AGTC

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AGTC

AGTC is a biotechnology company conducting human clinical trials of AAV-based gene therapies for the It is effective and provides long-lasting benefit.

With a highly specialized team of physicians and researchers, we use cutting-edge techniques to develop treatments for patients that have diseases caused by broken genes. We use gene therapy, which replaces those broken genes with normal functional genes, allowing a patient’s own body to produce proteins to treat their illness. A single injection provides long-lasting treatment, leading to a bette

r quality of life for patients worldwide. The innovative delivery method AGTC uses is the non-toxic adeno-associated virus (AAV), a safe virus that delivers healthy copies of the gene, replacing defective copies. AAV is an ideal delivery method because:

It is safe, having never been shown to cause disease. Its production is fully scalable and does not require animal-derived products. It has been approved for use in human clinical trials by U.S. and European regulatory agencies. AGTC has three ongoing ophthalmology development programs and proof-of-concept data in multiple indications. X-Linked Retinoschisis
An inherited form of retinal degeneration affecting young males, presenting with poor vision by school age. Visual acuity usually worsens during the teenage years and then can lead to serious complications such as vitreous hemorrhage or retinal detachment during adulthood. Achromatopsia
An inherited condition that is associated with visual acuity loss, extreme light sensitivity resulting in daytime blindness, and total loss of color discrimination. X-Linked Retinitis Pigmentosa
An inherited condition that causes boys to develop night blindness by the time they are ten and progresses to legal blindness by their early forties. Alpha-1 Antitrypsin Deficiency
One of the most common hereditary disorders in the world and the most commonly known genetic risk factor for emphysema, Alpha-1 can also cause liver disease.

15/07/2024

We are thrilled to announce the successful closing of our $170 million Series B, led by Forbion with contributions from existing investors Syncona Limited Oxford Science Enterprises and University of Oxford, as well as initial investments from TCGX and Advent Life Sciences. This represents a significant milestone in our journey to bring potentially transformative treatments to patients with blinding retinal diseases. The funds will be used to support the continued clinical development of AGTC-501 for X-Linked Retinitis Pigmentosa (XLRP) and our Dry Age-related Macular Degeneration (dAMD) program.

To read more about our Series B funding and pipeline momentum, click the link to the press release below.

https://www.beacontx.com/news-and-events/beacon-therapeutics-raises-170-million-in-series-b-funding-to-advance-development-of-ophthalmic-gene-therapies/

12/06/2024

Beacon Therapeutics is pleased to announce that the first patient has been treated in our Phase 2/3 VISTA clinical trial for our lead asset, AGTC-501. This is an important clinical milestone for AGTC-501 and another promising step toward a potential treatment for patients with X-Linked Retinitis Pigmentosa (XLRP), a blinding orphan disease for which there is no available treatment. VISTA is a randomized, controlled, masked, multi-center pivotal study evaluating the efficacy, safety, and tolerability of 2 doses of AGTC-501 for the treatment of XLRP compared to an untreated control group.

To learn more about our ongoing VISTA trial, click the link to the announcement press release below.

Related posts: Subretinal Gene Therapy Drug AGTC-501 for X-Linked Retinitis Pigmentosa Phase 2 Randomized, Controlled, Multicenter Clinical Trial (Skyline) 3-Month Results Beacon Therapeutics Announces Positive 12-Month Data from Phase 2 SKYLINE Trial of AGTC-501 in Patients with X-Linked Retinitis....

25/04/2024

We are excited to announce our Phase 2/3 Vista Clinical Trial is now open to enrollment. To learn more about our clinical trials and our science, visit beacontx.com.

08/02/2024

Beacon Therapeutics is excited to announce promising 12-month interim safety and efficacy results of the Phase 2 SKYLINE trial in patients with X-linked retinitis pigmentosa (XLRP). The 12-month data, presented by Mark Pennesi, M.D., Ph.D., FARVO, Director, Ophthalmic Genetics at the Retina Foundation at the 47th Annual Macula Society Meeting, represents another step toward a potential treatment for this devastating condition.

Follow Beacon Therapeutics for future updates! To read more about this data, please visit our website: https://www.beacontx.com/news-and-events/

01/11/2023

Follow Therapeutics for future posts!! Visit Beacon Therapeutics’ new exhibit booth at The American Academy of Ophthalmology’s annual meeting or check out Nadia Waheed’s presentation at Eyecelerator on Nov. 2nd

25/10/2023

Beacon Therapeutics is proud to continue sponsoring the Foundation Fighting Blindness’s Boston Vision Walk. Follow Beacon Therapeutics for future updates

12/06/2023

Following the acquisition of AGTC by Syncona Limited, Beacon Therapeutics has been launched with a £96 million funding round.

Beacon Therapeutics aims to pursue the development of a new generation of gene therapies to treat a range of rare and prevalent retinal diseases.

They will be taking forward AGTC-501, a gene therapy program currently in Phase II clinical trials for the treatment of X-linked retinitis pigmentosa ( ) as their lead asset.

AGTC executives David Fellows, Dr Nadia Waheed and Dr. Abraham Scaria will be joining the Beacon management team.

To keep up to date with our progress, give Beacon Therapeutics a follow!

Today marks the official launch of Beacon Therapeutics!

Here at Beacon Therapeutics, we are pursuing the development of a new generation of gene therapies to treat a range of rare and prevalent retinal diseases that result in blindness.

Syncona Limited, with additional investors including Oxford Science Enterprises, have provided £96 million ($120 million) to fund the acquisition of AGTC and provide capital to take each of our development candidates through to value inflection points.

For more information, read our full press release here: https://www.beacontx.com/insights/beacon-therapeutics-launch/.

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