49 reports

  • Gene therapy trends
  • Personalized/ precision medicine

Genome sequencing The process of determining the order of DNA bases in a genome.

  • Gene Therapy
  • United States
  • AbbVie Inc.
  • Amgen Inc.
  • Bristol-Myers Squibb Company
  • Global Cell and Gene Therapy Market by Other Applications 2018−2024 ($ million)
  • 21.22.2 Major Product Offerings

CRITICAL DO NOT HAVE A Β##/ Β## GENOTYPE.

  • Gene Therapy
  • World
  • Market Size
  • Gilead Sciences, Inc.
  • Novartis AG
  • LYS-SAF302 PHASE II/III TRIAL DESIGN
  • CTX-001 TRIAL DESIGN (LEFT) AGAINST ST-400 AND LENTIGLOBIN (RIGHT)

Results were presented for the two genotype subgroups.

  • Gene Therapy
  • Casebia Therapeutics LLP
  • Editas Medicine, Inc.
  • Novartis AG
  • Pfizer Inc.
  • 18. EXECUTIVE INSIGHTS

RF=## LANDSCAPE ##. ##.

  • Gene Therapy
  • Market Size
  • Supply
  • Lonza Group Ltd
  • Sanofi S.A.

Viral Vectors for Gene Therapy. ## (##), ##-##.

  • Gene Therapy
  • CAP-1002 group
  • Capricor Therapeutics, Inc.
  • Mirna Therapeutics, Inc.
  • ReNeuron Group plc

(USA) ##.

  • Gene Therapy
  • Genome Sequencing
  • Genomics Proteomics And Enabling Technology
  • World
  • Market Shares
  • EQUITY OFFERING
  • BLUEBIRD BIO RAISES USD651.3 MILLION IN PUBLIC OFFERING OF SHARES

LentiGlobin is also being investigated in Northstar-## (HGB-##), a Phase ## study in patients with TDT and non-ß##/ ß## genotypes, Northstar (HGB-##), a Phase ##/ ## study in patients with TDT and all genotypes, HGB-##, a Phase ## study in patients with TDT an

  • Gene Therapy
  • Pharmaceutical
  • Therapy
  • United States
  • Celgene Corporation

It outperforms comparative technologies in a number of applications, including our gene expression based RNA QC method and SNP genotyping.

  • Gene Therapy
  • Medical Biotechnology
  • United States
  • Company
  • Takara Bio Inc.
  • GENE THERAPY TO ACTIVATE CLN6 FOR BATTEN DISEASE - DRUG PROFILE
  • GENE THERAPY TO ACTIVATE TRIPEPTIDYL PEPTIDASE 1 FOR BATTEN DISEASE - DRUG PROFILE

SPKTPP-## acts as tripeptidyl peptidase ## activator.

  • Dementia
  • Gene Therapy
  • Therapy
  • United States
  • Product Initiative

TAKING INTO ACCOUNT AGE, BASELINE WALKING ABILITY AND GENOTYPE, BRINEURA-TREATED PATIENTS DEMONSTRATED FEWER DECLINES IN WALKING ABILITY COMPARED TO UNTREATED PATIENTS IN THE NATURAL HISTORY COHORT.

  • Gene Therapy
  • Pharmaceutical
  • Therapy
  • United States
  • BioMarin Pharmaceutical Inc.

Similarly, the universities portfolio in Preclinical and Discovery stages comprises ## and ## molecules, respectively.

  • Gene Therapy
  • Pharmaceutical
  • United States
  • World
  • Product Initiative

Similarly, the Universities' portfolio in Phase II, Phase I and Preclinical stages comprises ##, ## and ## molecules, respectively.

  • Gene Therapy
  • Neurological Disorder
  • Therapy
  • United States
  • Product Initiative

Similarly, the universities portfolio in Preclinical and Discovery stages comprises ## and ## molecules, respectively.

  • Gene Therapy
  • Pharmaceutical
  • Therapy
  • United States
  • Product Initiative
  • Nov 01, 2017: bluebird bio to Present New Data from Clinical Studies of LentiGlobin Gene Therapy in Transfusion-Dependent ß-Thalassemia at ASH Annual Meeting
  • GENE THERAPY FOR SICKLE CELL DISEASE AND THALASSEMIA - DRUG PROFILE

LentiGlobin is also being investigated in Northstar-## (HGB-##), a Phase ## study in patients with TDT and non-ß##/ ß## genotypes, Northstar (HGB-##), a Phase ##/ ## study in patients with TDT and all genotypes, HGB-##, a Phase ## study in patients with TDT an

  • Blood Disease
  • Gene Therapy
  • United States
  • Product Initiative
  • Incyte Corporation

The new facility will have broad genetic research capabilities and the capacity to launch and complete whole-genome sequencing projects rapidly.

