Archive for the ‘Gene Therapy Research’ Category

A pharmacist at a health-care centre in India dispenses free medicine to a person with sickle-cell disease.Credit: Rafiq Maqbool/AP Photo/Alamy

Last November, the United Kingdom became the first country to authorize the use of a therapy called Casgevy, based on CRISPR gene-editing technology, for the treatment of sickle-cell disease. Within a few weeks, three other countries the United States, Bahrain and Saudi Arabia had done the same. In December 2023, the United States also approved the use of Lyfgenia, another gene therapy, to treat the disease.

Sickle-cell disease kills nearly 400,000 people each year globally. It causes red blood cells to become sickle-shaped and clog blood vessels, which can result in severe pain and tissue damage, among other problems1. More than 75% of people with the disease one of the most common inherited disorders in the world are born in sub-Saharan Africa and India. The 2021 Global Burden of Disease Study, a regional and global effort to assess mortality and disability resulting from major diseases, found it to be the 12th leading cause of death globally in children under five1. In Africa, more than 50% of infants or children diagnosed with sickle-cell disease die before the age of five2.

Until the advent of gene therapies, the only cure was a bone-marrow transplant. This requires finding a healthy donor, followed by invasive treatment over weeks3.

Given all this, obtaining regulatory approval for two gene therapies is a phenomenal achievement. Yet at prices of US$2.2 million and $3.1 million per treatment, respectively, for Casgevy (exaxamglogene autotemcel) and Lyfgenia (lovotibeglogene autotemcel), the risk is that both will be withdrawn from the market because too few people or health-care systems can afford them.

This has already happened for four other gene therapies, including a related treatment called Zynteglo (betibeglogene autotemcel), for -thalassaemia. Another inherited blood disorder, -thalassaemia causes anaemia, tiredness and weakness in about 3% of the global population, but its incidence can reach 20% in regions including Africa, the Middle East and southeast Asia4. In 2021, Zynteglo was withdrawn from the European market after its developer, the pharmaceutical company Bluebird Bio, based in Somerville, Massachusetts, failed to persuade public bodies such as the United Kingdoms National Institute for Health and Care Excellence (NICE) to approve it. Zynteglo costs $1.8 million per treatment.

Researchers, drug developers and drug manufacturers are squandering the chance to make gene therapies viable even in wealthier countries, let alone transformative for the world. To seize the opportunity, they must take into account the populations most in need, as well as the global market for treatments both when developing and when valuing the drugs. This means reconsidering who is enrolled in clinical trials and where those trials are conducted. It also means partnering with low- and middle-income countries (LMICs), and facilitating the development and production of gene therapies in the nations that need them most.

Casgevy and Lyfgenia are among the latest examples of treatments (see Forging ahead) that involve making alterations to peoples genomes through the replacement, deletion or insertion of genetic material. Since 2004, regulators worldwide have authorized 30 other gene therapies for genetic disorders, including immune and red-blood-cell disorders, retinal degeneration and leukodystrophy a set of conditions affecting the central nervous system as well as for severe cases of some cancers5. Of the 12 genetic disorders that can now be treated using gene therapies, 9 were previously incurable.

Source: Ref. 5

For Casgevy and Lyfgenia, the challenges didnt start with costs. In both cases, participants in the clinical trials did not represent the populations most affected by the disease they are meant to treat.

For Casgevy, trial participants were from the United States, Canada or Europe. For Lyfgenia, the pilot trial included three people from France. All participants in the main trial were from the United States, and the drug was administered only in US clinics. But in North America and Europe, around 1 in 2,000 to 1 in 3,300 people have sickle-cell disease, compared with around 1 in 1,300 in South America and the Caribbean, 1 in 1,000 in India, 1 in 500 in the Middle East, and 1 in 100 in Africa6.

Whats more, because US and European regulators urge drug developers to focus first on those who are made sickest by the disease (which, in the United States and Europe, includes adults), all trial participants for both drugs were older than 12 and most were older than 21 despite the preponderance of sickle-cell disease among children globally. Casgevy is now being evaluated in a phase III clinical trial in people aged 211, but only in the United States, the United Kingdom, Germany and Italy.

This lack of representation in drug trials of those who are most affected by the disease is a violation of article 27 of the Universal Declaration of Human Rights, which states that everyone has the right to share in scientific advancements and its benefits. It is especially problematic for gene therapies for diseases that are prevalent in Africa, where human populations are the most genetically ancient and diverse in the world. Treatments might not work in genetic contexts different from those in which they were tested7, and to produce gene therapies that are as effective as possible globally, drug developers should be testing them in populations that are ancient in evolutionary terms.

Failure to consider the global population during trials could affect the effectiveness and safety of drugs in diverse populations. It is certainly affecting the pricing of gene therapies.

The multimillion-dollar price tags which exclude the costs of clinical care do not actually reflect how much the drugs cost to manufacture. Besides regulatory and research-and-development costs, these prices reflect the perceived maximum value that these therapies add to the person being treated and to society for example, through avoiding medical costs that would otherwise be incurred during someones life under standard clinical care. (People with sickle-cell disease might receive pain medication or transfusions of red blood cells during a pain crisis, say.) Such value-based pricing assessments are generally made by the drug developers themselves, or by a lengthy government review process. Typically, developers will evaluate their own products and governments and independent researchers conduct independent evaluations.

A person with sickle-cell disease receives a blood transfusion at a hospital in Kansas City, Missouri.Credit: Tammy Ljungblad/The Kansas City Star/Tribune News Service via Getty

Yet the way in which gene therapies are currently valued is riddled with difficulties.

Most value-based pricing models do not consider need, affordability or disease prevalence. Moreover, the perception of value varies for different populations and can change over time. In Europe and the United States, for example, sickle-cell disease is classed as a rare genetic disease (even though worldwide it is one of the most common inherited disorders). And this categorization changes how a drug to treat the disease is valued; societies are thought to be more willing to shoulder the costs of an expensive treatment if only a relatively small number of people are affected by the disease8.

Estimates of value added assume that recipients of treatments are cured for life. But the data presented by drug companies to authorities, such as the United Kingdoms National Health Service, often come from only two years of follow-up.

Those evaluating the drugs do not consider that lower-priced treatments might become available in the future say, if some part of the manufacturing process becomes automated. Evaluators also assume that manufacturing will only ever happen in high-income countries, even though drugs could be produced more cheaply in nations such as India, where the costs of labour and locally produced components, including gene-editing reagents, are much lower than in Europe or the United States.

Perhaps most crucially, drug evaluators use clinical data, for instance on the number of times people report a pain crisis to health-care providers, collected only from those in the countries where they expect the drug to be marketed9. In other words, estimates of the value of gene therapies are based on clinical data that are not collected from most of the people with the disease that the therapies are designed to treat.

All this means that gene therapies are perceived to be less cost-effective and more expensive to produce than they would be if their development, production, marketing and uptake shifted to countries where the relevant diseases are most prevalent. For sickle-cell disease, this includes Nigeria, India, the Democratic Republic of the Congo, Tanzania and Uganda10.

The capacity of LMICs to pursue research and development is much greater than many people in wealthier nations might assume. The Drugs for Neglected Diseases Initiative, an international non-profit organization, has brought 13 new drugs to market over the past 20 years, in part by including clinicians, researchers and trial participants from countries such as Ethiopia, Kenya, Malaysia, India and Brazil in the research-and-development cycle (see go.nature.com/3vj59yc).

Global inequities in COVID-19 vaccination have been a powerful reminder of the importance of local manufacturing of both vaccines and treatments. And governments across the world have been pushing hard for this.

A woman in Kano, Nigeria, massages the legs of her daughter, who has sickle-cell disease.Credit: KC Nwakalor/New York Times/Redux/eyevine

Through initiatives such as the New Partnership for Africas Development, the African Union is increasingly supporting the local manufacturing of treatments, vaccines and interventions such as mosquito nets, for example. Similar investment is happening in India. In October last year, Indias equivalent of the US Food and Drug Administration, the Central Drugs Standard Control Organization, approved the use of a home-grown chimeric antigen receptor T-cell therapy, called NexCAR19, to treat blood cancers.

The same government buy-in, national entities and infrastructure must support the local manufacturing of approved gene therapies, to give local populations a chance to access them. The cost savings made by shifting the manufacturing of some of these therapies to Africa or Asia could improve peoples access to them in certain high-income countries too. This is particularly true in Europe, which has more regulatory flexibility than does the United States.

Once it became clear, in 2022 and 2023, that regulators would be reviewing both Casgevy and Lyfgenia and probably approving their use for sickle-cell disease, other biotech companies, including Aruvant Biosciences, based in New York City; Sangamo, based in Richmond, California; and Bioverativ, part of the multinational drug-maker Sanofi; abandoned the development of three gene-therapy products for the disease. Although these pipelines were lagging behind those for Casgevy and Lyfgenia, clinical trials had been indicating that these other drugs would also be effective and safe.

