LENZ Therapeutics is focused on developing innovative ophthalmic pharmaceutical products aimed at restoring vision, particularly addressing the widespread issue of presbyopia, which affects nearly 2 billion people globally. Their lead program centers around Aceclidine, a pupil-selective miotic, which is being developed as a daily eye drop treatment known as LNZ100. This product aims to provide an effective solution for restoring near vision, allowing individuals to regain the ability to focus on close objects without the need for reading glasses. The company is currently advancing through various clinical trial phases, with a vision to bring this groundbreaking treatment to market.

The science behind LENZ Therapeutics' approach involves understanding the age-related loss of near vision due to the hardening of the eye's lens and the decreased effectiveness of the ciliary muscle. This results in a condition where individuals struggle to bring near objects into focus, leading to blurry vision. By leveraging the unique properties of Aceclidine, LENZ Therapeutics aims to offer a solution that not only addresses the symptoms of presbyopia but also enhances the quality of life for millions affected by this condition. Their commitment to scientific innovation and patient care drives their mission to free individuals from the limitations imposed by presbyopia.

United States

LENZ Therapeutics is a late-stage clinical company developing innovative ophthalmic pharmaceutical products that improve vision, focused on an eye drop.

Eye Diseases

LENZ Therapeutics

Eye disease

Presbyopia

LNZ100 is a once-daily, preservative-free ophthalmic solution developed by LENZ Therapeutics for the treatment of presbyopia. Its active ingredient, aceclidine, is a miotic agent that functions as an acetylcholine receptor agonist. Aceclidine induces pupil constriction (miosis) by selectively stimulating the iris sphincter muscle, resulting in a "pinhole effect" that enhances near vision by increasing depth of focus. Notably, this mechanism avoids overstimulation of the ciliary muscle, thereby minimizing the risk of a myopic shift and preserving distance vision—a common limitation in other miotic therapies like pilocarpine and carbachol.

Clinical trials have demonstrated that LNZ100 effectively reduces pupil diameter to below 2 mm, which is considered optimal for improving near vision in presbyopic patients. The treatment has shown a rapid onset of action, with significant improvements in near visual acuity observed within 30 minutes, and sustained efficacy lasting up to 10 hours post-administration. Importantly, these benefits were achieved without compromising distance vision.

Given its unique pupil-selective mechanism and favorable safety profile, LNZ100 has the potential to offer a non-invasive, reversible, and convenient therapeutic option for a broad range of presbyopic patients, including those in older age groups who may have diminished accommodative capacity.

LNZ100 

LNZ100 - 1.75% Aceclidine

Small Molecule Pharmaceuticals

Sickle cell disease

Graphite Bio’s first clinical candidate, GPH101, is an &nbsp;investigational next-generation gene-edited autologous HSC product &nbsp;candidate that is currently in development for sickle cell disease &nbsp;(SCD). SCD is a group of rare inherited blood disorders and among the &nbsp;most prevalent monogenic diseases. With GPH101, Graphite Bio aims to &nbsp;directly correct the underlying mutation that causes SCD to decrease the &nbsp;production of sickle hemoglobin and restore the expression of normal &nbsp;adult hemoglobin, thereby potentially curing the disease.
GPH101 &nbsp;is being studied in the CEDAR trial, a Phase 1/2 multi-center, &nbsp;open-label clinical trial designed to evaluate the safety, engraftment &nbsp;success, gene correction rates, total hemoglobin, as well as other &nbsp;clinical and exploratory endpoints and pharmacodynamics in patients with &nbsp;severe SCD.

LENZ Therapeutics

Company Details