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Nanoscope Therapeutics Enhances Mutation-Independent Retinal Gene Therapy Programs with Appointment of Allen … – PR Newswire

Posted: April 27, 2024 at 2:39 am

DALLAS, April 25, 2024 /PRNewswire/ -- Nanoscope Therapeutics, Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, announced today the appointment of Allen C. Ho, MD, FACS, FASRS, as Chief Medical Advisor, where he will help define the strategy of developing the gene mutation-agnostic therapies. Dr. Ho is Attending Surgeon and Director of Retina Research at Wills Eye Hospital and Co-Director of the Wills Eye Hospital Retina Service.

"We are excited to have Dr. Allen C. Ho join Nanoscope as Chief Medical Advisor," said Sulagna Bhattacharya, Nanoscope co-founder and CEO. "As a world-renowned retina specialist and leader in innovative treatments for retinal diseases, his insight will be invaluable to Nanoscope. MCO-010 has the potential to be the first gene therapy available for patients with advanced retinitis pigmentosa (RP), and Dr. Ho's contributions will help ensure that potential. We also look forward to adding his perspective to our development of MCO-010 for Stargardt disease, building upon the promising results of our Phase 2 STARLIGHT clinical trial."

"I look forward to working even more closely with the team members and leadership at Nanoscope who have been working on ambient light sensitive optogenetic strategies for retinal diseases over the past decade," said Dr. Ho. "The recent release of their 2-year Phase 2b RESTORE Trial data is an important moment for the retina community and I am pleased to help deliver this transformational treatment to serve those suffering from advanced RP. Further, I am also excited about the Nanoscope pipeline and translational potential of image-guided non-viral laser delivery of MCO-020 in improving vision in patients with geographic atrophy secondary to age-related macular degeneration".

Allen C. Ho, MD, FACS, FASRSAllen C. Ho MD, FACS is Attending Surgeon and Director of Retina Research and Co-Director of the Retina Service of Wills Eye Hospital, Professor of Ophthalmology at Thomas Jefferson University and partner of Mid Atlantic Retina. He maintains special interests in macular diseases, diabetic retinopathy, surgical retinal diseases and clinical trials investigating new treatments for vitreoretinal diseases including gene and cell therapies and new surgical drug delivery devices and techniques. His experience includes collaborative translational and clinical trial clinical research with expertise in study design, methodological testing, data analyses, surgical instrumentation and procedure development, execution and communication of these studies and their study results, and commercialization focusing on patient access to care. Dr. Ho has served on the US FDA Ophthalmic Device Panel, American Academy of Ophthalmology (AAO) Innovation Task Force, AAO Ophthalmic Retina Technology Assessment Committee, AAO Retina Measures Group, AAO IRIS Registry Committee and is a leader of Eyecelerator, the largest ophthalmic innovation meeting in the world.

AboutNanoscope Therapeutics, Inc.Nanoscope Therapeutics is developing gene-agnostic, sight-restoring optogenetic therapies for the millions of patients blinded by inherited retinal diseases, for which no cure exists. The company's lead asset, MCO-010, recently reported topline results from the RESTORE Phase 2b/3 multicenter, randomized, double-masked, sham-controlled clinical trial in the U.S. for retinitis pigmentosa (NCT04945772). The company has also recently completed their end-of-study visit for the Phase 2 STARLIGHT trial of MCO-010 therapy for patients with Stargardt disease (NCT05417126). MCO-010 has received FDA fast track designations and FDA orphan drug designations for both retinitis pigmentosa and Stargardt disease. Preclinical assets include non-viral laser-delivered MCO-020 gene therapy for geographic atrophy. To learn more, visit http://www.nanostherapeutics.com and follow them on LinkedIn.

Investor Contact: Argot Partners 212-600-1902 [emailprotected]

SOURCE Nanoscope Therapeutics

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Nanoscope Therapeutics Enhances Mutation-Independent Retinal Gene Therapy Programs with Appointment of Allen ... - PR Newswire

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Gene Therapy Trial for Sickle Cell Disease Publishes Final Results – Technology Networks

Posted: April 27, 2024 at 2:39 am

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In a landmark study, an international consortium led by researchers at Childrens Hospital of Philadelphia (CHOP) published the final results of a key clinical trial of the gene therapy CASGEVY (exagamglogene autotemcel) for the treatment of sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises (VOCs). The study found that 96.7% of patients in the study did not have any vaso-occlusive crises (VOCs) a blockage that results in lack of oxygen and painful episodes for at least one year, and 100% were able to remain hospitalization-free for the same length of time.