Two years of COVID-19 in Africa: lessons for the world

Agreements around intellectual property would need to be negotiated. But, in principle, abandoned drug-development pipelines could be transferred to LMICs immediately. This would jump-start research and development for gene therapies locally, without imposing high-income-country requirements on low-resource settings. In countries such as India, for example, the rules around what clinical treatments (if any) people should have received before undertaking gene therapy will differ from those derived from clinical data collected in the United States11.

Most of the intellectual property in cell and gene therapy is owned by academic research centres. But in cases where a commercial developer owns the intellectual property, tax incentives, expanded government funding or publicprivate partnerships could all support the transfer of technology to regions where the disease of interest is more prevalent.

To help to achieve this kind of technology transfer, governments of LMICs need to build the trust of US and European biotech corporations and academic research centres not just by increasing their own investment in research and training, but also by conducting transparent assessments of their countries scientific, infrastructural and funding capacity.

There is considerable political will to address diseases that hit local populations the hardest. This year, Tanzania committed 3.6 billion shillings (around US$1,400,000) to support the use of bone-marrow transplants to treat children with sickle-cell disease. And depending on how much their costs can be brought down, there could be a strong global market for gene therapies.

No matter their income level, parents will do anything they can to save their childs life. Since 2011, clinics in Nigeria have been providing bone-marrow transplants to Africans to treat sickle-cell disease. In India, hundreds of people have received a bone-marrow transplant to treat sickle-cell disease over the same period. In most cases, recipients and their relatives have crowdsourced the $25,00050,000 needed per treatment.

The Global Gene Therapy Initiative (GGTI), of which we are both founding members, aims to enable people anywhere in the world to find out more about the development of gene therapies. Last year, one of our colleagues, Elizabeth Merab, an award-winning Kenyan journalist in health and science, and a member of the GGTIs international advisory board, died from complications of sickle-cell disease at the age of 31.

In 2022, Merab addressed various stakeholders, including one of us (J.E.A.), at a meeting about the role of gene therapies in treating sickle-cell disease and HIV at the Sunnylands Estate in Rancho Mirage, California. It had been three years since news outlets around the world had reported how, in a much-anticipated experiment, clinicians had used a CRISPR-based gene therapy to try to treat sickle-cell disease in a woman called Victoria Gray.

At the Sunnylands Estate, Merab, who was diagnosed aged ten, told us how she had overheard the doctor tell her parents that her options were limited. Hydroxyurea, a drug that reduces symptoms for some people, became available in the 1980s, but didnt work for her. Later, she learnt about bone-marrow transplants, but her doctors told her that she would have to receive the treatment outside Africa and pay for it herself. Then, at the meeting, she learnt about gene therapy for sickle-cell disease.

The only thing more difficult than hearing that you have a disease for which there are no cures, is to hear that you have a disease for which cures are available, but they are not available to you, she told us.

Everyone with a devastating disease should have access to a cure when one exists. True progress will come only when low-income countries are included in the development of gene therapies.

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Make gene therapies more available by manufacturing them in lower-income nations - Nature.com

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JCR Pharmaceuticals Highlights Innovative Gene Therapy Research at the 7th International Forum of Lysosomal Disorders - The Bakersfield Californian

The gene therapy marketis predictedto experience rapid growth over the coming years. But with that growth will come many complexities that contract research organizations (CROs) must navigate to keep these unique clinical trials on track.

Despite just 39differentgene therapies recorded on the market in June 2023, according to GlobalData figures, more than 4,450 gene therapy drugs are currently in preclinical and clinical trials.

GlobalDatas State of the Pharmaceutical Industry 2024 report predicts that oncology will account for 44% of the cell and gene therapy (CGT) market by 2029. Trials are also increasing in other therapeutic areas, signalling the increase in research funding and product opportunity for diseasessuchas hemophilia, Alzheimers, primary immunodeficiencies, and even hereditary blindness.

The sponsor portfolio is definitelya lotmore diverse these days, notes Nan Shao, Executive Vice President of Global Operations at Veristat. They have greater confidence in cell and gene therapy. And because of that confidence, a lot more funding gets allocated even in the larger companies now investing in developing CGT.

Despite scientific and technological advances, the cost of gene therapy trials remains a significant hurdle, and reducing expenses is a considerable challengegiventhe inherent complications of such trials. That means CROs must find a middle ground for optimizing results within budget constraints.

With a limited number of patients available for trials of rare disease treatments, ensuring accurate data collection and recordkeeping is vital. All the more reason why executing your clinical trials with the right data management platform iskey for achieving accurate data collection, significant savings on trial costs, and a return on investment for pharma and biotech companies, while also ensuring more patients can access the transformative treatments coming from gene therapy trials.

Many of the genetic disorders thatcan be treatedwith gene therapy areextremely rare with some affecting just one person in a million.Gene therapy trials are limited by the number of patients available with certain conditions.As a result, it is vital tomake full use ofany data gathered.

CGT trials require scrupulous management of patients, sites, and sponsors to deliver successful outcomes.In the modern pharma industry,electronic data capture (EDC) has emerged as the best way to reduce inefficiencies, streamline data capture, and optimize a trials results.

Data is of the most critical value for success, and that dependency on data is only going to increasemore and more, notes Shao. However, in a CGT trial, we identified that it is hard for the typical EDCto be ableto capture all the data and adapt to the changes you need very quickly.

Mismanagement of patient data can trigger trial integrity risk or minimally cause inefficiencies in data collection, and place extra burden on sites and CROs to resolve discrepancies.Withgene therapy trial costs already running high, CROs must do everything they can to eliminate these risks and inefficiencies to ensure trials stay compliant, on schedule, and within budget.

Costing is a critical element for us as a CRO. Because many clients that reach out to us have the budget planned upfront, says Shao. These costs are critical to their decision to outsource to another provider, as they need to know we can keep their studies on time and on budget.

Moreover, because CGT trials often recruit froma very smallpatient pool, patient dropouts can severely disrupt a trial and add further costly delays. For cell and gene therapies, the deployment of an effective ePRO solution is critical for helping to foster patient engagement in trials, solicit patient input conscientiously, and ensure that patients understand everything required of them.

I worked on a Phase I cell therapy trial when I was a statistician myself, and it was very different from a typical Phase I study, explains Shao. There were a lot of unexpected challenges along the way regarding data and patients. You dont know how patientsare going toreact to some of these investigational therapies. So, you need tohave the abilityto quickly adjust your database as the study goes and typical EDC setups arent always able to accommodate this.

For their CGT trials, Shao and her team at Veristat have been using the cloud-based EDC platform from Zelta by Merative. The Zelta platform is uniquely flexible and scalable, allowing users to choose the modules they want for their study, making it compatible with virtually all trial types, regardless of complexity or size.

At its core, a trialsmainpurpose is to produce reliable data to understand the efficacy and safety of new treatments. Accurately recording a patients experience is vital. Yet an overcomplicated, hard-to-use EDC platform can also result in data anomalies due to a lack of user understanding, misinterpretations, or data that are missing entirely. The Zelta platform manages highly complex processes without compromising usability, thanks to features like flexible page layouts and automated queries, enabling CGT trial operators to get the most out of their data.

Shao estimates that Veristat has reduced clinical database costs by approximately 30% incertaintrials, resultinginhighlysignificant savings.Compared to other EDC solutions,Zelta has also helped Veristat reduce the database build duration oftheir complex CGT trialsfrom as long as 10 weeks to eight or less.

Being able to shave a couple of weeks off a trials schedule is huge for us, adds Shao. We save on costs, and it also leads to the quicker launch of a product, which is highly meaningful for those affected by diseases.

Alongside reducing the timeframe for launching life-saving therapiesto the market, Veristat found that the Zelta platform which offers ePRO integrated with EDC reduces the burden of technology integrations, both making managing patient data easier and helping patients engage better with trial processes.

Zelta EDC makes transferring patients from one site to another smoother. In long-term studies, patients regularly move to a different place, requiring their data to be easily transferred. Zelta EDC simplifies this process, thereby enhancing patient engagement.

Additional integrated modules, such as ePRO and eCOA forms, help improve a patientsunderstanding of their own conditions and treatments, while also reducing a CROs workload by automatically transporting inputted data to case report forms (CRFs). Scheduled notifications offered by the ePRO remind patients to input data at therighttimes and ensure that databases remain accurate.

For rare disease studies, you dont have a lot of patients you cant lose them, says Shao. Zeltas translation functionalities areespecially importantfor CGT trials, as patients aredistributed across the globeand speak different languages. This feature has absolutely made comprehension for patients far simpler. The Zelta platform is easy to use and has made the trial experience less stressful for patients, which is also highly important.

