Drugs in Dev. Genetic Disease
Undisclosed
United Kingdom 
Lead Product(s) : PKU Easy Microtabs Plus
Therapeutic Area : Genetic Disease
Study Phase : Undisclosed
Sponsor : Birmingham Women's and Children's NHS Foundation Trust
Deal Size : Inapplicable
Deal Type : Inapplicable
PKU EASY Microtabs Plus - Acceptability and Tolerance
Details : PKU Easy Microtabs Plus is a Dietary Supplement drug candidate, which is currently being evaluated in clinical studies for the treatment of Phenylketonurias.
Product Name : Undisclosed
Product Type : Dietary Supplement
Upfront Cash : Inapplicable
November 19, 2024
Lead Product(s) : PKU Easy Microtabs Plus
Therapeutic Area : Genetic Disease
Highest Development Status : Undisclosed
Sponsor : Birmingham Women's and Children's NHS Foundation Trust
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Lanadelumab
Therapeutic Area : Genetic Disease
Study Phase : Undisclosed
Sponsor : Takeda Pharmaceutical
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Lanadelumab is a Antibody drug candidate, which is currently being evaluated in clinical studies for the treatment of Angioedemas, Hereditary.
Product Name : Undisclosed
Product Type : Antibody, Unconjugated
Upfront Cash : Inapplicable
October 12, 2020
Lead Product(s) : Lanadelumab
Therapeutic Area : Genetic Disease
Highest Development Status : Undisclosed
Sponsor : Takeda Pharmaceutical
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : OTL-203
Therapeutic Area : Genetic Disease
Study Phase : Undisclosed
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Orchard Therapeutics Receives EMA PRIME Designation for OTL-203 for the Treatment of MPS-I
Details : EMA has granted Priority Medicines designation to OTL-203, an investigational ex vivo autologous hematopoietic stem cell gene therapy in development for the treatment of mucopolysaccharidosis type I at the San Raffaele Telethon Institute for Gene Therapy...
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
September 28, 2020
Lead Product(s) : OTL-203
Therapeutic Area : Genetic Disease
Highest Development Status : Undisclosed
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : OTL-203
Therapeutic Area : Genetic Disease
Study Phase : Undisclosed
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : New preliminary analyses of radiological outcome measures suggest that treatment with OTL-203 leads to stabilization or improvement in disease-related neurological abnormalities, as measured by brain and spine MRI.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
September 01, 2020
Lead Product(s) : OTL-203
Therapeutic Area : Genetic Disease
Highest Development Status : Undisclosed
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : OTL-203
Therapeutic Area : Genetic Disease
Study Phase : Undisclosed
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Orchard Therapeutics Announces Clinical Data Presentations at the 46th Annual Meeting of EBMT
Details : New interim data from OTL-203, an investigational gene therapy for the treatment of mucopolysaccharidosis type I (MPS-I), will be shared as part of an invited oral presentation titled ‘Gene Therapy in Leucodystrophies and Other Metabolic Disorders’.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
August 24, 2020
Lead Product(s) : OTL-203
Therapeutic Area : Genetic Disease
Highest Development Status : Undisclosed
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : OTL-203
Therapeutic Area : Genetic Disease
Study Phase : Undisclosed
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Orchard Therapeutics has received both orphan drug designation and rare pediatric disease designation from the U.S FDA for OTL-203, an ex vivo autologous hematopoietic stem cell (HSC) gene therapy in development for the treatment of mucopolysaccharidosis...
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
July 20, 2020
Lead Product(s) : OTL-203
Therapeutic Area : Genetic Disease
Highest Development Status : Undisclosed
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : OTL-203
Therapeutic Area : Genetic Disease
Study Phase : Undisclosed
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Orchard Therapeutics Presents New Interim Data from OTL-203 Proof-of-concept Study for MPS-I
Details : MPS-I clinical trial interim results demonstrated- all patients, treated with OTL-203, achieving hematological recovery in less than 45 days – a primary outcome measure – as well as supraphysiological levels of IDUA enzyme in the blood.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
May 15, 2020
Lead Product(s) : OTL-203
Therapeutic Area : Genetic Disease
Highest Development Status : Undisclosed
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : OTL-203
Therapeutic Area : Genetic Disease
Study Phase : Undisclosed
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Accepted abstracts include data on investigational gene therapies for neurometabolic conditions, including updated interim data from proof-of-concept trial in OTL-203 for Mucopolysaccharidosis Type I.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
April 29, 2020
Lead Product(s) : OTL-203
Therapeutic Area : Genetic Disease
Highest Development Status : Undisclosed
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : OTL-102
Therapeutic Area : Genetic Disease
Study Phase : Undisclosed
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Early clinical data support ex vivo hematopoietic stem cell gene therapy as a potentially promising treatment option for X-CGD.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
January 29, 2020
Lead Product(s) : OTL-102
Therapeutic Area : Genetic Disease
Highest Development Status : Undisclosed
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Lofexidine
Therapeutic Area : Genetic Disease
Study Phase : Undisclosed
Sponsor : Lucas Otieno Tina, MD MSc
Deal Size : Inapplicable
Deal Type : Inapplicable
Zinc Supplementation in Children With Sickle Cell Disease in Western Kenya
Details : Lofexidine is a Other Small Molecule drug candidate, which is currently being evaluated in clinical studies for the treatment of Anemia, Sickle Cell.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
September 26, 2017
Lead Product(s) : Lofexidine
Therapeutic Area : Genetic Disease
Highest Development Status : Undisclosed
Sponsor : Lucas Otieno Tina, MD MSc
Deal Size : Inapplicable
Deal Type : Inapplicable
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