Genetic Disease | Phase II/ Phase III Developments

Sanofi

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FRANCE France

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Positive topline results demonstrated by olipudase alfa, first and only investigational therapy in late-stage development for acid sphingomy...

Lead Product(s): Olipudase alfa

Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable January 30, 2020

Details:

Olipudase alfa is the first and only investigational enzyme replacement therapy in late-stage development for the treatment of ASMD.

PTC Therapeutics

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United States

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Royalty Pharma Acquires Royalty Interest in Risdiplam From PTC Therapeutics

Lead Product(s): Risdiplam

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: Phase II/ Phase III Product Type: Small molecule

Partner/Sponsor/Collaborator: Royalty Pharma

Deal Size: $650.0 million Upfront Cash: $650.0 million

Deal Type: Agreement July 20, 2020

Details:

Risdiplam, to be marketed by Roche, is an investigational, orally administered survival motor neuron-2 (SMN2) splicing modifier for the treatment of spinal muscular atrophy (SMA).

Orphazyme

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DENMARK Denmark

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Orphazyme Completes Rolling Submission of New Drug Application to U.S. FDA for Arimoclomol for Niemann-Pick Disease Type C

Lead Product(s): Arimoclomol

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: Phase II/ Phase III Product Type: Small molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable July 20, 2020

Details:

Orphazyme has completed the rolling submission of its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for its investigational therapy arimoclomol for the treatment of Niemann-Pick disease Type C (NPC).

Freeline

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United Kingdom

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Phase 1/2 Gene Therapy Biotech Freeline Therapeutics Files for a $100 million US IPO

Lead Product(s): FLT180a

Therapeutic Area: Genetic Disease Product Name: FLT180a

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

Partner/Sponsor/Collaborator: Undisclosed

Deal Size: $100.0 million Upfront Cash: Undisclosed

Deal Type: Public Offering July 17, 2020

Details:

The company's lead candidate, FLT180a, an investigational gene therapy medicinal product candidate for the treatment of hemophilia B, is currently in a Phase 1/2 trial and has dosed 10 patients as of June 15, 2020.

Freeline

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United Kingdom

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Freeline Closes Major $120M Funding Round

Lead Product(s): FLT180a

Therapeutic Area: Genetic Disease Product Name: FLT180a

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

Partner/Sponsor/Collaborator: Novo Holdings

Deal Size: $120.0 million Upfront Cash: Undisclosed

Deal Type: Series C Financing June 30, 2020

Details:

Freeline expects to use the proceeds from the financing to bring its lead program in Haemophilia B into a pivotal trial, as well as to continue its Phase 1/2 clinical program for Fabry Disease and further progress its pipeline programs for Gaucher Disease and Haemophilia A.

Zynerba Pharmaceuticals

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United States

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Zynerba Pharmaceuticals Announces Top Line Results from Pivotal CONNECT-FX Trial of Zygel™ (CBD Gel) in Fragile X Syndrome

Lead Product(s): Cannabidiol

Therapeutic Area: Genetic Disease Product Name: Zygel

Highest Development Status: Phase II/ Phase III Product Type: Small molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable June 30, 2020

Details:

Zygel did not achieve statistical significance versus placebo in the primary endpoint of improvement in the Social Avoidance subscale of the Aberrant Behavior Checklist – Community FXS (ABC-CFXS).

Freeline

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United Kingdom

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Freeline Announces Acceptance of Late Breaking Abstract at the International Society on Thrombosis and Haemostasis (ISTH) 2020 Meeting

Lead Product(s): FLT180a

Therapeutic Area: Genetic Disease Product Name: FLT180a

Highest Development Status: Phase II/ 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 June 29, 2020

Details:

Updated data will be presented in an oral presentation on the efficacy and safety of AAV gene therapy FL180a in the B-AMAZE study in severe haemophilia B patients.

The Lundquist Institute

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Unique Clinical Trial Investigates a New Treatment Approach for Fatal ‘Childhood Alzheimer’s’ and Continues Despite COVID-19 Pandemic

Lead Product(s): Anakinra

Therapeutic Area: Genetic Disease Product Name: Kineret

Highest Development Status: Phase II/ Phase III Product Type: Peptide

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable June 24, 2020

Details:

The trial, which began in May, involves 20 children with Sanfilippo syndrome, who will be given anakinra, which blocks the activity of interleukin-1 (IL-1), a protein in the body that causes joint damage, thereby reducing inflammation.

ProQR Therapeutics

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Netherlands

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ProQR Announces Virtual Presentation of Phase 1/2 Sepofarsen Data Through the Association for Research in Vision and Ophthalmology (ARVO)

Lead Product(s): Sepofarsen

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: Phase II/ 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 June 08, 2020

Details:

The presentation will highlight results of a phase 1b/2 trial of intravitreal (IVT) sepofarsen (QR-110) antisense oligonucleotide in Leber congenital amaurosis 10 (LCA10) due to p.Cys998X mutation in the CEP290 gene.

Lysogene

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FRANCE France

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Lysogene Announces Important Updates on AAVance Phase 2/3 Clinical Trial with LYS-SAF302 in MPS IIIA

Lead Product(s): LYS-SAF302

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: Phase II/ 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 June 05, 2020

Details:

Following discussions with the U.S. FDA, a clinical hold was issued for the clinical trial AAVance, a global Phase 2/3 clinical trial of LYS-SAF302, a gene therapy for the treatment of Mucopolysaccharidosis Type IIIA (MPS IIIA, also known as Sanfilippo syndrome type A).

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Find Drugs for Genetic Disease in Phase II/ Phase III Clinical Development

Phase II/ Phase III

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Companies

Sanofi

Positive topline results demonstrated by olipudase alfa, first and only investigational therapy in late-stage development for acid sphingomy...

PTC Therapeutics

Royalty Pharma Acquires Royalty Interest in Risdiplam From PTC Therapeutics

Orphazyme

Orphazyme Completes Rolling Submission of New Drug Application to U.S. FDA for Arimoclomol for Niemann-Pick Disease Type C

Freeline

Phase 1/2 Gene Therapy Biotech Freeline Therapeutics Files for a $100 million US IPO

Freeline

Freeline Closes Major $120M Funding Round

Zynerba Pharmaceuticals

Zynerba Pharmaceuticals Announces Top Line Results from Pivotal CONNECT-FX Trial of Zygel™ (CBD Gel) in Fragile X Syndrome

Freeline

Freeline Announces Acceptance of Late Breaking Abstract at the International Society on Thrombosis and Haemostasis (ISTH) 2020 Meeting

The Lundquist Institute

Unique Clinical Trial Investigates a New Treatment Approach for Fatal ‘Childhood Alzheimer’s’ and Continues Despite COVID-19 Pandemic

ProQR Therapeutics

ProQR Announces Virtual Presentation of Phase 1/2 Sepofarsen Data Through the Association for Research in Vision and Ophthalmology (ARVO)

Lysogene

Lysogene Announces Important Updates on AAVance Phase 2/3 Clinical Trial with LYS-SAF302 in MPS IIIA