Zeltas high performance and the willingness of their team to support customers so that the platform can adapt to any trials needs are furtherkeybenefits from a CRO perspective.

In this industry, you have to be able to scale up and meet the different complexities of trials, notes Shao. Through this partnership with Zelta and with the familiarity of many years of work, thechannels of communication between us are streamlined. We can provide feedback and advance the Zelta product so it can better support our work. The Zelta team are very efficient at addressing questions, and that willingness to collaborate is super important to us as we are always under high pressure to deliver.

Adaptable, scalable, and intuitive for patients, Zelta can help CROs manage data demands for even the most complex cell and gene therapy trials.

To learn more about how Zelta by Merative can support CROs in tackling the biggest challenges facing their clinical trials, download the specially commissioned report below.

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Gene therapy: How the CRO of Veristat cut trial database costs by 30% - Clinical Trials Arena

Lewes, Delaware, July 17, 2024 (GLOBE NEWSWIRE) -- The Global Gene Therapy Market Size is projected to grow at a CAGR of 26.88% from 2024 to 2031, according to a new report published by Verified Market Research. The report reveals that the market was valued at USD 7.53 Billion in 2024 and is expected to reach USD 50.6 Billion by the end of the forecast period.

Download PDF Brochure: https://www.verifiedmarketresearch.com/download-sample?rid=40223

Browse in-depth TOC on Global Gene Therapy Market Size

202 - Pages

126 Tables

37 Figures

Scope Of The Report

Gene Therapy Market Overview

Technological Advancements in Genetic Engineering: The gene therapy market is being propelled forward by rapid breakthroughs in genetic engineering technologies. These advancements improve the accuracy and effectiveness of gene therapies, which in turn attract investments and stimulate market expansion. Maintaining a lead in the use of technology guarantees a competitive advantage and creates opportunities for generating additional income.

Increasing Prevalence of Genetic Disorders: The increasing prevalence of genetic abnormalities worldwide is a major factor contributing to the growth of the gene therapy market. The increasing number of patients in search of efficient treatments has led to a boom in the demand for gene therapies. The increasing number of patients presents significant prospects for enterprises to enhance their market share and impact.

Favorable Regulatory Environment: The time-to-market for gene therapies is being reduced as a result of the acceleration of the approval process by supportive regulatory frameworks. Regulatory agencies are increasingly acknowledging the potential of gene therapy, offering incentives and streamlined development pathways. This advantageous environment fosters investment and innovation, which in turn propels market expansion.

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High Treatment Costs: The exorbitant expenses linked to gene therapy treatments continue to be a substantial obstacle. These expenditures have the potential to restrict patient availability and burden healthcare budgets. Businesses must prioritize cost-reduction measures and implement value-based pricing models in order to overcome this obstacle and improve their market penetration.

Ethical and Safety Concerns: The gene therapy market is constrained by ethical issues and potential safety risks. Public acceptance and regulatory clearances can be impeded by concerns regarding the long-term effects of genetic changes. To achieve market expansion, it is necessary to address these concerns by implementing transparent communication and strict safety measures.

Limited Healthcare Infrastructure in Developing Regions: The lack of sufficient healthcare infrastructure in underdeveloped nations hinders the implementation of gene treatments. The market expansion can be hindered by the limited availability of advanced medical facilities and experienced professionals. Establishing strategic alliances and making investments in healthcare development are crucial in order to access these new markets and stimulate future expansion.

Geographic Dominance:

North America has a strong hold on the gene therapy market because of its well-developed healthcare infrastructure, substantial investments in research and development, and favorable regulatory rules. This dominance expedites market expansion as the region evolves into a center for innovation, enticing multinational corporations and promoting progress. The market expansion is also facilitated by Europe's robust biopharmaceutical industry and favorable laws. On the other hand, the Asia-Pacific region exhibits significant potential for quick expansion as a result of escalating investments in healthcare and a growing emphasis on genetic research.

Gene Therapy Market Key Players Shaping the Future

Major players, including Pfizer, Novartis AG, Biogen, Gilead Sciences, Inc., MolMed S.p.A., Spark Therapeutics, Inc., Orchard Therapeutics plc., SIBIONO, Bluebird bio, Inc., Shanghai Sunway Biotech Co. Ltd. and more, play a pivotal role in shaping the future of the Gene Therapy Market. Financial statements, product benchmarking, and SWOT analysis provide valuable insights into the industry's key players.

Gene Therapy Market Segment Analysis

Based on the research, Verified Market Research has segmented the global Gene Therapy Market into Product, Indication, Delivery Method, And Geography.

To get market data, market insights, and a comprehensive analysis of the Global Gene Therapy Market, please Contact Verified Market Research.

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Gene Therapy Market Surges to USD 50.6 Billion by 2031, Propelled by 26.88% CAGR - Verified Market Research - GlobeNewswire

"It is very disheartening for a patient to know there is a potential treatment available, but they can't access it because of where they reside," says Dr. Harsha Rajasimha. "Biopharmaceutical sponsors also struggle to find eligible patients, missing genetic diversity."

While the underlying science for the design and development of CGTx is advancing rapidly, the industry is successfully applying AI-driven drug discovery algorithms to generate drug candidates faster than ever before waiting for clinical evidence generation of safety and efficacy. However, the operational and logistical efficiency gains are yet to catch up to execute over 1000 gene therapy and over 2000 cell therapy clinical trials that are currently active. Most of these qualified CGTx clinical trial sites are in the U.S. and the E.U., while most patients worldwide remain without access.

As Dr. Harsha Rajasimha, CEO and Founder of Jeeva Clinical Trials Inc., notes, "It is very disheartening for a patient or family to know that there is a potential treatment available somewhere, but they can't access it because of where they reside. On the other hand, Biopharmaceutical sponsors are unable to identify enough eligible patients to enroll from within the geographies they operate in. Even when they are successful in enrolling the target number of patients, they are likely missing the genetic diversity or other critical aspects of the disease heterogeneity. Moreover, each genetic disease has a unique distribution of prevalence globally and can disproportionately affect different populations."

Ignoring genetic diversity weakens treatment effectiveness across populations. This has real-world consequences, as exemplified by the Indian diaspora in the US. A study examining 193 rare disease trials found that participants of Indian descent accounted for just 1% of the patient pool. (6) The general dearth of data on Indian patients has led to situations where they received FDA-approved treatments that were ultimately ineffective for their genetic profiles.

Challenges Unique to CGTx clinical trials CGTx represents a novel and advanced treatment modality with one-and-done administration. Clinical trials for these therapies demand patient-friendly trial designs that fit into their lifestyle with the least burden. These studies tend to involve much smaller patient population compared to other trials involving small molecule drugs or biologics. This means, researchers will need to gather more frequent data points from smaller patient populations including from caregivers and observers of the patients. Most of these patients are severely debilitated due to the disease's nature requiring special needs, choice of communication channels such as video, audio, texting, email, and other special accommodations.

Ensuring long-term patient safety remains challenging for CGTx. The FDA requirement for 15 years of safety follow-up after treatment demands integrated clinical trial management infrastructure including remote patient engagement and patient-centric apps. Additionally, CGTx trials have a need to be more inclusive of non-English speakers and more decentralized due to sparse distribution of the target populations.

Given this set of complex requirements, researchers have relied on CROs selecting 20-30 different software tools and point solutions to make one clinical study successful. Net result is siloed data and fragmented infrastructure resulting in significant integration and interoperability costs.

Patient-Centric Solution to Modernize Genetic Disease Clinical Research There is good news! Jeeva anticipated the need for human-centric and inclusive clinical research software solutions early on and has developed and validated mature solutions for almost six years now.

Team Jeeva is pioneering patient-centric clinical research software and CRO solutions to lead the way for genetic disease research and CGTx clinical trials at an Enterprise scale, centralizing the management of all studies under a single-login and subscription-based service. "As a rare dad and patient advocate, I realized the only way to address these challenges holistically is with a comprehensive unified solution rather than with piecemeal point solutions that do not integrate and automate the process end-to-end," Rajasimha recounts. And adds, "We cared enough to make time and investments to thoughtfully develop a cost-effective and human-centric solution for patient registries, natural history studies, CGTx clinical trials, and long-term follow-up studies."

Today Jeeva delivers optimized solutions that combines software, experts, and standard processes collectively as a turnkey configurable solution including study specific patient engagement portals. The company's comprehensive clinical research solutions cover software or CRO solutions for protocol design, IRB submissions, site qualification and initiation, patient screening, referral, randomization or enrollment, clinical data management, clinical trial management, patient engagement, biostatistical analysis, and reporting or manuscript preparation for publications. Optimizing all these processes under one-login makes it cost-effective for small population clinical trials or population scale registries or cohort studies.

