatment of Phenylketonuria

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星期五, 十月 27, 2023
PKU, Standard Market Design, AAV, Tokyo Stock Exchange, Phenylketonuria, Genzyme, Clinical trial, Code, Phenylalanine hydroxylase, PAH, Pharmaceutical industry, Sanofi

LA JOLLA, Calif., Oct. 26, 2023 (GLOBE NEWSWIRE) -- MediciNova, Inc., a biopharmaceutical company traded on the NASDAQ Global Market (NASDAQ:MNOV) and the Standard Market of the Tokyo Stock Exchange (Code Number: 4875), today announced that its collaborator Genzyme Corporation, a subsidiary of Sanofi, has treated the first patient in a clinical trial of SAR444836 for the treatment of phenylketonuria (PKU).

Key Points: 
  • LA JOLLA, Calif., Oct. 26, 2023 (GLOBE NEWSWIRE) -- MediciNova, Inc., a biopharmaceutical company traded on the NASDAQ Global Market (NASDAQ:MNOV) and the Standard Market of the Tokyo Stock Exchange (Code Number: 4875), today announced that its collaborator Genzyme Corporation, a subsidiary of Sanofi, has treated the first patient in a clinical trial of SAR444836 for the treatment of phenylketonuria (PKU).
  • SAR444836 is a phenylalanine hydroxylase (PAH) replacement gene therapy product based on adeno-associated virus (AAV) vector technology which is covered under MediciNova’s assignment agreement with Genzyme Corporation.

Poseida Therapeutics Provides Updates and Financial Results for the First Quarter of 2023

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星期二, 五月 9, 2023
Lymphoma, Amgen, Executive, Partnership, Drug development, Phenylalanine hydroxylase, Gene, CD19, Kite Pharma, Takeda, Lymphoid leukemia, CD20, Ornithine, PAH, Epithelium, Transposase, ESMO, Roche, Cancer, Breast, Oncology, Biomarker, Cell, AAV, American Society of Gene and Cell Therapy, PKU, Phenylalanine, Research and development, CAR, Development, Retirement, Genetic disorder, Novartis, Patient, Investment, SAN, Haemophilia, OTC, Mouse, Rare, European Society for Medical Oncology, Therapy, IND, News, Society, Pharmaceutical industry, Vaccine, Cryptocurrency, Research, Phenylketonuria

SAN DIEGO, May 9, 2023 /PRNewswire/ -- Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage cell and gene therapy company advancing a new class of treatments for patients with cancer and rare diseases, today announced updates and financial results for the first quarter ended March 31, 2023.  

Key Points: 
  • SAN DIEGO, May 9, 2023 /PRNewswire/ -- Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage cell and gene therapy company advancing a new class of treatments for patients with cancer and rare diseases, today announced updates and financial results for the first quarter ended March 31, 2023.
  • The Company currently expects to present further clinical updates for the program at a medical meeting in 2023.
  • The Company currently expects to present further clinical updates for the program at a medical meeting in 2023, subject to clearance with Roche.
  • Net loss was $38.8 million for the first quarter ended March 31, 2023, compared to $58.1 million for the same period of 2022.

Agios Reports Business Highlights and First Quarter 2023 Financial Results

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星期四, 五月 4, 2023
Glioma, PK, Thalassemia, Phenylalanine hydroxylase, SG, Anti-transglutaminase antibodies, PAH, Phenylketonuria, Sickle cell disease, Sale, PKU, AGIO, MDS, Agios, Patient, Administration, RISE, IDH, Biophysical environment, ESG, Research and development, Agios Pharmaceuticals, Rare, FDA, Research, R, Security, IND, Myelodysplastic syndrome, Cryptocurrency, Pharmaceutical industry, Nursing, Electric motor, Video game, Social

CAMBRIDGE, Mass., May 04, 2023 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, today reported business highlights and financial results for the first quarter ended March 31, 2023.