  • Gene Therapy
  • Medical Biotechnology
  • Pharmaceutical
  • Therapy
  • Biogen Idec Inc.

In the ACHM CNGA## trial, the company has enrolled two patients in the low dose group and its first international site in Israel has begun genotyping and screening their patient pool in order to begin enrollment.

  • Gene Therapy
  • Therapy
  • United States
  • Company Operations
  • Applied Genetic Technologies Corporation

## ## Preclinical ## ## Discovery ## ## Total ## ## Products in Phase II/ III and I/ II are reflected under Phase III and Phase II, respectively Source: Global Markets Direct NUMBER OF PRODUCTS UNDER DEVELOPMENT FOR MUCOPOLYSACCHARIDOSIS I (MPS I) (HURLER SYNDROME ), H## 2017 Stage

  • Gene Therapy
  • Pharmaceutical
  • United States
  • Product Initiative
  • PTC Therapeutics, Inc.

Patients displayed a range of CF genotype mutations in ##-Mar-2018 ##-Mar-2018 the CFTR.

  • Gene Therapy
  • Therapy
  • United States
  • Company
  • Galapagos NV

It offers personalized tumorgraft development, drug studies and genome sequencing as part of personalized oncology solutions.

  • Gene Therapy
  • Medical Biotechnology
  • Therapy
  • United States
  • Pfizer Inc.

A study in patients with the rare point mutation genotype is actively enrolling subjects.

  • Gene Therapy
  • Pharmaceutical
  • United States
  • Company Operations
  • Product Initiative

A Phase ##/ ## clinical trial of the drug is currently underway in Japan.

  • Gene Therapy
  • Therapy
  • United States
  • Product Initiative
  • Sarepta Therapeutics, Inc.

The molecules developed by companies in Phase II, Phase I, IND/ CTA Filed, Preclinical and Discovery stages are ##, ##, ##, ## and ## respectively.

  • Gene Therapy
  • Pathology
  • Psychotic Disorder
  • Therapy
  • Protalix BioTherapeutics, Inc.
  • GENE THERAPY FOR SICKLE CELL DISEASE AND THALASSEMIA - DRUG PROFILE
  • BB-305 - DRUG PROFILE

Patients with the HbSS genotype, and non-HbSS genotypes.

  • Blood Disease
  • Gene Therapy
  • United States
  • Product Initiative
  • Novartis AG

This multiple-dose study will assess the safety, tolerability, efficacy, and pharmacokinetics of SRP-## in DMD patients with genotypes amenable to exon-## skipping.

  • Cell Therapy
  • Gene Therapy
  • United States
  • Product Initiative
  • Sarepta Therapeutics, Inc.

Given that genotyping in IRDs has only recently been added to the diagnostic armamentarium, comprehensive natural history studies in patients with autosomal recessive mutations in RPE## had been lacking, " said Katherine A.

  • Gene Therapy
  • Therapy
  • United States
  • Company Operations
  • Spark Therapeutics

The Phase II clinical trial will evaluate the safety, tolerability and efficacy of PF-## in boys aged ## to less than ## years old diagnosed with DMD regardless of genotype.

  • Gene Therapy
  • Therapy
  • United States
  • Company
  • Product Initiative

A single ## mg (base equivalent) dose was given to subjects prospectively identified as slow and rapid acetylators by phenotype and genotype.

  • Gene Therapy
  • Neurological Disorder
  • Therapy
  • United States
  • Product Initiative

ONE PATIENT IN HGB-## IS HOMOZYGOUS FOR THE IVS##-## MUTATION AND HAS A SEVERE CLINICAL PRESENTATION SIMILAR TO THAT SEEN IN SS##/ SS## GENOTYPES.

  • Blood Disease
  • Gene Therapy
  • Pharmaceutical
  • Therapy
  • Product Initiative
  • GENE THERAPY TO ACTIVATE TRIPEPTIDYL PEPTIDASE 1 FOR BATTEN DISEASE - DRUG PROFILE

The molecules developed by Companies in Phase II, Preclinical and Discovery stages are ##, ## and ## respectively.

  • Dementia
  • Gene Therapy
  • United States
  • Product Initiative
  • Abeona Therapeutics, Inc.

Alpha-## antitrypsin deficiency, or " alpha-##, is a single gene disorder.

  • Gene Therapy
  • Healthcare
  • Pharmaceutical
  • Therapy
  • United States