"Having validated the clinical research software solution for use in settings such as GCP, 21 CFR part 11, HIPAA, with academic medical centers, Children's hospital, and biopharma sponsors, we are partnering with clinical-stage biopharmaceutical sponsors to accelerate the development of affordable CGTx as well as health economics outcomes research (HEOR) studies", Rajasimha highlights.

"With the Diverse and Equitable Participation in Clinical Trials (DEPICT) Act now in play, (7) sponsors of clinical trials must submit a 'diversity action plan' outlining how they will achieve diverse participant enrollment," says Rajasimha. The Jeeva's inclusive clinical research solutions align with the recent FDA guidance to Improve Enrollment of Participants from Underrepresented Populations in Clinical Trials. (8)

Leading Organizations are Choosing Jeeva to Speed up Clinical Research Modern decentralized and hybrid clinical trials powered by AI are key to unlocking medical breakthroughs. They can boost access and capture richer patient data by making trials remote and inclusive, ensuring diverse representation of patients in their native languages.

COMBINEDBrain, a non-profit organization for rare neurodevelopmental disorders, partnered with Jeeva for this study to capture both quantitative and qualitative data with minimal patient burden. Dr. Terry Jo Bichell, Founder of COMBINEDBrain, states, "We are pleased to collaborate with Jeeva for the eCOA module for our investigator initiator observational study to understand independent toileting abilities by individuals with neurogenetic brain disorders." The study aims to understand the effect of treatments on independent toileting abilities via BYOD surveys with branching logic and scoring, while reducing caregiver and patient burden. Using Jeeva electronic clinical outcomes assessments (eCOA), a software solution that allows researchers to collect, track, and analyze patient and observer reported data, researchers aim to accelerate clinical research programs for various CNS disorders.

CGTx is among the most expensive treatments ever produced, with some exceeding $2 million per dose. (9) We need bold and disruptive models coming from anywhere in the world to ensure access and affordability of CGTx. Jeeva is honored to partner with ImmunoACT to support their vision for making affordable CAR-T Cell therapies. "We needed an innovative platform for our first clinical trial with minimal financial burden and not requiring us to carry a huge IT infrastructure internally. Collaboration with Jeeva was an excellent choice for our CAR-T cell/gene therapy for leukemia and B-cell lymphoma which recently received approval from the Indian regulatory agency for a tenth of the cost compared to the existing CAR-T therapies. This collaboration has been fruitful in storing and sharing clinical data as per regulatory requirements," says Dr. Rahul Purwar, Founder of ImmunoACT and Associate Professor at the Indian Institute of Technology (IIT), Mumbai, India.

Rajasimha and his team were at the ASGCT annual conference earlier this year in Baltimore, MD. Last week, Rajasimha moderated a session on cross-border data sharing for accelerating genetic disease diagnosis and insights at the North America Rare Disease Summit (NARDS) 2024 in Chicago, IL, and will be chairing the Indo US Bridging RARE Summit Nov 16-18, 2024, in New Delhi, India.

Jeeva Pledges Free Software Solutions for N-of-1 Clinical Trials In a groundbreaking move, Jeeva Clinical Trials Inc. has pledged to provide its software free of charge for all N-of-1 clinical trials like the recently conducted phase 1 trial, AAV gene therapy for hereditary spastic paraplegia type 50 indefinitely. (10) This commitment aims to support personalized medicine and ensure that even the rarest conditions receive the attention they deserve, fostering a more inclusive and effective approach to clinical research.

"This is how we walk the talk of patient-centricity in clinical trials", says an optimistic Rajasimha.

About Jeeva Clinical Trials Inc. ("Jeeva") Founded by Dr. Harsha Rajasimha after his deeply personal, family, and professional experiences, Jeeva's mission to "Modernize Clinical Research with Human-Centric Software and CRO solutions" propels their innovative clinical trial management platform with AI and workflow automation under one bundled subscription. Jeeva empowers global biopharmaceutical, medical device, and nutraceutical sponsors, simplifying patient engagement and evidence generation, achieving 70% greater efficiency in research. Trusted by prestigious organizations like Frantz Viral Therapeutics and George Mason University, their technology supports diverse study designs, from traditional to fully decentralized models, across disease areas including oncology and rare diseases. With empathy and determination, team Jeeva has developed a patient-centric and unified platform that enables sponsors to conduct clinical trials more effectively, breaking down barriers and fostering a globally inclusive and diverse participant pool. This includes facilitating remote patient participation. Discover more about how Jeeva is reimagining the future of clinical trials athttps://jeevatrials.com/.

References 1. "Current FDA Approved Gene & Cell Therapies Mirus Bio." Mirusbio, 31 May 2024, mirusbio.com/fda-approved-gene-cell-therapies/. Accessed 5 July 2024. 2. "Gene Therapy Market Size Poised to Surge USD 52.40 Billion by 2033." BioSpace, 18 Apr. 2024, biospace.com/article/releases/gene-therapy-market-size-poised-to-surge-usd-52-40-billion-by-2033/. 3. "FDA Expands Approval of Gene Therapy for Patients with Duchenne Muscular Dystrophy." FDA, 20 June 2024, fda.gov/news-events/press-announcements/fda-expands-approval-gene-therapy-patients-duchenne-muscular-dystrophy. 4. Gardner, Jonathan. "Takeda Drug for Rare Types of Epilepsy Misses Goal in Late-Stage Trial." BioPharma Dive, 24 June 2024, biopharmadive.com/news/takeda-ovid-dravet-lennox-gastaut-failure-soticlestat/719115/. 5. Pagliarulo, Ned. "Biogen Gene Therapy Misses Goal in Eye Disease Study." BioPharma Dive, 14 May 2021, biopharmadive.com/news/biogen-gene-therapy-eye-study/600204/#:~:text=An%20experimental%20gene%20therapy%20did. Accessed 10 July 2024. 6. Chakraborty, Monoswi, et al. "Rare Disease Patients in India Are Rarely Involved in International Orphan Drug Trials." PLOS Global Public Health, vol. 2, no. 8, 15 Aug. 2022, p. e0000890, doi.org/10.1371/journal.pgph.0000890. 7. "Diverse and Equitable Participation in Clinical Trials Act or the DEPICT Act." Congress.gov, Congress of the United States, 3 Feb. 2022, congress.gov/bill/117th-congress/house-bill/6584. 8. "Diversity Action Plans to Improve Enrollment of Participants from Underrepresented Populations in Clinical Studies." fda.gov, 3 July 2024, fda.gov/regulatory-information/search-fda-guidance-documents/diversity-action-plans-improve-enrollment-participants-underrepresented-populations-clinical-studies. 9. Stein, Rob. "NPR Choice Page." Npr.org, 2019, npr.org/sections/health-shots/2019/05/24/725404168/at-2-125-million-new-gene-therapy-is-the-most-expensive-drug-ever. 10. Dowling JJ, Pirovolakis T, Devakandan K, Stosic A, Pidsadny M, Nigro E, et al. AAV gene therapy for hereditary spastic paraplegia type 50: a phase 1 trial in a single patient. Nature Medicine [Internet]. 2024 Jun 28;16. Available from: nature.com/articles/s41591-024-03078-4

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Walking the Talk of Patient-Centricity in Cell and Gene Therapy Trials - PR Web

Creative Biolabs introduces recombinant viral vector development mechanisms and features some essential products. New York, USA - July 17, 2024 - Gene therapy has emerged as a beacon of hope for treating many intractable diseases, although it somehow remains a subject of debate. Central to the success of gene therapy are viral vectors, which play a critical role in delivering therapeutic genes to target cells. Creative Biolabs undertakes viral vector development to deliver validated vector products, thus supporting scientists and pharmaceutical companies in their quest for novel, potent gene therapies.

Image: https://www.getnews.info/uploads/0c59d26f78bf6dd58aafee5bc68d7d8c.jpg

The Impact of Viral Vectors on Gene Therapy

"Recent industry news, released at the beginning of July about adeno-associated viruses (AAVs), has highlighted advancements in gene therapy, emphasizing the critical role of viral vectors in these successes. Viral vectors are engineered viruses used to deliver genetic material into cells, a crucial step in gene therapy. Their design, efficiency, and safety directly impact therapeutic outcomes. With advancements in viral vector technology, the potential to treat genetic disorders, cancers, and other diseases has significantly improved." According to a scientist at Creative Biolabs.

Custom Recombinant Viral Vector Development [https://www.creative-biolabs.com/gene-therapy/viral-vector-design-and-construction.htm]

The design and construction of recombinant viral vectors are critical components of effective gene therapy. Creative Biolabs excels in developing high-efficiency viral vectors that deliver therapeutic DNA accurately and safely. Their expertise encompasses a variety of viral vectors, including adenovirus, adeno-associated virus, lentivirus, and retrovirus.