Key Points: 
  • Launch: Generated $5.6 million in U.S. net revenue for the first quarter of 2023, the fourth full quarter following FDA approval.
  • A total of 89 patients are on PYRUKYND® therapy, representing a 14 percent increase over the fourth quarter of 2022.
  • Net Loss: Net loss was $81.0 million for the first quarter of 2023 compared to $94.8 million for the first quarter of 2022.
  • ET to discuss first quarter 2023 financial results and recent business activities.

Agios Reports Fourth Quarter and Full Year 2022 Financial Results

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星期四, 二月 23, 2023
SG, Sickle cell disease, Rare, Exposition, AGIO, Continuous operation, Workforce, IND, Agios, Security, Phenylalanine hydroxylase, FDA, Anti-transglutaminase antibodies, PAH, Patient, Phenylketonuria, Agios Pharmaceuticals, R, ASH, Growth, Research and development, PKU, Thalassemia, Hematology, Sale, RISE, Administration, PK, Myelodysplastic syndrome, Society, Pyruvate kinase, Pharmaceutical industry, Dietary supplement, Electric motor, Video game, EU

CAMBRIDGE, Mass., Feb. 23, 2023 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, today reported business highlights and financial results for the fourth quarter and year ended Dec. 31, 2022.

Key Points: 
  • Launch: Continued to execute launch, generating $4.3 million in U.S. net revenue for the fourth quarter of 2022, the third full quarter following FDA approval.
  • Revenue: Net U.S. product revenue from sales of PYRUKYND® was $4.3 million for the fourth quarter of 2022, and $11.7 million for the full year ended Dec. 31, 2022.
  • Cost of Sales: Cost of sales was $0.4 million for the fourth quarter of 2022 and $1.7 million for the full year ended Dec. 31, 2022.
  • ET to discuss fourth quarter and year end 2022 financial results and recent business activities.

Poseida Therapeutics Hosts Third Annual Virtual R&D Day Highlighting Novel Pipeline Assets and Latest Technology Innovations

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星期三, 二月 22, 2023
DNA, Science, City, OTC, AAV, Roche, Synthetic biology, Mouse, Oncology, Breast, Genetic engineering, Ammonia, Patient, TCR, Gene, PAH, Canadian Aviation Regulations, Webcast, European Society of Gene and Cell Therapy, Therapy, Genetics, Rare, Phenylalanine hydroxylase, Phenylalanine, DSM-IV codes, SAN, Epithelium, ESMO, European Society for Medical Oncology, PKU, Genetic disorder, Cancer, Pharmaceutical industry, Vaccine, Phenylketonuria, Takeda

SAN DIEGO, Feb. 22, 2023 /PRNewswire/ -- Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage cell and gene therapy company advancing a new class of treatments for patients with cancer and rare diseases, today announced that the Company plans to highlight its clinical and preclinical pipeline progress during a virtual R&D Day to be held today at 10:00am ET / 7:00am PT.

Key Points: 
  • "R&D Day is our annual showcase for the innovative and exciting science we are advancing at Poseida that continues to drive our leadership in the field of cell and gene therapies," said Mark Gergen, Chief Executive Officer of Poseida Therapeutics.
  • The Company will present advancements in hybrid technology highlighting the potential for single treatment cures across multiple diseases.
  • Site-specific Super piggyBac DNA Delivery, first unveiled at the Company's R&D Day in February 2022, has continued to advance.
  • Registration for this virtual event and access to the live webcast will be available on the Investors & Media section of the Company's website, www.poseida.com .

Evozyne Creates AI Model with NVIDIA for Novel Protein Design, Expected to Accelerate Drug Development

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星期四, 一月 12, 2023
Paragon, Machine learning, AI, Gene editing, Drug development, NVIDIA, Biology, Phenylalanine hydroxylase, Protein, Conference, Imagination, Health, Therapy, Engineering, PAH, Research, Deep learning, Nature, Genetic disorder, Medical device, Vaccine, Antibiotics, Fine chemical

CHICAGO, Jan. 12, 2023 /PRNewswire/ -- Evozyne today revealed a breakthrough AI model, developed in collaboration with NVIDIA, that the pharmaceutical industry can use to design therapeutic proteins.

