X
[{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orchard Therapeutics Receives Positive CHMP Opinion for Libmeldy\u2122 for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Orchard Therapeutics"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orchard Therapeutics Reviews Recent Portfolio Progress and Reports Third Quarter 2020 Financial Results","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Orchard Therapeutics"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orchard Therapeutics Announces FDA Clearance of IND Application for OTL-200 for Metachromatic Leukodystrophy (MLD)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Orchard Therapeutics"},{"orgOrder":0,"company":"Diurnal Group","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Diurnal Seeks UK MHRA Marketing Approval for Chronocort to Treat Patients with Rare Condition Congenital Adrenal Hyperplasia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"Approved","date":"January 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Diurnal Group"},{"orgOrder":0,"company":"Diurnal Group","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Diurnal Group plc - Chronocort\u00ae Phase 3 and Safety Extension Study Results Published in JCEM","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"Approved","date":"February 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Diurnal Group"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AstraZeneca\u2019s Phase III FoCus Trial of ALXN1840 to Treat Wilson Disease Meets Primary Endpoints Showing Improvement in Copper Mobilisation","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"August 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"AstraZeneca"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Eplontersen Met Co-primary and Secondary Endpoints in Interim Analysis of the NEURO-TTRansform Phase III trial For Hereditary Transthyretin-mediated Amyloid Polyneuropathy (ATTRv-PN)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"UNITED KINGDOM","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"AstraZeneca"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Alexion Pharmaceuticals","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ALXN1840 Shows Rapid and Sustained Improvement in Copper Mobilization From Tissues, Potentially Closing Treatment Gaps for Wilson Disease Community","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"AstraZeneca"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ALXN1840 Shows Rapid and Sustained Improvement in Copper Mobilisation From Tissues, Potentially Closing Treatment Gaps for Wilson Disease Community","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"AstraZeneca"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"RA Capital Management","pharmaFlowCategory":"D","amount":"$188.0 million","upfrontCash":"Undisclosed","newsHeadline":"Orchard Therapeutics Announces Strategic Financing Totalling up to $188 Million","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"March 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Orchard Therapeutics"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Eplontersen Demonstrated Sustained Benefit in Phase III Trial for Hereditary Transthyretin-Mediated Amyloid Polyneuropathy (ATTRv-PN) Through 66 Weeks","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"UNITED KINGDOM","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"AstraZeneca"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Guggenheim Securities, LLC","pharmaFlowCategory":"D","amount":"$188.0 million","upfrontCash":"Undisclosed","newsHeadline":"Orchard Therapeutics Announces Second Closing of Strategic Financing, Resulting in $34 Million of Additional Capital","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"Approved","date":"June 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Orchard Therapeutics"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"San Raffaele-Telethon Institute for Gene Therapy","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orchard Therapeutics Announces First Patient Randomized in Registrational Trial of OTL-203 for MPS-I Hurler Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Orchard Therapeutics"}]
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Details:
OTL-203 is an investigational hematopoietic stem cell gene therapy which uses a modified virus to insert a functional copy of the IDUA gene into a patient’s cells. It is being evaluated in Phase 3 clinical trials for MPS-I Hurler Syndrome.
Lead Product(s):
OTL-203
Therapeutic Area: Genetic Disease
Product Name: OTL-203
Highest Development Status: Phase III
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
San Raffaele-Telethon Institute for Gene Therapy
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
February 05, 2024
Details:
The Company intends to use the net proceeds to accelerate commercialization of Libmeldy (atidarsagene autotemcel), a gene therapy treatment for metachromatic leukodystrophy, and advance the company’s HSC gene therapy R&D portfolio and approval of OTL-200 in the U.S. for MLD.
Lead Product(s):
Atidarsagene Autotemcel
Therapeutic Area: Genetic Disease
Product Name: Libmeldy
Highest Development Status: Phase III
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Guggenheim Securities, LLC
Deal Size: $188.0 million
Upfront Cash: Undisclosed
Deal Type: Private Placement
June 26, 2023
Details:
ION-682884 (eplontersen) is an investigational LIgand-Conjugated Antisense (LICA) medicine designed to inhibit the production of TTR protein. It is being developed as a monthly self-administered subcutaneous injection to treat all types of ATTR.
Lead Product(s):
Eplontersen
Therapeutic Area: Genetic Disease
Product Name: ION-682884
Highest Development Status: Phase III
Product Type: Large molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
March 27, 2023
Details:
The Company intends to use the net proceeds to accelerate commercialization of Libmeldy and advance the company’s HSC gene therapy R&D portfolio and approval of OTL-200 in the U.S. for metachromatic leukodystrophy (MLD), advancing its clinical and pre-clinical stage programs.
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:
RA Capital Management
Deal Size: $188.0 million
Upfront Cash: Undisclosed
Deal Type: Private Placement
March 06, 2023
Details:
ALXN1840 (WTX101) is a potential new once-daily, oral medicine in development for the treatment of Wilson disease. This investigational, novel molecule is designed to selectively and tightly bind to and remove copper from the body’s tissues and blood.
Lead Product(s):
Tiomolibdate Choline
Therapeutic Area: Genetic Disease
Product Name: ALXN1840
Highest Development Status: Phase III
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Alexion Pharmaceuticals
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
June 27, 2022
Details:
Results from positive FoCus trial in Wilson disease showed that WTX101 (ALXN1840), met its primary endpoint demonstrating three-times greater copper mobilisation from tissues compared to SoC including in patients who had been treated previously for an average of 10 years.
Lead Product(s):
Tiomolibdate Choline
Therapeutic Area: Genetic Disease
Product Name: WTX101
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
June 23, 2022
Details:
Eplontersen (IONIS-TTR-LRX) is a LICA investigational medicine designed to reduce the production of transthyretin, or TTR protein, to treat all types of ATTR, a systemic, progressive and fatal disease.
Lead Product(s):
Eplontersen
Therapeutic Area: Genetic Disease
Product Name: IONIS-TTR-LRX
Highest Development Status: Phase III
Product Type: Large molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
June 21, 2022
Details:
ALXN1840, a potential new once-daily, oral medicine, demonstrated approximately three times greater copper mobilisation than SoC. ALXN1840 demonstrated approximately three times greater copper mobilisation from tissues than standard-of-care treatments.
Lead Product(s):
Tiomolibdate Choline
Therapeutic Area: Genetic Disease
Product Name: WTX101
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
August 26, 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.
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 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.
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