Recombinant Adeno-Associated Virus

Due to their demonstrated safety and efficacy in both preclinical and clinical settings (over 162 Phase I, II, and III clinical trials, and clinical efficacy has been achieved in at least six human diseases), recombinant adeno-associated viruses [https://www.creative-biolabs.com/gene-therapy/category-recombinant-adeno-associated-virus-304.htm] are favored in gene therapy. Creative Biolabs develops a variety of rAAV serotype vectors, including AAV2, AAV5, AAV8, and AAV9, which have different tissue-tropism and can express transgenes for an extended period of time.

"From initial concept to final toxicity and safety assessments, we offer comprehensive services for the development of sophisticated adeno-associated virus vectors, including design, purification, and titration."

Creative Biolabs' online store lists some popular products and also welcomes requests for AAV customization.

Recombinant Adenovirus

Adenoviruses are powerful tools for gene therapy, particularly for cancer and other applications requiring high transgene expression. Creative Biolabs manages the development of recombinant adenoviruses and can tailor the project according to varying research objectives.

"Our adenoviruses are advantageous in their ability to infect both dividing and non-dividing cells, have a high packaging capacity of up to 8 kb, and have the outstanding safety of not being integrated into the host chromosome." The scientist added.

Recombinant Lentivirus

Recombinant lentivirus [https://www.creative-biolabs.com/gene-therapy/category-recombinant-lentivirus-306.htm] vectors represent a batch of efficient vehicles that can contribute to long-term gene expression and highly effective gene therapy. Creative Biolabs' expertise ensures the generation of lentiviral vectors with high transduction efficiency and stability, which can potentially apply to preclinical gene therapy R&D against vascular diseases, diabetes mellitus, rheumatoid arthritis, cancers, etc.

For more information about Creative Biolabs' viral vector development services and to explore their comprehensive product offerings, visit https://www.creative-biolabs.com/gene-therapy.

About

Creative Biolabs is a leading biotechnology company specializing in gene therapy development, antibody production, and immunotherapy solutions. With a strong focus on innovation and customer service, Creative Biolabs provides a wide range of high-quality products and services to support biomedical research and drug development worldwide. Media Contact Company Name: Creative Biolabs Contact Person: Candy Swift Email: Send Email [http://www.universalpressrelease.com/?pr=how-do-vectors-reign-over-the-efficacy-of-gene-therapy-creative-biolabs-breaks-out-the-facts] Phone: 1-631-830-6441 Country: United States Website: https://www.creative-biolabs.com/gene-therapy

This release was published on openPR.

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How Do Vectors Reign Over the Efficacy of Gene Therapy? Creative Biolabs Breaks Out the Facts - openPR

Ocugen has dosed the first patient with retinitis pigmentosa (RP) in its phase 3liMeliGhTtrial (NCT06388200) of OCU400 gene therapy.1

Patients with RP associated with mutations in multiple genes currently have no therapeutic options. As a retinal surgeon, I am encouraged by the therapeutic potential of OCU400 to provide long-term benefit to RP patients, Lejla Vajzovic, MD, FASRS, Director, Duke Surgical Vitreoretinal Fellowship Program, Associate Professor of Ophthalmology with Tenure Adult and Pediatric Vitreoretinal Surgery and Disease, Duke University Eye Center, and Retina Scientific Advisory Board Chair, Ocugen, said in a statement.1 OCU400 is a novel modifier gene therapy approach that could initiate a paradigm shift in the treatment of RP and to field of ophthalmology.

The phase 3 trial will last 1 year and have a sample size of 150 participants, at least 8 years of age, half with RHO gene mutations and half that have gene agnostic RP. Each arm is randomized 2:1 to receive either 2.5 x 1010 vg/eye of OCU400 or control. The studys primary endpoint is achieving an improvement of at least 2 Lux levels from baseline in the study eyes on Luminance Dependent Navigation Assessment (LDNA). Ocugen announced that it had received investigational new drug application clearance for a phase 3 trial of OCU400 in April 2024.2

The current OCU400 Phase 3 study is very exciting and gives hope for thousands of individuals with RP, Benjamin Bakall, MD, PhD, Director, Clinical Research, Associated Retina Consultants (ARC) and Clinical Assistant Professor, University of Arizona, College of Medicine Phoenix, added.2 I am encouraged that we may have a potential treatment option to preserve or improve the vision in RP patients regardless of gene mutation, and very pleased that the first patient dosing in the Phase 3 liMeliGhT clinical trial was performed at ARC.

READ MORE: Multicharacteristic Opsin Gene Therapy Improves BCVA, MLSDT in Retinitis Pigmentosa

OCU400 has previously been evaluated in a phase 1/2 trial, data from which suggests positive trends in Best-Corrected Visual Acuity (BCVA) and Multi-Luminance Mobility Testing (MLMT), and Low-Luminance Visual Acuity (LLVA) among treated eyes. In this study, most patients (89%; n = 16/18) demonstrated preservation or improvement in the treated eye either on BCVA or LLVA or MLMT scores from baseline, and 80% (n = 8/10) ofpatients with an RHOmutation experienced either preservation or improvement in MLMT scores from baseline. Most (78%; n = 18) also had preservation or improvement in treated eyes in MLMT scores from baseline.

We are grateful for our continued collaboration with Dr. Bakall and the team at ARC, said Huma Qamar, MD, MPH, Chief Medical Officer, Ocugen, added.1 "We are excited to expand our enrollment to include more centers and patients representing a diverse array of RP gene mutations, which will be a validation of this novel gene therapy platform. We will provide updates as our progress continues."

Each clinical milestone achieved by OCU400 brings us closer to providing a one-time treatment for life to patients living with RP, Shankar Musunuri, PhD, MBA, Chairman, CEO and cofounder, Ocugen, added.1 Dosing the first patient is especially significant and makes our dedication to serving RP patients300,000 in the U.S. and Europe and 1.6 million worldwidemore tangible.

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Ocugen Doses First Patient With Retinitis Pigmentosa Gene Therapy in Phase 3 Trial - CGTLive

The development of drugs and gene therapies for rare diseases is costly. It requires significant investments in advanced technology, clinical trials, and regulatory approvals. But what happens when the development of such gene therapies is paused once they are no longer commercially viable?

A few weeks ago, Swiss biopharma Basilea announced it will sell and transfer all rights for the companys glioblastoma candidate lisavanbulin (BAL101553) to the Glioblastoma Foundation for an undisclosed initial purchase cost. The charitable organisation plans to take over lisavanbuilins post-access trial programme, allowing patients from previous clinical studies to continue receiving the drug.

Similarly, in April 2024, Great Ormond Street Hospital (GOSH) in the UK announced that it is attempting to obtain the licence for gene therapy simoladagene autotemcel to treat the rare disease adenosine deaminase-deficient severe combined immunodeficiency (ADA-SCiD) on a non-profit basis. This came after the University College London-spinout Orchard Therapeutics, which planned to bring it to market, dropped out of its development plans. If successful, this will be the first time that an NHS trust has the authorisation to market a drug for this kind of treatment.

ADA-SCiD is a rare genetic disorder marked by a severely deficient immune system due to mutations in the ADA gene, which is crucial for producing the enzyme adenosine deaminase. It causes a severely compromised immune system, leading to recurrent, severe infections, and developmental delays.

Across three clinical trials conducted in the US and UK, 50 patients with ADA-SCiD were treated with simoladagene autotemcel, which was co-developed by labs at UCL and University of California Los Angeles. The overall survival with the gene therapy was 100% in all studies up to 36 months. Event-free survival, defined as no need for enzyme-replacement therapy or rescue allogeneic haematopoietic stem-cell transplantation, reached 97% and 95% at 24 months, respectively, in the two US studies (NCT01852071 and NCT02999984) and 95% in the UK study (NCT01380990).

The gene therapy is currently available to patients in clinical trials, but it is important for these therapies to be approved. Dr. Claire Booth, who leads the ADA-SCiD clinical trial at GOSH, says We want to ensure that these proven treatments can be offered to patients and families quickly and simply, without the need to look at compassionate use approaches.

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One of the main challenges associated with developing gene therapies is funding. It is difficult to get funding from venture capital or private investors due to small patient populations and uncertain returns. Grants can fund early clinical trials, but not the full development and approval process, which requires more funding, explains Dr. Donald Kohn, lead investigator of the ADA-SCiD programme at UCLA, and Distinguished Professor, Microbiology, Immunology and Molecular Genetics.

Another huge challenge is the expensive commercial-grade manufacturing costs. Commercial feasibility plays a big role in pharma companies progressing drug development and this may be challenging in ultra-rare diseases with small patient populations. Advanced therapies like gene therapy are costly and complex to produce.