The findings, published today in theNew England Journal of Medicine, provide the complete details of the critical clinical trial that led to the FDA approval of CASGEVY for the treatment of sickle cell disease in December 2023.

Sickle cell disease is a lifelong condition that causes intense pain due to deformed blood cells that can cause blockages in blood vessels. This can also lead to strokes, organ damage, and shortened lives.

Researchers have been studying the use of gene therapy and CRISPR technology to edit portions of DNA in people with inherited or genetic disorders, like sickle cell disease. In the case of sickle cell disease, the CASGEVY process edits DNA within the patients own cells and enables the patient to produce a different form of hemoglobin in their red blood cells. Clinical trials at CHOP and other sites have shown that successful gene editing can prevent cells from developing the distinctive crescent shape apparent in sickle cell disease and have eliminated pain episodes in almost all patients. CASGEVY was the first FDA-approved therapy developed with CRISPR technology.

In this clinical trial, sickle cell patients who were having significant issues with their disease began to see their problems resolve within months and improve their quality of life significantly, said senior study authorStephan A. Grupp, MD, PhD, Section Chief of theCellular Therapy and Transplant Section, Inaugural Director of the Susan S. and Stephen P. Kelly Center for Cancer Immunotherapy, and Medical Director of theCell and Gene Therapy Laboratoryat CHOP. Grupp was also one of the principal investigators in the clinical trials that led to the approval of CASGEVY and the leader of the studys steering committee.

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A total of 44 patients received exa-cel with a median follow up of 19.3 months. In a total of 30 patients with sufficient follow-up data to be evaluated, 29 (96.7%) were free of VOCs for at least 12 consecutive months. This information is an update for the US Prescribing Information for CASGEVY, which includes an evaluation of 31 patients resulting in a response rate of 93.5%. The safety of treatment was comparable to treatment with hematopoietic and progenitor stem cells, and no malignancies were reported as a result of treatment.

This study was supported by Vertex Pharmaceuticals and CRISPR Therapeutics.

For more information about CASGEVYs prescribing information, warning and precautions, and adverse reactions, visithere.

Additionally, the results of a clinical trial on the efficacy of exa-cel for the treatment of -thalassemia were also published today in theNew England Journal of Medicine. The preliminary results of the trial led to the FDA approval of CASGEVY for transfusion-dependent -thalassemia in January 2024. Grupp was also one of the principal investigators for this clinical trial.

Reference:Frangoul Haydar, Locatelli Franco, Sharma Akshay, et al. Exagamglogene autotemcel for severe sickle cell disease. NEJM. 2024. doi: 10.1056/NEJMoa2309676

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source. Our press release publishing policy can be accessed here.

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Gene Therapy Trial for Sickle Cell Disease Publishes Final Results - Technology Networks

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US approves gene therapy treatment for hemophilia | National | elpasoinc.com – El Paso Inc.

Posted: April 27, 2024 at 2:39 am

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US approves gene therapy treatment for hemophilia | National | elpasoinc.com - El Paso Inc.

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Regeneron and Mammoth team up to develop gene editing therapies – Pharmaceutical Technology

Posted: April 27, 2024 at 2:39 am

Regeneron Pharmaceuticals has collaborated with Mammoth Biosciences to develop in vivo clustered regularly interspaced short palindromic repeats (CRISPR)-based gene editing therapies.

This partnership aims to research, develop, and commercialise treatments that can target multiple tissues and cell types.

As part of the agreement, Mammoth is set to receive $100m, which includes $95m in equity investment at signing, along with an upfront payment from Regeneron.

The company could also receive up to $370m per target in milestone payments, along with royalties on future net sales of all collaboration products.

Additionally, Mammoth retains the option to co-fund and share profits on most collaboration programmes.

Regeneron, in turn, gains broad access to editing technologies of Mammoth for five and a half years, with an option to extend for two more years.

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The companys expertise in developing adeno-associated viral vectors (AAVs) and antibody engineering will be combined with Mammoths ultracompact nucleases and associated gene editing systems, to create disease-modifying medicines.

These medicines can be delivered to tissues beyond the liver, a current limitation in the field.

Regenerons AAVs are being developed using antibody-based targeting to improve genetic medicine payload delivery to specific tissues and cell types.

Regeneron Genetic Medicines senior vice-president and co-head Christos Kyratsous said: After years spent developing our next-generation delivery approaches, we are eager to combine them with Mammoths gene editing systems to better match payload, delivery system and disease type.

