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[{"company":"BrainVectis","sponsor":"AskBio","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"AskBio Acquires BrainVectis to Expand its Clinical Pipeline for Neurodegenerative Diseases","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"BrainVectis"},{"company":"Vivet Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vivet\u2019s Second Gene Therapy Product, VTX-803 for PFIC3, Receives US and European Orphan Drug Designation","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Vivet Therapeutics"},{"company":"SparingVision","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"SparingVision Receives European Orphan Designation for its Drug Candidate SPVN06 Dedicated to Inherited Retinal Dystrophies","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"SparingVision"},{"company":"Novasep","sponsor":"Sensorion","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Sensorion and Novasep Announce Signature of a Gene Therapy Product Development and Manufacturing Agreement","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Novasep"},{"company":"VECT-HORUS","sponsor":"Undisclosed","pharmaFlowCategory":"D","amount":"$14.5 million","upfrontCash":"Undisclosed","newsHeadline":"VECT-HORUS Completes Its \u20ac12 Million Fund-Raising Operation","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"FRANCE","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"VECT-HORUS"},{"company":"Vivet Therapeutics","sponsor":"Mirum Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Mirum Pharmaceuticals Ink License Agreement for Vivet\u2019s Gene Therapy Programs Targeting Progressive Familial Intrahepatic Cholestasis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Vivet Therapeutics"},{"company":"SATT Conectus","sponsor":"Lysogene","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Lysogene Enters into an Exclusive Worldwide License Agreement with SATT Conectus","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"SATT Conectus"},{"company":"Variant","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"VARIANT Receives European Orphan Drug Designation for its Gene Therapy Product Candidate, VAR002, for the Treatment of Both Leber Congenital Amaurosis and Cone-Rod Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Variant"},{"company":"Atamyo Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Atamyo Therapeutics Obtains First Regulatory Authorization in Europe to Initiate a Clinical Trial for ATA-100, its Gene Therapy to Treat Limb-Girdle Muscular Dystrophy Type 2I\/R9","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Atamyo Therapeutics"},{"company":"Lysogene","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Lysogene Publishes Positive Preliminary Preclinical Results in the Treatment of Fragile X Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Lysogene"}]

Lysogene

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Lysogene Publishes Positive Preliminary Preclinical Results in the Treatment of Fragile X Syndrome

Details:

The publication of a peer-reviewed article for FXS01 in EMBO Molecular Medicine showing that adeno-associated viral (AAV) vector-delivered diacylglycerol kinase (DGKk) achieves long-term rescue of fragile X syndrome (FXS) in a mouse model of disease.

Lead Product(s): FXS01

Therapeutic Area: Genetic Disease Product Name: FXS01

Highest Development Status: Preclinical Product Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable April 21, 2022

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Atamyo Therapeutics

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Atamyo Therapeutics Obtains First Regulatory Authorization in Europe to Initiate a Clinical Trial for ATA-100, its Gene Therapy to Treat Limb-Girdle Muscular Dystrophy Type...

Details:

First approval of ATA-100, was received from UK MHRA its gene therapy for the treatment of the fukutin-related protein limb-girdle muscular dystrophy Type 2I/R9.

Lead Product(s): ATA-100

Therapeutic Area: Genetic Disease Product Name: ATA-100

Highest Development Status: Preclinical Product Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable December 06, 2021

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Variant

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VARIANT Receives European Orphan Drug Designation for its Gene Therapy Product Candidate, VAR002, for the Treatment of Both Leber Congenital Amaurosis and Cone-Rod Dystroph...

Details:

VAR002 is a CRX-expressing AAV vector designed as a relevant therapeutic approach for gene supplementation and a promising drug candidate for mutation-agnostic gene therapy. VAR002 prevents photoreceptor degeneration without adverse effects linked with CRX overexpression.

Lead Product(s): VAR002

Therapeutic Area: Genetic Disease Product Name: VAR002

Highest Development Status: Preclinical Product Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable November 24, 2021

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SATT Conectus

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Lysogene Enters into an Exclusive Worldwide License Agreement with SATT Conectus

Details:

Lysogene will be responsible for the preclinical and clinical development, manufacturing, regulatory activities, and commercialization of the drug candidate, globally. which includes FXS01, a gene therapy candidate for the treatment of Fragile X syndrome.

Lead Product(s): FXS01

Therapeutic Area: Genetic Disease Product Name: FXS01

Highest Development Status: Preclinical Product Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Lysogene

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Licensing Agreement June 11, 2021

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Vivet Therapeutics

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Mirum Pharmaceuticals Ink License Agreement for Vivet’s Gene Therapy Programs Targeting Progressive Familial Intrahepatic Cholestasis

Details:

Vivet will continue to advance the preclinical studies for VTX-803 and VTX-802 for PFIC3 and PFIC2. Mirum has the exclusive option to license the programs after which Mirum would lead the clinical development and any future commercialization of the programs.

Lead Product(s): VTX-803

Therapeutic Area: Genetic Disease Product Name: VTX-803

Highest Development Status: Preclinical Product Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Mirum Pharmaceuticals

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Collaboration April 12, 2021

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VECT-HORUS

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VECT-HORUS Completes Its €12 Million Fund-Raising Operation

Details:

The funding round supports company's program focusing on identifying new vectors. Using the company's VECTrans® platform, nanoparticles of mRNA, as well as ASOs and siRNAs, can be vectorized in order to treat both diseases with a genetic component and cancers.

Lead Product(s): Nucleic acid based therapeutics

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: Preclinical Product Type: Large molecule

Partner/Sponsor/Collaborator: Undisclosed

Deal Size: $14.5 million Upfront Cash: Undisclosed

Deal Type: Series D Financing January 20, 2021

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Novasep

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Sensorion and Novasep Announce Signature of a Gene Therapy Product Development and Manufacturing Agreement

Details:

Novasep will be in charge of developing and manufacturing the two AAV vectors designed for the Sensorion OTOF-GT project and will supply Drug Product batches to support preclinical and clinical studies.

Lead Product(s): OTOF-GT,Aav Vectors

Therapeutic Area: Genetic Disease Product Name: OTOF-GT

Highest Development Status: Preclinical Product Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Sensorion

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Agreement October 27, 2020

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SparingVision

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SparingVision Receives European Orphan Designation for its Drug Candidate SPVN06 Dedicated to Inherited Retinal Dystrophies

Details:

SPVN06 uses a gene therapy-based approach independent of mutated genes and by a single subretinal injection of proprietary neurotrophic factors, aims at stopping and preventing the degeneration of photoreceptors leading to blindness.

Lead Product(s): SPVN06

Therapeutic Area: Genetic Disease Product Name: SPVN06

Highest Development Status: Preclinical Product Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable June 30, 2020

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Vivet Therapeutics

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Vivet’s Second Gene Therapy Product, VTX-803 for PFIC3, Receives US and European Orphan Drug Designation

Details:

VTX-803 is Vivet’s second gene therapy product to be granted Orphan Drug Designation. VTX-803 is currently under IND-enabling studies and initiation of clinical development is planned for the second part of 2021 in both the US and the EU.

Lead Product(s): VTX-803

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: Preclinical Product Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable June 01, 2020

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BrainVectis

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AskBio Acquires BrainVectis to Expand its Clinical Pipeline for Neurodegenerative Diseases

Details:

The acquisition adds BrainVectis's Huntington’s disease drug candidate BV-CYP01 to AskBio’s program.

Lead Product(s): BV-CYP01

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: Preclinical Product Type: Cell and Gene therapy

Partner/Sponsor/Collaborator: AskBio

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Acquisition April 22, 2020

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