Simoladagene autotemcel is composed of autologous CD34+ haematopoietic stem and progenitor cells (HSPCs) transduced ex vivo with a self-inactivating lentiviral vector encoding human ADA. Lentiviral vector production represents a large portion of the high costs associated with such therapies.

The cost of reagents needed to make a gene therapy product is high, but we hope that this may come down in the future, says Booth.

One company that is trying to tackle high manufacturing costs is Ori Biotech. The biotech, which is headquartered in London, UK and Woodcliff, New Jersey, has developed an automated platform that streamlines the cell therapy manufacturing process.

[Oris] IRO platform takes the most labour-intensive and time-consuming parts: activation, transduction, expansion, and harvest, and automates them into one integrated system without any manual interventions or tubing, said Ori Biotechs CEO Jason Foster.

In doing so, the platform aims to increase throughput, lower cost, and improve reliability by reducing failure rates. Ori has worked with companies like North Carolina-based Inceptor Bio to scale up manufacturing of their chimeric antigen receptor (CAR)-M, CAR-T and CAR-NK programmes. One of its candidates IB-T101 is set to enter clinical trials in Q4 2024.

In addition to funding challenges and manufacturing costs, there might also be differences based on locations in how gene therapies are made accessible to patients, says Kohn. He explained that national healthcare systems, like those in Europe, may have some advantage in funding rare disease gene therapies compared to the US healthcare system that includes multiple private payers. With a single national payer, developers only need to negotiate reimbursement with one entity rather than many private insurers. Additionally, Kohn said national systems can recognise that an initially high-cost therapy might save money over many years if it treats a fatal or chronic rare disease.

Just as a nonprofit hospital like GOSH is attempting to license EFS-ADA, there are other cases of individuals taking matters into their own hands, via an untraditional route. Terry Pirovolakis started Elpida Therapeutics, a not-for-profit biotech, after his son Michael was diagnosed with a rare disease called spastic paraplegia type 50 (SPG50) at five months old.

After Michael received his diagnosis, Pirovolakis said: We were told, Go home, love him. Theres not much we can do. Hell be paralysed from the waist down by the age of 10, quadriplegic by the age of 20. I just couldnt accept that fate for my child.

Elpida, which is set up as a company but operates as a nonprofit, was initially funded with money from philanthropists. Pirovolakis explained that the model works through partnerships with companies, institutions, and hospitals. When the organisation partners with a hospital, the latter agrees to treat a certain number of patients for free, or at a very low cost. In exchange, Elpida gives the hospital a promissory note.

He explains that funds through incentives from the US Food and Drug Administration (FDA) like priority review vouchers (PRV) for a gene therapy product will be used to pay the hospitals, and to hopefully fund more programmes.

Theyre [the hospital] taking on a mutual risk with us knowing that were trying to do good, not just trying to make money, said Pirovolakis.

Elpida aims to use any money it makes after paying the promissory notes to fund more programmes. Earlier this month, Pirovolakis mentioned that the FDA greenlit Elpidas plans to initiate a Phase III study of its lead candidate Melpida for patients with the rare disorder SPG50. Eight children will be dosed in the trial that is set to begin in August 2024. This is off the back of an ongoing Phase I/II (NCT05518188) trial.

Pirovolakis says there can be a socially acceptable way of dealing with the therapies once theyre deemed commercially unviable. Pharma companies want to help find therapies for rare diseases but also need to consider their best interests. So, when they drop it [a therapy], [giving it] to another company, or back to the university.thats an ethical way of doing things.

When they hold it for five or 10 years, looking for a financial partner to recoup their lost assets, that is not the right way of doing things.

Despite the promise of these alternate models, Pirovolakis delivers a stark reminder: Our model isnt ideal; it is out of necessity.

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Left in limbo: When pharma halts rare disease research - Pharmaceutical Technology

"Gene therapy is the ultimate team sport within science, it's one of the reasons I love it, not only in terms of the stages of development of the product, but also often developing the gene or cell therapy itself. You need to figure out how you're going to deliver it, what's the route of administration? What's the capsid, or delivery vehicle that you're going to use? How do you design the transgene? How do you consider an animal model? That naturally lends itself to bringing together a lot of different expertise.

As gene and cell therapies grow in prevalence, continuing early research in the field is paramount to improving multiple aspects of these novel therapies. A number of researchers from Mass General Brigham (MGB) presented work dealing with new technologies and new methods aiming to improve gene and cell therapy and its delivery to different tissues, particularly the brain, atthe American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting, held May 7 to 10, 2024, in Baltimore, Maryland.

CGTLivespoke with Nathan Yozwiak, PhD, head of research, Cell and Gene Therapy Institute, MGB, to learn more about the research presented at the meeting and other research that MGB is engaged in with cell and gene therapy. He discussed the MGB Gene and Cell Therapy Institute and emphasized its value as a resource for sponsors, investigators, and researchers involved with gene and cell therapies to hopefully speed up the process of developing these therapies. He stressed the importance of collaboration when developing and then assessing gene and cell therapies and how administrating these therapies is a big collaborative lift.

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Nathan Yozwiak, PhD, on Collaboration for Cell and Gene Therapy Development - CGTLive

For a third time, Sarepta Therapeutics has convinced a top Food and Drug Administration official to overrule the prevailing view of their staff and approve a drug for Duchenne muscular dystrophy.

On Thursday evening, the FDA announced it expanded the approval of Elevidys, Sareptas Duchenne gene therapy, to cover nearly all patients, regardless of age or wheelchair status, despite the fact that the drug failed a large, Phase 3 trial last year.

That decision, documents concurrently released by the agency show, was made almost exclusively by Peter Marks, the agencys director of the Center for Biologics Evaluation and Research. He overruled three review teams and two top lieutenants, who wrote that the data Sarepta submitted cast significant uncertainty regarding the benefits of treatment.

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Berlin, Germany, June 21, 2024 Today Bayer AG, together with Charit Universittsmedizin Berlin, presented plans for the construction of the Berlin Center for Gene and Cell Therapies. The project is being substantially financed and supported by Germanys Federal Government as well as the State of Berlin. The aim of the joint project is to bring these groundbreaking technologies to patients more quickly while creating a leading biotech ecosystem for innovative therapies in Berlin.

The potential for cell and gene therapies (CGTs) is considered to be enormous. By targeting diseases at the genetic and cellular level, CGTs could offer options to people for whom conventional therapies have failed or where no effective treatment is currently available.

In order to translate basic research into benefits for patients faster, Charit and Bayer will establish the Berlin Center for Gene and Cell Therapies on the Bayer campus at Berlin Nordhafen. The center will support start-ups bringing their innovative approaches in the area of cell therapies and gene therapies into clinical development. To achieve this, the center will include a so-called incubator with fully equipped laboratory space and a production area certified according to the standards of good manufacturing practice (GMP). Incubators are facilities that accompany start-ups developing their innovative ideas and helping them build viable business models. These early-stage companies will receive advice on regulatory requirements, clinical trials, patent rights and business development. To operate the incubator, Bayer and Charit will establish a joint public-private, non-profit company with limited liability. Charit will own 67 percent of this company, with Bayer owning the remaining 33 percent.

The Berlin Center for Gene and Cell Therapies will bring together research, development and the manufacturing of cell therapies and gene therapies in the heart of Berlin. Purposely embedding it within the Berlin ecosystem, a European capital with a large number of biomedical and healthcare facilities, it is intended to become a creative and interactive hub for biotech innovations in the area of CGT. The project is funded by both the Federal Ministry of Education and Research and the State of Berlin. Construction is planned to begin in 2025.

Olaf Scholz, Federal Chancellor of Germany: "With the kick-off of the new translation center, we are also celebrating a unique form of collaboration between science, industry and politics. This institution will become the core of an entire organism of gene and cell-based therapies. To achieve this, we need scientists and entrepreneurs who see both the smallest details in the cell nucleus as well as the big picture: the medicine of the future that provides answers to the big questions that Rudolf Virchow already posed. Here in Germany we have both: bold research and innovative companies. I wish you every success with this visionary project!"

Bettina Stark-Watzinger, Federal Minister of Education and Research: Gene and cell therapies offer a tremendous opportunity for more targeted and therefore better treatment of patients. Currently, however, the path from the research laboratory to the patient's bedside often still takes too long. With the translation center for gene and cell therapies, we are now taking a big step forward in Germany. Science and industry are coming together in one location to turn ideas into reality and to help translate scientific findings into medical practice more quickly through spin-offs and start-ups. As an essential component of the National Strategy for Gene and Cell Therapies, the Federal Ministry of Education and Research is supporting the establishment of the Center with around

80 million euro. In doing so, we are once again strengthening Germany's position as a leading location for biomedical innovations.

