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Topical Gene Therapy Effective in Treating Debilitating Eye Condition – InventUM – University of Miami

By: Josh Baxt | February 07, 2024 | 4 min. read| Share Article Summary

Researchers at the University of Miami Miller School of Medicines Bascom Palmer Eye Institute have shown that a new gene therapy, B-VEC (VYJUVEK), is effectively treating severe eye issues in a pediatric patient with a rare disease, dystrophic epidermolysis bullosa (DEB), that also affects skin and other organs.

This is the first time topical gene therapy has been used in the eye. The case study was published on February 7 in the New England Journal of Medicine.

I had been treating this patient for several years, and there was little we could do for him, said Alfonso L. Sabater, M.D., Ph.D., associate professor of clinical ophthalmology at the Miller School and director of Bascom Palmers Corneal Innovation Lab. We tried surgery, platelet-rich plasma and other approaches, but nothing worked, until now.

Caused by a mutation in the COL7A1 gene,DEBimpairs typeVII collagenproductionand can leadto severe blisteringand scar formationon skin, inside the mouth and stomach and on the eyes.

The patient, now 14,had beencoming to Bascom Palmerfor DEB treatmentsince he was 4.With blisters on his skin and eyes,and other issues, hehadlostsignificant visionand had an associated condition,symblepharon, in which the eyelid and eyestucktogether.

He couldnt see much at all, said Dr. Sabater, who has been treating the patient since he was 8. Every timeweconductedsurgery, the scar tissuewouldcome back,and it wasjustgetting worse.I told him,Lets wait until we can find something that can fix theunderlyinggenetic problem.

B-VECwas designed to do just that.The topical gelcontainsre-engineeredherpesvirusesarmed withfunctionalCOL7A1 genes.When B-VEC is applied,thevirusesdelivertheirgenetic payload, generating more normal type VII collagen production.

As soon as he learned about B-VEC,Dr. Sabaterrecognized its potential to help his patient and possibly others. However, before that could happen, they would have to conduct preclinical research toprovethe therapy would beeffectivein eyes.

The team worked closelywithB-VEC developer Krystal Biotech toreformulate the therapy as an eyedrop andconduct the studies. Because the therapywas already in clinical trials forskin blistering, it was relatively easy togainexpanded accessto treat DEB patients with eye issues.

Once they received the go-ahead from the Food & Drug Administration, the team began administering B-VEC to the patient,withexcellent results.

Thedifference this timewasthat,when we started applying the gene therapy, thescartissue was not coming backafter surgery, said Dr. Sabater.Its been ayear and a half,and it hasnt grown back.Inthe past,itwouldhave comeback in three months.Now that he can see, he no longer needshismom tohelphimwith his homeworkandhe can watch TVandplay video games.He hasnormal vision.

Tags: Bascom Palmer Eye Institute, Dr. Alfonso Sabater, dystrophic epidermolysis bullosa, gene therapy

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Medicines access: Unlocking the immense potential in cell and gene therapy – Clinical Trials Arena

There are an estimated 300 million people worldwide who suffer from rare illnesses, with limited choices for treatment. But cell and gene therapies have the potential to transform medical treatments for those people, even providing possible cures to debilitating conditions and life-threatening illnesses where there are currently limited options.

Research is increasingly showing that such therapies can modify the bodys cells to effectively enable it to repair itself, and new, personalised treatments can even help meet the exact needs of individuals. Treatments once thought of as miraculous are now edging closer to becoming a reality for greater numbers of patients.

Last year set an annual record for cancer cell and gene therapies. The US FDAs approval of two chimeric antigen receptor T-cell (CAR-T) therapies for blood malignancies as first-line treatments brings the total number of CAR-Ts available to patients in the US and Europe to six.

In 2019, FDA officials estimated that the agency would receive more than 200 investigational new drug applications per year for cell and gene treatments. By 2025, the FDA is expected to approve ten to 20 cell and gene therapies a year.

According to the GlobalData Pharma Intelligence Centre, the cell and gene therapy market is expected to grow at a compound annual growth rate (CAGR) of 25%-30% over the next few years, signalling an area of huge potential for the pharmaceutical and biotech industries. And for the pharma and biotech companies participating in medicines access programs for cell and gene therapy, a world of vital real-world data becomes available, offering valuable insight on how effective treatments are for patients, as well as providing the opportunity to generate revenue before regulatory approval has been granted or during the trial itself.