Mammoth Biosciences co-founder and CEO Trevor Martin added: Mammoths ultracompact CRISPR systems address the size constraints of viral delivery and complement Regenerons targeted AAV technologies. We look forward to working with Regeneron to enable all-in-one AAV delivery and unlock the true potential of in vivo gene editing.

Last month, Regeneron secured the US Food and Drug Administrations extended approval of Praluent as an adjunct to diet and other low-density lipoprotein cholesterol-lowering treatment to include paediatric subjects of the age eight and above with heterozygous familial hypercholesterolemia.

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Regeneron and Mammoth team up to develop gene editing therapies - Pharmaceutical Technology

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Sequencing Hope: New film details Alabama girl’s historic gene therapy in Minnesota – KARE11.com

Posted: April 27, 2024 at 2:39 am

After her life-saving treatment in 2021, 7-year-old Celia Grace Hamlett's story of hope became part of the Minneapolis St. Paul International Film Festival.

MINNEAPOLIS A documentary that just premiered during the Minneapolis St. Paul International Film Festival marks the latest twist in a medical journey that has spanned thousands of miles and last several years for the family of a young girl from Alabama.

"We've been so excited and waiting on this," said Gary Hamlett as he walked into the premiere of "Sequencing Hope" with his 7-year-old daughter Celia Grace Hamlett.

KARE 11 first connected with Celia Grace and her family nearly three years ago, when she and her family traveled from their home in Alabama to undergoing an experimental gene therapy treatment at M Health Fairview Masonic Children's Hospital in 2021 that offered her a chance at a normal life.

"(MLD) affects the brain, it affects the nerves, it's progressive and it's lethal," said Dr. Paul Orchard, the Pediatric Blood and Marrow Transplant Physician at M Health Fairview who petitioned the FDA to make Celia Grace the first patient to receive gene therapy to treat MLD in the United States. "I think it's really an opportunity to move the field forward and to better treat the kids."

Thefilm documents how Celia Grace has done exactly that. It follows her and her family through the long journey to that therapy, and her life in the years since.

"She is doing so well, you would never be able to tell she was sick," said Kassie Hamlett, Celia Grace's mother. "Everything is the same as it was, so it has not progressed any at all, so just praying it continues to stay that way."

The history made by Celia Grace came too late for Pat Lang. Her daughter, Maddie Lang, was diagnosed with MLD long before gene therapy was an option.

The documentary also followed Pat's care for Maddie before she died from MLD in 2022.

"The fact there is now something that can save them is a beautiful thing," Pat Lang said. "Am I jealous? You bet. But is it a great thing? You bet."

Just before the documentary premiered, another great thing happened. The FDA approved the gene therapy treatment, opening the door to many others.

"It's a very exciting thing to be able to be able to offer this now and it's not just MLD, it's sickle cell disease, and a number of other inherited diseases," Dr. Orchard said. "They are going to have the same type of opportunity, so this is really where the field is going to go."

"I want people to see our documentary and not give up hope," Kassie said. "The road is rough, but the end is very rewarding."

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Sequencing Hope: New film details Alabama girl's historic gene therapy in Minnesota - KARE11.com

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Meeting the cell and gene therapy scale-up challenge – Life Sciences Intellectual Property Review

Posted: April 27, 2024 at 2:39 am

Improving patient access is a UN goal but for companies transitioning from trials to full-scale production, IP is essential, explains Katherine Collins of Mewburn Ellis.

One of the pivotal challenges in the development and commercialisation of cell and gene therapies (CGT) lies in scaling up production. This challenge is particularly pronounced as companies transition from clinical trials to full-scale commercialisationa major step in the lifecycle of any innovation of this kind.

It could also be a significant factor in achieving good health and wellbeing for all.

The inability to scale up the manufacturing process to produce the required amounts for clinical trials and commercial therapy can lead to the failure of a product.

As part of the approval process, regulatory bodies require thorough documentation of the production processes, making it difficult to alter manufacturing processes later in the commercialisation process.

Draft FDA guidance titled "Manufacturing Changes and Comparability for Human Cellular and Gene Therapy Products" addresses this issue by stating: We recommend that any extensive manufacturing changes be introduced prior to initiating clinical studies that are intended to provide evidence of safety and effectiveness in support of a Biologics License Application (BLA).

Scale-up concerns grow

As concerns around manufacturing and scale-up have increased, investors commonly look at whether companies have well-thought-out plans for scaling up. In a survey of factors that were most likely to influence their decision-making process for investing in a cell and gene therapy company, manufacturing and scale-up were chosen as a top-three factor by 37% of respondents in 2018.