Prof. Dr. Karl Lauterbach, Federal Minister of Health: Targeted gene therapies, personalized cancer vaccines and novel antibody-drug conjugates, together with artificial intelligence, will open a new era in medicine and entirely new prospects for patients. These revolutionary treatment approaches place high demands on laboratories, hospitals and doctors. This joint project of Bayer and Charit is an ideal partnership to learn together and make rapid progress.

Bill Anderson, Chairman of the Board of Management (CEO) of Bayer AG: Despite great advances in research and technology, there are still many diseases that are without cure and which affect the lives of millions. To these people, cell and gene therapies offer great hope. Only through close partnerships across borders, new approaches and quick action can we make real progress towards our objective of curing diseases that were long considered incurable.

Stefan Oelrich, Member of the Board of Management, Bayer AG and President of Bayers Pharmaceuticals Division: The close proximity between research and production within the Berlin Center for Gene and Cell Therapies will be unique in Germany. Together with the Charit we want to help translate scientific knowledge in the area of cell therapies and gene therapies into innovative treatment approaches for patients as quickly as possible. With the Berlin Center for Gene and Cell Therapies, it is our vision to establish a biotech ecosystem, which unites different players, providing international appeal way beyond the city of Berlin.

Prof. Dr. Heyo K. Kroemer, Chief Executive Officer of Charit: Cell therapies and gene therapies represent a major medical advance; they can help where conventional methods reach their limits. As these are highly innovative drugs, their development, however, is much more complex than that of other medicines. If we want to bring these therapies to patients as quickly as possible, we need to take new routes. With the intensified partnership between Charit and Bayer, we want to initiate structural development to bring Berlin to the forefront of this pioneering technology both nationally and internationally. In doing so, we also create and maintain value and thus jobs in the country. This is a big step in Berlin, for Berlin and for Germany.

Astrid Lurati, Chief Financial and Infrastructure Officer of Charit: "As part of this project, the two partners Charit and Bayer are combining their respective expertise to further advance the developments in the field of gene and cell therapy both nationally and internationally. This approach is unique in Germany and demonstrates the innovation power of Berlin and its major healthcare players in their efforts to reimagine the medical care of tomorrow already today. The extensive support, which we are receiving both from the State of Berlin as well as the Federal Government underlines the importance of this project. With the Berlin Center for Gene and Cell Therapies we are breaking new ground together and I would like to thank everyone involved for the excellent cooperation."

Kai Wegner, Governing Mayor of Berlin: Berlin is a strong and leading location for science, research and medicine and therefore for the healthcare industry as a whole. The newly emerging translation center for gene and cell therapies is an excellent example of this. The State of Berlin has supported this lighthouse project from the beginning as we are convinced of the potential that gene and cell therapies provide. The collaboration between Charit and Bayer in one of the most innovative areas of medicine is a clear signal that together we can make Berlin and Germany a pioneer in this field. It is our common goal to help more patients with the most modern therapies, where todays medicine still reaches its limits. In addition, the location provides an excellent environment, which we will continue to strengthen by further developing the Bayer site into a life science campus.

Franziska Giffey, Mayor and Berlin State Senator for Economic Affairs, Energy and Public Enterprises: The close collaboration between companies and the excellent research in the city is a recipe for success to achieve our goal of making Berlin the number one innovation hotspot in Europe. With the Bayer and Charit translation center for gene and cell therapies we are moving a big step closer to reaching this goal. It brings together two internationally renowned medical pioneers and will benefit from its location in Berlin, one of the most successful start-up ecosystems in the world. The new center has enormous economic potential and will attract talent and investment. This is very good news for the growing business location of Berlin.

The Berlin Center for Gene and Cell Therapies is being developed by iQ spaces, a project developer specialized in laboratory real estate, on the Bayer campus at Berlin Nordhafen. Across 18,000 sqm, the ten-story building is divided into an incubator with fully equipped laboratory and office space to accommodate 15 to 20 start-ups in various stages of development, as well as a GMP-certified manufacturing facility for the development of cell and gene therapies up to clinical phase II. The building was designed by the architectural firm HENN.

About cell therapies and gene therapies Cell therapies and gene therapies (Advanced Therapy Medicinal Products, ATMPs) are among the most important innovations in the healthcare sector. They have the potential to fundamentally change the treatment of cancer, autoimmune diseases, neurodegenerative diseases and many rare genetic diseases. The novel therapies are based on genes, tissues or cells and therefore often contain living components. These products, which are therefore also referred to as living drugs, can be better tailored to individual patients than traditional medicines and are particularly suitable for the treatment of diseases that were previously untreatable or difficult to treat. Although several hundred clinical studies for the development of cell therapies and gene therapies are currently ongoing, only a small number of such products is currently approved in Europe. The goal of Bayer and Charit is for the Berlin Center for Gene and Cell Therapies to bridge this translation gap.

About Charit Universittsmedizin Berlin With more than 100 departments and institutes across four campuses and 3,293 beds, Charit Universittsmedizin Berlin is one of Europes largest university medical centers. At Charit, the areas of research, teaching, and medical and patient care are closely interconnected. Averaging about 20,000 employees Charit-wide and some 23,500 across the entire group of companies, Berlins university medicine organization remained one of the capital citys largest employers in 2023. Charit is a leader in diagnosis and treatment of particularly severe, complex, and rare diseases and health conditions. A medical school and university medical center in one, Charit enjoys an outstanding reputation worldwide, combining first-class patient care with excellence in research and innovation, state-of-the-art teaching, and high-quality training and education. Everything Charit does revolves around people and their health. Charit pursues translational research in which scientific findings are applied to prevention, diagnostics, and treatment and clinical observations inform new approaches in research in turn. At Charit, the goal is to actively help shape the medicine of the future to benefit patients. https://www.charite.de/en/

About Bayer Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. In line with its mission, Health for all, Hunger for none, the companys products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2023, the Group employed around 100,000 people and had sales of 47.6 billion euros. R&D expenses before special items amounted to 5.8 billion euros. For more information, go to http://www.bayer.com.

Find more information at https://pharma.bayer.com/ Follow us on Facebook: http://www.facebook.com/bayer

Forward-Looking Statements This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayers public reports which are available on the Bayer website at http://www.bayer.com. The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments.

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Berlin Center for Gene and Cell Therapies kicked off in Berlin - The Palm Bayer

Visiongain has published a new report entitled Cell & Gene Therapy Cold Chain Logistics Market Report 2024-2034: Forecasts by Component (Cryogenic Shippers, Cryogenic Storage Freezers, Ultra-Low Freezers, Cold Chain Management Systems, Shipment and Storage Medium, Cryogenic Packout Kits, Others), by Services (Transportation, Storage, Packaging), by Mode of Transport (Air Transport, Ground Transport, Water Transport), by Holding Temperature Range (Cryogenic, Refrigerated, Ambient, Others), by End-users (Biopharmaceutical and Biotechnology Companies, Academic & Research Institutes, Others) AND Regional and Leading National Market Analysis PLUS Analysis of Leading Companies.

The global cell and gene therapy cold chain logistics market is estimated at US$1,877.3 million in 2024 and is projected to grow at a CAGR of 15.5% during the forecast period 2024-2034.

Escalating Adoption of Cell Therapies and Gene Therapies is Driving the Demand for Cold Chain Logistics

Recent advancements in cell and gene therapy have led to the approval of numerous novel treatments for conditions such as blood disorders and cancer. On March 14, 2024, the FDA granted approval to Bristol Myers Squibb's Breyanzi, marking it as the inaugural CAR T cell therapy for the treatment of relapsed or refractory chronic lymphocytic leukaemia (CLL) and small lymphocytic leukaemia. Additionally, on February 17, 2024, the FDA granted accelerated approval to Iovance Biotherapeutics' Amtagvi, a one-time cell therapy for unresectable or metastatic melanoma, with ongoing studies to confirm its efficacy. As the adoption of these therapies increases, the demand for proper storage, distribution, and logistics of cell and gene therapy products is expected to rise. Transporting these therapies to various countries requires a highly controlled and regulated process to ensure their safety and efficacy, which will significantly boost the cold chain logistics market.

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Visiongains 359-page report provides 146 tables and 198 charts/graphs. Our new study is suitable for anyone requiring commercial, in-depth analyses for the cell and gene therapy cold chain logistics market, along with detailed segment analysis in the market. Our new study will help you evaluate the overall global and regional market for Cell and Gene Therapy Cold Chain Logistics. Get financial analysis of the overall market and different segments including type, process, upstream, downstream, and company size and capture higher market share. We believe that there are strong opportunities in this fast-growing cell and gene therapy cold chain logistics market. See how to use the existing and upcoming opportunities in this market to gain revenue benefits in the near future. Moreover, the report will help you to improve your strategic decision-making, allowing you to frame growth strategies, reinforce the analysis of other market players, and maximise the productivity of the company.