While real-world data on its own is not enough to secure marketing authorisation, demonstrating how a medicine works in a real-world setting, especially in orphan therapies, can strengthen any evidence submitted, potentially helping to gain approval.

Despite the benefits and market outlook, most businesses are reluctant or unable to invest in a single gene therapy at a time. This is frequently due to a number of hurdles in the development process. Consequently, access remains severely limited for patients.

Take the regulatory approval process, for example. For cell and gene therapies this can be complicated and variable among health agencies globally, which can affect the availability and access to innovative medicines across countries. Pre-approval for cell and gene therapy has only come to the fore in the last few years and authorisation often only exists in a handful of countries. For instance, smaller regions in Eastern Europe have lower availability of centrally approved products compared with countries such as Germany.

Currently, only a handful of health authorities have specific gene therapy regulations, with others prone to using existing guidelines for compassionate use in early access regulations. This is a template that often doesnt fit for gene therapy. While the guidelines allow access, they dont necessarily aid in reimbursement or the ongoing tracking of whether the product is successful over a long period.

Then theres the issue of funding. While cell and gene therapies have the potential to cure several conditions, the technology is still in its infancy and remains prohibitively expensive. Typically, the medicines in development are high-value, niche products that are only formulated in countries where there is sufficient demand or wealth to pay for them. Payment schemes can be put into place although this process must be tightly managed, and crowdfunding has also been used to fund treatment, but it is not without risk.

According to Rebecca Bibby, general manager at leading medicines access solutions provider BAP Pharma, addressing these barriers is crucial for ensuring timely access to medicines for patients: Niche disease groups are where these medicines can really help, but its often not as widely known amongst pharma companies that there are access regulations for gene therapy, as well as heavily regulated processes to help provide access to countries where products arent licensed.

Streamlining medicines access with the expertise of a third-party provider can help pharma and biotech companies to overcome many of these challenges. Working with both the patient and the pharmaceutical company to help address accessibility issues, a medicines access provider supports both sides of the coin, developing payment plans or confirming funding availability, overseeing document preparation and translation, navigating the regulatory bodies and negotiating for both parties to ensure access to potentially life-saving treatment for the patient.

According to Bibby: There are a lot of nuances in the process that make it a complex environment. At BAP Pharma, we work for both sides to make sure that the product is available to those who need it, whilst also meeting regulatory requirements and managing follow-ups required by the innovator built into the project.

BAP Pharma has a dedicated medicines access division that works to bridge the gap between patients and the companies developing these advanced medical treatments. It partners with its clients to provide bespoke medicines access programs that ensure patients can get the treatment they need, whether it is pre-license or post-MA.

Its impossible for pharmaceutical companies to understand the intricacies of every territorys regulations and how they change depending on the type of product because their main focus is to manage commercial products and marketing authorisations, says Bibby. We manage access programs every day and have collated all that expertise in-house, so we fully understand the requirements to support our clients and HCPs.

It is about listening, understanding, and having the knowledge to help. The main benefit is getting patients treated and that can be forgotten amid all the regulations. There is a financial element, but the part we get the biggest reward from is getting drugs to patients who have an unmet medical need.

To learn more about overcoming challenges and maximising opportunities in medicines access, download the free whitepaper below.

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Eyedrop Version of Krystals Gene Therapy Vyjuvek Restores Sight in Teen – BioSpace

Pictured: Doctor administers eye drops to a boy/iStock,Wavebreakmedia

Krystal Biotechs topical gene therapy Vyjuvek (beremagene-geperpavec) in an eyedrop formulation has restored the vision of a 13-year-old boy with the genetic condition dystrophic epidermolysis bullosa that scarred his eyes, according to a case brief published Thursday in The New England Journal of Medicine.

The patientnamed Antonio, as reported by the Associated Presspresented with chronic wounds and webbed hands and fingers, with a best-corrected visual acuity of 20/40 in the right eye and 20/80 in the left eye. Slit-lamp examination revealed scar tissue in both eyes.

