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[{"company":"Hitachi Chemicals","sponsor":"Bluebird Bio","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Hitachi Chemical Advanced Therapeutics Solutions and apceth Biopharma ink Clinical and Commercial Manufacturing Pact with Bluebird Bio","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"JAPAN","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Hitachi Chemicals"},{"company":"JCR Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"JCR Pharmaceuticals Completes Phase 1\/2 Clinical Trial Notification of JR-171 for mucopolysaccharidosis type I","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"JAPAN","productType":"Large molecule","productStatus":"Approved","date":"July 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"JCR Pharmaceuticals"},{"company":"JCR Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"JCR Announces First Patient Dosed in Phase1\uff0f2 Global Clinical Trial of JR-171 for MPS I","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"JAPAN","productType":"Large molecule","productStatus":"Approved","date":"October 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"JCR Pharmaceuticals"},{"company":"JCR Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"EMA Grants Orphan Drug Designation to JR-171 for the Treatment of Mucopolysaccaridosis Type I (MPS I)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"JAPAN","productType":"Large molecule","productStatus":"Approved","date":"March 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"JCR Pharmaceuticals"},{"company":"Astellas Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Astellas Provides Update on ASPIRO Clinical Trial of AT132 in Patients with X-linked Myotubular Myopathy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"JAPAN","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Astellas Pharma"},{"company":"Astellas Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Astellas Reports Update to September 1 Announcement on the ASPIRO Clinical Trial of AT132 in Patients with X-linked Myotubular Myopathy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"JAPAN","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Astellas Pharma"},{"company":"Astellas Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Astellas Announces Positive Safety Data from the FORTIS Study of AT845 in Adults with Late-Onset Pompe Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"JAPAN","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Astellas Pharma"},{"company":"Astellas Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Astellas Announces FDA Update on the FORTIS Clinical Trial of AT845 in Adults with Late-Onset Pompe Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"JAPAN","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Astellas Pharma"}]

Astellas Pharma

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Astellas Announces FDA Update on the FORTIS Clinical Trial of AT845 in Adults with Late-Onset Pompe Disease

Details:

AT845, a novel gene replacement therapy using an AAV8 vector under a muscle-specific promotor to deliver a functional copy of the GAA gene, for the treatment of LOPD.

Lead Product(s): AT845

Therapeutic Area: Genetic Disease Product Name: AT845

Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable June 26, 2022

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Astellas Pharma

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Astellas Announces Positive Safety Data from the FORTIS Study of AT845 in Adults with Late-Onset Pompe Disease

Details:

Astellas is developing AT845, a novel gene replacement therapy using an AAV8 vector, under a cardiac- and skeletal muscle-specific promotor, to deliver a functional copy of the GAA gene, for the treatment of Late-Onset Pompe Disease (LOPD).

Lead Product(s): AT845

Therapeutic Area: Genetic Disease Product Name: AT845

Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable February 07, 2022

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Astellas Pharma

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Astellas Reports Update to September 1 Announcement on the ASPIRO Clinical Trial of AT132 in Patients with X-linked Myotubular Myopathy

Details:

ASPIRO is a two-part, multinational, randomized, open-label ascending dose trial to evaluate the safety and preliminary efficacy of AT132 in XLMTM patients less than five years of age.

Lead Product(s): Resamirigene Bilparvovec

Therapeutic Area: Genetic Disease Product Name: AT132

Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable September 14, 2021

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Astellas Pharma

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Astellas Provides Update on ASPIRO Clinical Trial of AT132 in Patients with X-linked Myotubular Myopathy

Details:

This decision follows the reporting of a recent serious adverse event (SAE) in a study participant due to abnormal liver function tests (LFTs) observed in the weeks following dosing of the AT132 investigational gene therapy product at a lower dose (1.3x1014 vg/kg).

Lead Product(s): Resamirigene Bilparvovec

Therapeutic Area: Genetic Disease Product Name: AT132

Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable September 01, 2021

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JCR Pharmaceuticals

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EMA Grants Orphan Drug Designation to JR-171 for the Treatment of Mucopolysaccaridosis Type I (MPS I)

Details:

JR-171 is a blood-brain-barrier (BBB)-penetrating form recombinant α-L-iduronidase that was developed using JCR’s proprietary J-Brain Cargo® BBB technology.

Lead Product(s): alpha L Idosiduronase

Therapeutic Area: Genetic Disease Product Name: JR-171

Highest Development Status: Phase I/ Phase II Product Type: Large molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable March 30, 2021

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JCR Pharmaceuticals

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JCR Announces First Patient Dosed in Phase1／2 Global Clinical Trial of JR-171 for MPS I

Details:

In this study, MPS I patients will be treated with JR-171 for at most 12 weeks to evaluate the safety of JR-171 as the primary endpoint and exploratory efficacy and pharmacokinetics as secondary endpoints.

Lead Product(s): alpha L Idosiduronase

Therapeutic Area: Genetic Disease Product Name: JR-171

Highest Development Status: Phase I/ Phase II Product Type: Large molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable October 30, 2020

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JCR Pharmaceuticals

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JCR Pharmaceuticals Completes Phase 1/2 Clinical Trial Notification of JR-171 for mucopolysaccharidosis type I

Details:

JR-171 is a blood-brain-barrier (BBB)-penetrating recombinant α-L-iduronidase, the product candidate for the treatment of patients with MPS I, to which J-Brain Cargo®, JCR’s proprietary BBB penetration technology, is applied.

Lead Product(s): alpha L Idosiduronase

Therapeutic Area: Genetic Disease Product Name: JR-171

Highest Development Status: Phase I/ Phase II Product Type: Large molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable July 31, 2020

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Hitachi Chemicals

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Hitachi Chemical Advanced Therapeutics Solutions and apceth Biopharma ink Clinical and Commercial Manufacturing Pact with Bluebird Bio

Details:

The two subsidiaries, Hitachi Chemical Advanced Therapeutics Solutions (HCATS) and Apceth Biopharma, will be in charge of late-clinical development and manufacturing of LentiGlobin at facilities in Germany (Apceth) and the U.S. (HCATS).

Lead Product(s): LentiGlobin

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Bluebird Bio

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Agreement May 21, 2020

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