Genetic Disease | Preclinical Developments

Ocugen

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Ocugen Granted FDA Orphan Drug Designation for OCU400 Gene Therapy for the Treatment of RHO Mutation-Associated Retinal Degenerative Disease

Lead Product(s): AAV-hNR2E3

Therapeutic Area: Genetic Disease Product Name: OCU400

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 July 27, 2020

Details:

Third orphan drug designation for the same product, OCU400, is unique in Ophthalmology gene therapy and demonstrates its potential to treat many Inherited Retinal Degenerative diseases (IRDs).

Vita Therapeutics

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Vita Therapeutics Receives Orphan Drug Designation from FDA for New Novel Treatment

Lead Product(s): VTA-110

Therapeutic Area: Genetic Disease Product Name: VTA-110

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 July 23, 2020

Details:

VTA-110, is a potential first-in-class allogenic iPSC-based therapy that has shown the ability to repair and regenerate healthy muscle in preclinical studies and has the potential to benefit patients with DMD.

Triplet Therapeutics

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Triplet Therapeutics Selects Clinical Candidate for Novel Treatment of Repeat Expansion Disorders

Lead Product(s): TTX-3360

Therapeutic Area: Genetic Disease Product Name: TTX-3360

Highest Development Status: Preclinical Product Type: Large molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable July 21, 2020

Details:

Recent experiments by Triplet demonstrated that a single dose of TTX-3360 induced significant knockdown of the target gene mRNA at 29 days in the cortex (>75%) and caudate (>45%) in non-human primates.

Renovion

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Renovion Announces Publication of Research Demonstrating the Impact of the Novel Treatment, Arina-1 on Mucus Transport in Cystic Fibrosis

Lead Product(s): Ascorbic Acid,Glutathione,Bicarbonate

Therapeutic Area: Genetic Disease Product Name: ARINA-1

Highest Development Status: Preclinical Product Type: Small molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable July 09, 2020

Details:

The paper provides an overview of the impact of ARINA-1 on mucus transport and shows that the ARINA-1 moves mucus significantly better than currently available options in cystic fibrosis (CF).

Erasmus University Medical Center

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Stem Cell Gene Therapy Restores Glycogen Levels, Aids Movement in Mouse Study

Lead Product(s): Hematopoietic Stem Cell Gene Therapy

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 July 08, 2020

Details:

Findings suggest that blood stem cell gene therapy, in which a patient’s own stem cells are modified to produce the acid alpha-glucosidase (GAA) enzyme, may be a promising approach for treating people with classic infantile Pompe disease.

Catalyst Biosciences

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Early Data Support Catalyst Biosciences’ Potential Hemophilia B Gene Therapy

Lead Product(s): FIX-Gene Therapy

Therapeutic Area: Genetic Disease Product Name: CB 2679d-GT

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 July 08, 2020

Details:

The new data include a direct comparison between CB 2679d-GT and the Padua variant in a mouse model of hemophilia B.

Hansa Biopharma

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SWEDEN Sweden

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Hansa Biopharma Inks Agreement With Sarepta Therapeutics to Develop and Promote Imlifidase as Pre-treatment Ahead of Gene Therapy

Lead Product(s): Imlifidase

Therapeutic Area: Genetic Disease Product Name: Undisclosed

Highest Development Status: Preclinical Product Type: Large molecule

Partner/Sponsor/Collaborator: Sarepta Therapeutics

Deal Size: $407.5 million Upfront Cash: $10.0 million

Deal Type: Licensing Agreement July 02, 2020

Details:

Hansa Biopharma Granted Sarepta an exclusive, worldwide license to develop and promote imlifidase as a pre-treatment to enable Sarepta gene therapy treatment in Duchenne muscular dystrophy (DMD) and Limb-girdle muscular dystrophy (LGMD).

SparingVision

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FRANCE France

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

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

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.

Moderna Therapeutics

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Moderna Highlights Advances in Platform Science and Innovative Vaccine Research at Third Annual Science Day

Lead Product(s): mRNA-3745

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 02, 2020

Details:

Preclinical data show that 3’-idT-stabilized mRNA encoding phenylalanine hydroxylase (PAH), the enzyme missing or dysfunctional in the metabolic disorder phenylketonuria (PKU), supports sustained reduction of serum phenylalanine levels in a PKU mouse model.

Intellia Therapeutics

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Regeneron and Intellia Therapeutics Expand Collaboration to Develop Crispr/Cas9-Based Treatments

Lead Product(s): CRISPR/Cas9 based gene therapy

Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

Partner/Sponsor/Collaborator: REGENERON

Deal Size: $100.0 million Upfront Cash: $70.0 million

Deal Type: Agreement June 01, 2020

Details:

Under the amended agreement, Regeneron will have rights to discover and develop CRISPR/Cas9-based therapeutic products for an additional five in vivo liver targets, for a total of up to 15 targets.

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Find Drugs for Genetic Disease in Preclinical Development

Preclinical

Product Type

Companies

Ocugen

Ocugen Granted FDA Orphan Drug Designation for OCU400 Gene Therapy for the Treatment of RHO Mutation-Associated Retinal Degenerative Disease

Vita Therapeutics

Vita Therapeutics Receives Orphan Drug Designation from FDA for New Novel Treatment

Triplet Therapeutics

Triplet Therapeutics Selects Clinical Candidate for Novel Treatment of Repeat Expansion Disorders

Renovion

Renovion Announces Publication of Research Demonstrating the Impact of the Novel Treatment, Arina-1 on Mucus Transport in Cystic Fibrosis

Erasmus University Medical Center

Stem Cell Gene Therapy Restores Glycogen Levels, Aids Movement in Mouse Study

Catalyst Biosciences

Early Data Support Catalyst Biosciences’ Potential Hemophilia B Gene Therapy

Hansa Biopharma

Hansa Biopharma Inks Agreement With Sarepta Therapeutics to Develop and Promote Imlifidase as Pre-treatment Ahead of Gene Therapy

SparingVision

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

Moderna Therapeutics

Moderna Highlights Advances in Platform Science and Innovative Vaccine Research at Third Annual Science Day

Intellia Therapeutics

Regeneron and Intellia Therapeutics Expand Collaboration to Develop Crispr/Cas9-Based Treatments