Antonio initially underwent surgery on his left eye but the lesions returned after three months and visual acuity worsened to 20/400 about a year after the procedure. Eventually, blisters also began to develop in his right eye. Antonio continued to receive aggressive interventionsincluding a second surgery and other topical and injected treatmentsbut lesions continued to recur.

In 2020, the patient participated in a Phase III trial of Vyjuvek, which triggered significant healing of his wounds. After the FDA cleared an Investigational New Drug application for compassionate use of Vyjuvek for Antonios eye scarring, his doctors performed another round of surgery on his right eye, followed by the topical administration of the Vyjuvek eyedrops.

Within three months and after a total of 19 Vyjuvek doses, Antonios corneal epithelium fully healed based on an evaluation using slit-lamp examination and optical coherence tomography. At eight months, assessments revealed complete epithelial healing and imaging found no signs of corneal scarring, recurrence or other abnormalities.

Antionio also demonstrated better visual acuity after Vyjuvek treatment, improving to 20/25 at eight months. Intraocular pressure and retinal integrity had also normalized following the intervention.

In terms of safety, the NEJM paper flagged one surgery-related serious adverse event that required prolonged hospitalization and resulted in vomiting. However, Vyjuvek treatment was not halted and the patient was eventually discharged.

Our data support further investigation of [beremagene geperpavec gene therapy] in the care of patients with dystrophic epidermolysis bullosa with ocular surface involvement, the researchers wrote in the brief, noting that Antonios case supports the potential of Vyjuvek to promote epithelial healing in the cornea.

Vyjuvek is a topical gene therapy that delivers two functional copies of the COL7A1 gene, which in dystrophic epidermolysis bullosa (DEB) is made faulty by genetic mutations, leading to dysfunctional type VII collagen proteins. DEB manifests as extremely fragile skin prone to wounding and tearing even from light contact. Some cases of DEB can also affect the eyes.

In May 2023, the FDA approved Vyjuvek for the treatment of DEB in patients aged six months and older, making it the first-ever redosable gene therapy in the market.

Tristan Manalac is an independent science writer based in Metro Manila, Philippines. Reach out to him on LinkedIn or email him at tristan@tristanmanalac.com or tristan.manalac@biospace.com.

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Gene therapy as eye drops improves vision for DEB boy – Epidermolysis Bullosa News

The gene therapy beremagene geperpavec (B-VEC), applied directly as eye drops, improved the vision in a boy with eye scarring related to recessive dystrophic epidermolysis bullosa (DEB), according to a case report.

Our data support further investigation of B-VEC in the care of patients with dystrophic epidermolysis bullosa with ocular surface involvement, its authors wrote.

The first-of-its-kind case was described in The New England Journal of Medicinein a paper, titled Ocular Gene Therapy in a Patient with Dystrophic Epidermolysis Bullosa.

B-VEC uses a modified version of a herpes simplex virus (HSV-1) to deliver to cells a healthy version of the COL7A1 gene, mutations in which cause DEB. Krystal Biotech developed a gel formulation of the gene therapy designed to be applied to skin wounds called Vyjuvek, which last year made history as the first treatment for DEB to receive approval in the U.S.

We are excited by this initial data suggesting additional applications of our proprietary HSV-1-based gene therapy platform to treat ocular diseases, and we are working with the FDA [U.S. Food and Drug Administration] to get B-VEC approved for the treatment of DEB patients with lesions in the eye, Suma Krishnan, president of research and development at Krystal, said in a company press release.

DEB is characterized by fragile skin that easily tears and blisters. In more than one in four patients, the disease also affects the eyes, which can lead to wounds or scarring on the surface of the eye that impair vision.

Patients with ocular complications have no corrective treatment options leaving them at risk of severe vision loss, said Alfonso Sabater, MD, PhD, senior author of the study at the University of Miami Miller School of Medicine.

The subject of the report is a 13-year-old boy with DEB who had participated in the Phase 3 GEM-3 clinical trial (NCT04491604), one of the studies that paved the way for the approval of Vyjuvek. In the study, the boys skin wounds had been responding well to the gel-based gene therapy.

Since early childhood, the boy had been experiencing eye problems, including substantial scarring over the eyes. By the time his vision was deteriorating, he underwent surgeries twice to correct a fusion between the globe and the inner surface of the eyelid. However, the same issue, called a symblepharon, recurred a few months after both surgeries.