Key Points: 
  • New AI model, co-developed by Evozyne and NVIDIA, can create novel therapeutic proteins for a new era of drug development
    CHICAGO, Jan. 12, 2023 /PRNewswire/ -- Evozyne today revealed a breakthrough AI model, developed in collaboration with NVIDIA, that the pharmaceutical industry can use to design therapeutic proteins.
  • Evozyne began a collaboration with NVIDIA in 2022 to develop a new deep learning model that can learn the rules of protein function and use these rules to design new proteins with improved functions.
  • The model, known as the Protein Transformer Variational AutoEncoder (ProT-VAE), is built on NVIDIA BioNeMo , a framework for efficiently training and deploying large language models for biology.
  • Using the Evozyne ProT-VAE model and NVIDIA BioNeMo framework, the team designed new synthetic PAH variants predicted to have "supernatural" function.

Homology Medicines Announces World’s First Gene Editing Clinical Trial for PKU

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星期二, 十月 12, 2021
Private Securities Litigation Reform Act, Company, Brain, National, PNH, Data, Adult, Liver, COVID-19, Homologous recombination, Antibody, Food, Cell, Metabolism, GLOBE, Blood, Failure, Institutional review board, Safety, Medical Affairs Bureau, European Medicines Agency, Population, SEC, FDA, Genome, News, Mutation, Gene editing, Prevalence, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Phenix, EMA, Hunter syndrome, Fast track (FDA), PKU, Phenylketonuria, Therapy, Phenylalanine hydroxylase, Solution, Cell division, IND, DNA, Phenylalanine, Patient, Clinical trial, Diet, Disease, Technology, Paroxysmal nocturnal hemoglobinuria, Operation, Nasdaq, Pharmaceutical industry, Vaccine

BEDFORD, Mass., Oct. 12, 2021 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today the pheEDIT Phase 1 clinical trial for HMI-103, a one-time, in vivo product candidate that utilizes a gene editing approach for phenylketonuria (PKU), based on the Investigational New Drug Application (IND) clearance from the U.S. Food and Drug Administration (FDA). HMI-103 will be the world’s first gene editing candidate for PKU to enter clinical trials from Homology’s dual gene therapy and gene editing technology platform, and with the launch of pheEDIT Homology moves closer to its goal of offering solutions for both adults and pediatric patients with PKU.

Key Points: 
  • HMI-103 will be the worlds first gene editing candidate for PKU to enter clinical trials from Homologys dual gene therapy and gene editing technology platform, and with the launch of pheEDIT Homology moves closer to its goal of offering solutions for both adults and pediatric patients with PKU.
  • Todays milestone is the culmination of our teams tireless work to translate our gene editing technology from an academic discovery into a clinical program for people with PKU, said Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines.
  • Homology expects that the first patient in the pheEDIT clinical trial will be dosed following requisite Institutional Biosafety Committee and Institutional Review Board approvals at the clinical sites.
  • Homology also announced today an update from its ongoing Phase 2 pheNIX clinical trial evaluating HMI-102 gene therapy in adults with PKU.

Claritas Announces Additional Data in Validated Animal Model of PAH Demonstrating Ability of R-107 to Prevent, Treat, and Reverse Established Disease

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星期三, 七月 21, 2021
Phenylalanine hydroxylase

R-107 is the first and only drug to demonstrate a durable reversal of established disease in a validated animal model of PAH.

Key Points: 
  • R-107 is the first and only drug to demonstrate a durable reversal of established disease in a validated animal model of PAH.
  • As previously disclosed, R-107 was evaluated in a validated animal model of PAH.
  • Following are additional details regarding these data:
    Prevention of Disease Progression: The data demonstrate that R-107 therapy halts the otherwise unstoppable progression of PAH.
  • For the reasons discussed above, Claritas will also develop R-107 as a nitric oxide therapy for PAH, including life-threatening COVID-19 associated PAH.