Allergan and Editas Medicine Announce Initial Data from Natural History Study to Evaluate Patients with Leber Congenital Amaurosis 10 (LCA10)
The Companies initiated the natural history study to prospectively evaluate and confirm the course of disease in patients and determine the reproducibility and stability of ophthalmic assessments over time. This knowledge has informed the planned Phase 1/2 interventional clinical trial design for AGN-151587 (EDIT-101), an experimental CRISPR genome editing medicine being investigated for the treatment of LCA10. Massachusetts Eye and Ear, an international center for treatment and research and a teaching hospital of
“Better understanding the course of disease for patients with CEP290-associated retinal degeneration is a key step towards the interventional clinical trial for a CRISPR-based experimental medicine to treat this disease,” said
Twenty-one patients with LCA10-IVS26 mutations in CEP290 were included in this analysis of baseline characteristics and key clinical assessments, including visual acuity and full-field threshold sensitivity changes. Initial data from the natural history study suggest multiple clinical assessments should prove informative in the planned Phase 1/2 interventional trial.
AGN-151587 is set to be the first in vivo, or inside the body, CRISPR-based medicine administered to people anywhere in the world. In the planned Phase 1/2 clinical trial,
About AGN-151587 (EDIT-101)
AGN-151587 (EDIT-101) is a CRISPR-based experimental medicine under investigation for the treatment of Leber congenital amaurosis 10 (LCA10). AGN-151587 is administered via a subretinal injection to reach and deliver the gene editing machinery directly to photoreceptor cells.
About Leber Congenital Amaurosis
Leber congenital amaurosis, or LCA, is a group of inherited retinal degenerative disorders caused by mutations in at least 18 different genes. It is the most common cause of inherited childhood blindness, with an incidence of two to three per 100,000 live births worldwide. Symptoms of LCA appear within the first years of life, resulting in significant vision loss and potentially blindness. The most common form of the disease, LCA10, is a monogenic disorder caused by mutations in the CEP290 gene and is the cause of disease in approximately 20‑30 percent of all LCA patients.
Allergan’s success is powered by our global colleagues’ commitment to being Bold for Life. Together, we build bridges, power ideas, act fast and drive results for our customers and patients around the world by always doing what is right.
With commercial operations in approximately 100 countries,
For more information, visit Allergan’s website at www.Allergan.com.
As a leading genome editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cpf1 (also known as Cas12a) genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. For the latest information and scientific presentations, please visit www.editasmedicine.com.
Allergan Forward-Looking Statements
Statements contained in this press release that refer to future events or other non-historical facts are forward-looking statements that reflect Allergan’s current perspective on existing trends and information as of the date of this release. Actual results may differ materially from Allergan’s current expectations depending upon a number of factors affecting Allergan’s business. These factors include, among others, the difficulty of predicting the timing or outcome of
Editas Medicine Forward-Looking Statements
This press release contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995. The words ‘‘anticipate,’’ ‘‘believe,’’ ‘‘continue,’’ ‘‘could,’’ ‘‘estimate,’’ ‘‘expect,’’ ‘‘intend,’’ ‘‘may,’’ ‘‘plan,’’ ‘‘potential,’’ ‘‘predict,’’ ‘‘project,’’ ‘‘target,’’ ‘‘should,’’ ‘‘would,’’ and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include statements regarding the Companies’ plans with respect to the planned Phase 1/2 clinical trial for AGN-151587 (EDIT-101), including initiating patient screening by mid-year and dosing patients in the second half of 2019. Editas Medicine may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of Editas Medicine’s product candidates; availability and timing of results from preclinical studies and clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products and availability of funding sufficient for Editas Medicine’s foreseeable and unforeseeable operating expenses and capital expenditure requirements. These and other risks are described in greater detail under the caption “Risk Factors” included in Editas Medicine’s most recent Annual Report on Form 10-K, which is on file with the
Manisha Narasimhan, Ph.D.
(862) 261 8820
Source: Editas Medicine, Inc.