Ophthalmology | Phase I/ Phase II Developments

Editas Medicine

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United States

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First patient treated with CRISPR medicine in phase 1/2 trial

Lead Product(s): AGN-151587

Therapeutic Area: Ophthalmology

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 04, 2020

Details:

The BRILLIANCE clinical trial is evaluating AGN-151587 (EDIT-101) in patients with Leber congenital amaurosis 10 (LCA10). It is the first gene editing medicine to be evaluated inside the body.

ReNeuron

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United Kingdom

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ReNeuron Announces Positive Long-term Data in Retinal Clinical Trial

Lead Product(s): hRPC stem cell

Therapeutic Area: Ophthalmology

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 February 24, 2020

Details:

Company will present clinical and preclinical results in a number of diseases where mast cells and/or eosinophils have been shown to play a role in disease pathology.

Agtc

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AGTC Completes Enrollment in the Two Highest Dose Groups of its Ongoing Phase 1/2 Clinical Trial for X-Linked Retinitis Pigmentosa

Lead Product(s): AGTC-501

Therapeutic Area: Ophthalmology

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 February 19, 2020

Details:

The Phase 1/2 clinical trial is evaluating the safety and efficacy of sub-retinal injection of AGTC-501 for the treatment of X-linked retinitis pigmentosa (XLRP) caused by mutations in the RPGR gene.

Lineage Cell Therapeutics

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United States

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Lineage Provides Positive Update on Clinical Study of OpRegen®

Lead Product(s): Retinal pigment epithelial cell

Therapeutic Area: Ophthalmology

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 February 06, 2020

Details:

All five patients in Cohort 4, those with better baseline vision and which represent the Company’s intended patient population, have shown increases in best corrected visual acuity (BCVA).

ProQR Therapeutics

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Netherlands

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ProQR Receives Rare Pediatric Disease Designation from FDA for QR-421a

Lead Product(s): QR-421a

Therapeutic Area: Ophthalmology

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 January 30, 2020

Details:

QR-421a is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of vision loss in Usher syndrome type 2 and non-syndromic retinitis pigmentosa.

Eyevensys

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GERMANY Germany

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Eyevensys Closes $30M Series B Financing

Lead Product(s): EYS606

Therapeutic Area: Ophthalmology

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

Partner/Sponsor/Collaborator: Boehringer Ingelheim Venture Fund

Deal Size: $30.0 million Upfront Cash: Undisclosed

Deal Type: Series B Financing January 08, 2020

Details:

The company will use the funds to continue the development of its clinical lead candidate EYS606 for the treatment of chronic non-infectious uveitis (NIU), including the launch of its Electro Study.

Allysta Pharmaceuticals

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Allysta Pharmaceuticals, Inc. Doses First Patient with ALY688 Ophthalmic Solution in Phase 1/2a Dry Eye Study

Lead Product(s): ALY688

Therapeutic Area: Ophthalmology

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 January 08, 2020

Details:

Allysta Pharmaceuticals, Inc. announced dosing of the first patient in its Phase 1/2a trial (ALY688-201) of ALY688 Ophthalmic Solution for the treatment of dry eye disease.

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Editas Medicine

First patient treated with CRISPR medicine in phase 1/2 trial

ReNeuron

ReNeuron Announces Positive Long-term Data in Retinal Clinical Trial

Agtc

AGTC Completes Enrollment in the Two Highest Dose Groups of its Ongoing Phase 1/2 Clinical Trial for X-Linked Retinitis Pigmentosa

Lineage Cell Therapeutics

Lineage Provides Positive Update on Clinical Study of OpRegen®

ProQR Therapeutics

ProQR Receives Rare Pediatric Disease Designation from FDA for QR-421a

Eyevensys

Eyevensys Closes $30M Series B Financing

Allysta Pharmaceuticals

Allysta Pharmaceuticals, Inc. Doses First Patient with ALY688 Ophthalmic Solution in Phase 1/2a Dry Eye Study