However, by 2021, this number had increased to 48% of respondents. Similarly, the number of respondents ranking manufacturing and scale-up challenges in the top three greatest barriers in the decision to invest in a CGT product opportunity increased from 45% to 63%.

These aspects mean that the "rush to the clinic" approach that was commonly taken with small molecule drugs does not work well for cell and gene therapies. Instead, aspects of scale-up and manufacturing ought to be considered early in the development process.

The realisation that manufacturing was a significant hurdle to getting to market and for successful commercialisation of a product has shifted the focus of many cell and gene therapy companies.

However, for the cell and gene therapy industry, scaling up is not only a hurdle but also an opportunity for innovation. Intellectual property plays a vital role in protecting and commercialising such innovations.

Automation changes the game

For cell and gene therapy companies, any improvement or advantage in their production process can be a valuable asset, potentially eligible for patent protection. Many companies in the field are focusing on platforms dedicated to enabling efficient scale-up. For such companies, having a strong IP portfolio can allow them to attract the investment required to increase capacity and market reach, providing their scale-up solutions to a wider audience.

Automation is emerging as an important component in the scaling up of cell therapies. Companies such as Mytos, Miltenyi BioTec, and Cellular Origins have produced automated, closed cell processing platforms. These platforms promise not only larger and more efficient batches of cells but also enhanced reproducibility. This minimisation of variability is a crucial aspect in meeting regulatory standards and ensuring consistency in therapeutic outcomes.

There is also a push to move to a full and comprehensive digitised approach to data collection. Previous manual and/or paper-based recording can be a bottleneck to batch release of a product. Furthermore, capturing the full data during processing and manufacture of the cells allows for a more thorough understanding of the underlying causes of process deviation and differences in batch outcomes. By integrating digitised data collection early on in development, processes can be improved and optimised more quickly.

AI-based optimisation can also play a role in scale-up. For example, Form Bio uses AI-based optimisation to design vectors for the expression of CARs and T-cell receptors in therapeutic cells. The AI model predicts secondary and tertiary structures in the vector that could lead the replication machinery to fall off and optimizes the sequence to prevent this.

Manufacturing aspect

The improvement and streamlining of manufacturing play a huge role in patient access. Labour costs represent a significant portion of the overall expense in cell therapy production. It is estimated that labour costs make up around 50% of the cost of a cell therapy product.

Therefore, manufacturing issues are not only a barrier to the development of a product, but once approved, they may also be a barrier to patient access. With the high costs of manufacturing being a major factor in the high per-patient costs of such therapies, the products become inaccessible to many.

Reducing the manufacturing costs would therefore contribute to the commercial viability of a therapy and facilitate wider patient access. With the withdrawal of several cell and gene therapy products from certain regional markets due to commercial reasons, thinking ahead and investing in the manufacturing process to make it as financially viable as possible is crucial.

As well as technological advances in manufacturing per se, a move towards partnerships and the sharing of manufacturing resources can also help provide solutions to these issues.

The investment in the processes and facilities required for manufacturing cell and gene therapy products is a barrier for many small and even mid-sized companies in this space. Outsourcing the manufacturing to contract development manufacturing companies (CDMOs) or partnering with a larger company are two routes to overcome this issue. As well as providing the physical manufacturing resources, CDMOs and larger, more experienced companies can bring their expertise to a given products development to increase the chances of success.

Portfolio approach: More efficiency, different risks

Some companies and investors in the field are seeking to resolve these issues by taking a portfolio-based approach. They may secure common manufacturing approaches, technologies, and/or infrastructure that allow for consistency and predictability in operations across the portfolio companies' various assets. This approach allows companies to spend less money on infrastructure and focus their resources on the actual development of a product.

As with any approach that involves the sharing of technologies and resources, having strong IP in place around a partys assets is important. Companies with strong IP portfolios are also more attractive to investors and potential partners as it demonstrates the companys ability to protect its innovations and maintain a competitive advantage in the market.

In conclusion, the scaling-up challenge is a critical point in the development of cell and gene therapies. Companies that invest time in planning a route forward have a much greater chance of success.

Companies are currently developing and investing in scalable technologies and automation, as well as looking to data-driven approaches to address current hurdles. As the cell and gene therapy field progresses, these advancements pave the way for more therapies to successfully move from development, to clinical trials, and on to commercialisation.

Therefore, with advances in manufacturing of cell and gene therapy products ultimately leading to wider patient access, these technologies could play a key role in the UNs third Sustainable Development Goal, which is to ensure healthy lives and promote well-being for all at all ages.

Katherine Collins is an associate, patent attorney (life sciences) at Mewburn Ellis.

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