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Lead Product(s): Hydrocortisone
Therapeutic Area: Genetic Disease Product Name: Chronocort
Highest Development Status: Phase III Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 02, 2021
Details:
The Phase 3 study results published by the JCEM found that although the standard-deviation-score-focused primary endpoint of the study was missed, Chronocort® improved morning and early afternoon biochemical control for adults with CAH over standard glucocorticoid therapy.
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Lead Product(s): Hydrocortisone
Therapeutic Area: Genetic Disease Product Name: Chronocort
Highest Development Status: Phase III Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 12, 2021
Details:
The submission for the MHRA is based on the data from the Company’s Phase 3 study, an open-label safety extension study of Chronocort® and written formal Scientific Advice received in April 2019 confirming the clinical and regulatory pathway for Chronocort® for CAH treatment.
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Lead Product(s): LentiGlobin Gene Therapy
Therapeutic Area: Genetic Disease Product Name: Bb1111
Highest Development Status: Phase III Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable December 07, 2020
Details:
Positive patient-reported quality of life outcomes assessed with validated PROMIS-57 demonstrate clinically meaningful reductions in pain intensity at Month 12 post-LentiGlobin for SCD treatment.
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Lead Product(s): Betibeglogene autotemcel
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase III Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable December 05, 2020
Details:
Among 32 patients enrolled in the 13-year, long-term LTF-303 study, 64% of patients treated in the phase 1/2 portion and 90% in the phase 2 portion achieved transfusion independence (TI) after treatment with beti-cel.
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Lead Product(s): OTL-200
Therapeutic Area: Genetic Disease Product Name: OTL-200
Highest Development Status: Phase III Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 19, 2020
Details:
FDA has cleared the company’s Investigational New Drug application for OTL-200, an autologous, hematopoietic stem cell, lentiviral vector-based gene therapy in development for the treatment of metachromatic leukodystrophy.
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Lead Product(s): LentiGlobin,Plerixafor
Therapeutic Area: Genetic Disease Product Name: BB305
Highest Development Status: Phase III Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 04, 2020
Details:
bluebird bio announces confirmation of its general agreement with the U.S.FDA that the clinical data package required to support a BLA submission for LentiGlobin™ for sickle cell disease will be based on data from a portion of patients in the HGB-206 study Group C.
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Lead Product(s): Libmeldy
Therapeutic Area: Genetic Disease Product Name: OTL-200
Highest Development Status: Phase III Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 03, 2020
Details:
Libmeldy TM receives positive CHMP opinion for the treatment of Early-onset metachromatic leukodystrophy (MLD); U.S. IND application on Track for Year End 2020.
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Lead Product(s): Libmeldy
Therapeutic Area: Genetic Disease Product Name: OTL-200
Highest Development Status: Phase III Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable October 16, 2020
Details:
The CHMP’s positive opinion will now be reviewed by the European Commission (EC), which has the authority to grant marketing authorization for Libmeldy in the European Union (EU).
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Lead Product(s): Elivaldogene autotemcel
Therapeutic Area: Genetic Disease Product Name: Lenti-D
Highest Development Status: Phase III Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable October 02, 2020
Details:
Data from the Phase 2/3 Starbeam study (ALD-102) formed the basis of the MAA, which is also supported with data from the ongoing Phase 3 ALD-104 study and the long-term follow-up study (LTF-304).
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Lead Product(s): LentiGlobin
Therapeutic Area: Genetic Disease Product Name: BB305
Highest Development Status: Phase III Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 23, 2020
Details:
Clinical data from the completed Phase 1/2 HGB-205 study, the ongoing Phase 1/2 HGB-206 study and ongoing long-term safety and efficacy follow-up study LTF-303 supported the PRIME application for LentiGlobin for SCD.
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