Because the boy had good response to Vyjuvek for skin wounds, it was proposed that B-VEC applied into his eyes might provide a more long-lasting solution to improve his vision. To facilitate this, scientists at Krystal created an eye-drop formulation of the gene therapy. After running needed preclinical testing and obtaining clearance from the FDA, the B-VEC eye drops were applied.

At the time he received the gene therapy, the patients vision was poor enough that he was considered legally blind. His right eye, which was more affected, had a visual acuity labelled as hand motion, which refers to testing whether or not a patient sees movement of the examiners hand directly in front of his eyes.

A third surgery for a symblepharon in the right eye was undertaken. Then starting immediately after the surgery, B-VEC was applied to the eyes. The gene therapy was given three times per week for the first two weeks, then weekly, for a total of 19 doses.

The boy also wore a bandage contact lens for a month after the procedure, and his eyes were treated with a steroid (to reduce inflammation) and an antibiotic (to prevent infection).

Eight months following the procedure, the boys eye had healed, and the symblepharon had not recurred. The visual acuity in his right eye was measured at 20/25, which was a substantial improvement.

Over the course of follow-up, the patient experienced serious complications from a gastrointestinal surgery. While its impossible to say definitively that B-VEC didnt cause these complications, the researchers said it seems unlikely the eye-applied gene therapy was related to intestinal surgery complications. The surgery complications were resolved with appropriate care, and no other major safety issues were reported.

Larger studies and longer follow-up are needed to provide confirmation of our findings, the team concluded.

We are encouraged by the improvements observed in the patient following B-VEC administration as an eyedrop directly to the affected eye and believe this data is supportive of further investigation in DEB patients with ocular complications, Sabater said. If approved, this approach could drastically benefit these patients.

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Glaucoma 360: ‘Innovation is marching on’ – Ophthalmology Times

Video Transcript

Editor's note- This transcript has been edited for clarity.

We are here today at Glaucoma 360 in San Francisco, California, with Dr. Andrew, Iwach and Dr. Adrienne Graves. Please tell us how you think the meeting is going so far, and also what you are looking forward to most in the development of glaucoma treatment and diagnosis.

Well, thank you for having us, first of all, and we're actually thrilled and really excited today. We've just finished the morning session. This is our 13th annual Glaucoma 360 New Horizons Forum. When we first started well, first of all, there was no MIGS device on the market. So a lot has changed since then. A few people said to us at the beginning, "Do you really think it should be an annual meeting? There's not all that much innovation in Glaucoma?" Well, this is our 13th year and innovation is marching on incredibly. And it has been an exciting meeting so far. We have had some interesting sessions so far. We are so pleased with the both the industry participation, the KOL participation, and the entrepreneurs and innovators. There's there's so much happening, and we're really looking at some of the most exciting things including neuroprotection, and vision restoration, among other things.

I mean, it really has been an amazing morning, so far. We had Sean Ianchulev give a nice summary of the many things that he's working on, and he also announced a new product that he's hoping to bring to market very soon. Then we had Ike Ahmed, who gave the big picture. He brought out his crystal ball and kind of predicted where we will be. We've had presentations from industry panelists who are very open, really telling us where they are what they want to do over the next year. It is really setting us up for our 2025 meeting, because they've told us kind of where they want to be. I'm looking forward to getting updates next year. And that's one of the nice things as Adrienne said, when we started this people say, oh, gosh, is there enough? In fact, we even shared some Medicare data that shows that, my goodness, there's such changes in what we're doing. This is a very dynamic, active time. We also had input from a patient who came up on stage said this makes a difference.

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Revolution in sickle cell disease treatment on the way in Europe – The Pharma Letter

Genetic disease company Vertex Pharmaceuticals (Nasdaq: VRTX) has secured conditional marketing authorization for Casgevy (exagamglogene autotemcel) in Europe.

Also known as exa-cel, the product is a CRISPR/Cas9 gene-edited therapy for people with severe sickle cell disease (SCD).

Contingent on further data confirming benefit, the European Commission has authorized marketing of the product for certain patients over the age of 12, where a matched stem

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