What are the Current Market Drivers?

Growth in Cancer and Genetic Disorders

Genetic disorders, stemming from abnormalities in genetic makeup and chromosomes, present a range of physical and mental health challenges globally. Disorders like alpha-1 antitrypsin deficiency, cystic fibrosis, beta thalassemia, haemophilia, and sickle cell disease have gained prominence due to factors including heightened awareness, advancements in diagnostic technology, and environmental and lifestyle changes. Low-income nations, in particular, face challenges such as inadequate diets, exposure to environmental toxins, and unhealthy lifestyles, increasing the risk of genetic abnormalities.

The WHO estimates that 5% of the global population carries sickle-cell disease, hemoglobin disorders, or thalassaemia, with over 300,000 babies born annually with severe hemoglobin disorders. Thalassemia, prevalent in regions like the Mediterranean, Middle East, Southeast Asia, and Africa, affects approximately 4.4 out of every 10,000 live births globally, with up to 40% of Southeast Asia's population being genetic carriers. These factors are poised to drive demand for cell and gene therapies in these regions, further emphasizing the necessity for proper transportation and storage of these biotherapeutics.

Increasing Strategic Initiatives Among Market Players Are Shaping the Industry's Future

The intricate processes involved in manufacturing, storing, and delivering cell and gene therapies demand a high level of expertise, specialized infrastructure, and adherence to stringent regulatory standards for global distribution. Major market players, spanning manufacturers to specialized logistics companies, are increasingly pursuing strategic initiatives such as launching new services and products, forging collaborations, and forming agreements to ensure the safe and efficient storage and transportation of these temperature-sensitive biologics. Regulatory bodies are also actively involved, offering crucial support to ensure compliance with international regulations and guidelines for therapeutic transportation. Significant initiatives include AmerisourceBergen's World Courier announcing the implementation of a real-time location monitoring solution across its multi-use packages in April 2023, aimed at enhancing shipment visibility during transit on a global scale.

Get Detailed ToC https://www.visiongain.com/report/cell-gene-therapy-market-2024/

Where are the Market Opportunities?

Stringent Government Regulations for Cell and Gene Therapy Transportation Are Expected to Boost the Market Growth

Stringent government regulations for cell and gene therapy transportation and storage present significant growth opportunities for market players by necessitating strict compliance measures. Companies must adhere to meticulous documentation standards, such as detailed records of temperature monitoring and handling procedures. For example, logistics providers like DHL and UPS offer specialized software to streamline these processes, ensuring regulatory compliance. Validation of cold chain processes is critical, involving extensive studies and costs to confirm the efficacy of temperature-controlled packaging, as seen with services from companies like Pelican BioThermal. Quality control measures, including audits and real-time monitoring, are essential for maintaining product integrity, with firms like Cryoport exemplifying strict adherence to these standards, thereby ensuring the safe and effective delivery of biologic products.

Surge in Cell and Gene Therapies Pipeline

The cell and gene therapy landscape is witnessing a surge in clinical pipeline strength and product approvals, propelled by technological advancements and substantial investments in research and development. This momentum is driving a significant increase in clinical studies focused on cell and gene therapeutics across various medical indications, extending beyond genetic disorders. Major biopharmaceutical companies are actively exploring the potential of these therapies for a diverse range of conditions.

Recent clinical findings have highlighted promising outcomes, particularly in areas like autoimmune disorders, where therapies such as regulatory T cells and mesenchymal stem cells have shown potential as future drug candidates, with approximately 1000 cell and gene therapies currently in the product development pipeline. For instance, on 4 January 2024, AbbVie and Umoja Biopharma announced two exclusive agreements to develop CAR-T cell therapies using Umoja's VivoVec platform. In future approval of these temperature sensitive biotherapeutics is estimated to increase necessity for temperature controlled and specific storage and transportation of these biologics to patients and healthcare providers in various parts of the globe.

Competitive Landscape

The market is highly fragmented with a lot of companies and products available for cold chain logistics of cell and gene therapies. The major players in the market are Cencora, Inc., Cardinal Health, Catalent Inc., Thermo Fisher Scientific Inc., Marken (a UPS Company), A.P. Moller Maersk, CryoPort Inc., DHL, FedEx, Arvato SE, NMDP BioTherapies, BioLife Solutions Inc., BioStor Systems Inc., Yourway, Atelerix Ltd., Nordic Cold Chain Solutions, MasterControl Solutions, Inc., TrakCel, Modality Solutions, and sedApta s.r.l. Companies focusing on the cell and gene therapy cold chain market utilize specialized solutions, integrated logistics, and regulatory compliance expertise. Modality Solutions and CryoPort Inc. offer customized cold chain processes, whereas DHL, FedEx, and Marken provide temperature-controlled global logistics services. Cardinal Health and Modality Solutions excel in regulatory compliance, assisting clients in navigating industry regulations to ensure the safe transportation of therapies.

Recent Developments

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About Visiongain

Visiongain is one of the fastest-growing and most innovative independent market intelligence providers around, the company publishes hundreds of market research reports which it adds to its extensive portfolio each year. These reports offer in-depth analysis across 18 industries worldwide. The reports, which cover 10-year forecasts, are hundreds of pages long, with in-depth market analysis and valuable competitive intelligence data. Visiongain works across a range of vertical markets with a lot of synergies. These markets include automotive, aviation, chemicals, cyber, defence, energy, food & drink, materials, packaging, pharmaceutical and utilities sectors. Our customised and syndicatedmarket research reportsoffer a bespoke piece of market intelligence customised to your very own business needs.

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Cell and Gene Therapy Cold Chain Logistics market is projected to grow at a CAGR of 15.5% by 2034: Visiongain - GlobeNewswire

The CIFFREO trial (NCT04281485) of fordadistrogene movaparvovec, an investigational mini-dystrophin gene therapy for Duchenne muscular dystrophy (DMD) developed by Pfizer, did not meet its primary end point of motor function improvement vs placebo among boys aged 4 to 7 years, the company announced in a press release.1

Improvement in motor function among ambulatory patients, determined by change in North Star Ambulatory Assessment at 1 year post treatment, was the main end point in the study. There were also no significant improvements in key secondary end points, including 10-meter run or walk speed and time to rise from floor.

While the question of gene therapys long-term efficacy remains, it is a promising route of therapy for patients with DMD | Image credit: luchschenF - stock.adobe.com

We are extremely disappointed that these results did not demonstrate the relative improvement in motor function that we had hoped, Dan Levy, MD, PhD, development head for Duchenne muscular dystrophy at Pfizer, said in a statement. We plan to share more detailed results from the study at upcoming medical and patient advocacy meetings, with the goal of ensuring that learnings from this trial can help improve future clinical research and development of treatment options that can improve care for boys living with Duchenne muscular dystrophy. We are grateful for the boys, their families, advocates, and the investigators who have participated in this research and the continuing effort to advance treatment options for this debilitating disease.

While the overall safety profile of the treatment was largely manageable in the CIFFREO trial, the study has paused dosing in its crossover portion due to a fatal serious adverse event in the phase 2 DAYLIGHT trial (NCT05429372), a multicenter, single-arm study evaluating safety and dystrophin expression after treatment with fordadistrogene movaparvovec in boys aged 2 to less than 4 years.2 In a community letter, Pfizer announced the loss of the patient but was still working to understand the its cause at the time.3

In the CIFFREO trial, adverse events were generally mild to moderate, and serious treatment-related adverse events generally responded to clinical management.1

DMD, the most common form of muscular dystrophy in children, is characterized by muscle breakdown over time due to patients without a functional dystrophin gene.4 Patients with DMD produce little to no dystrophin, which is a crucial protein for muscle strength.

Gene therapy is a promising area of research for patients with DMD, with the goal being to stabilize the disease by inducing production of a shortened but functional version of dystrophin. Lost muscle cannot be brought back by gene therapy, so it is not a curative therapy, but gene therapies have demonstrated promise for symptom mitigation and even strength improvement among some children.

The first gene therapy for DMD was FDA approved in June 2023 for patients aged 4 through 5 years.5 Delandistrogene moxeparvovec-rokl (Elevidys) was approved through the FDAs accelerated approval pathway and remains the only approved gene therapy for this patient population. The treatment was given the green light based on data from a randomized trial establishing increased expression of micro-dystrophin protein in treated patients.

While the question of gene therapys long-term efficacy remains, it is a promising route of therapy for patients with DMD.4 Alternative approaches to delivery are also being studied, with an aim of better targeting the muscles that need dystrophin or producing versions of dystrophin that are more functional compared with current gene therapies.

References

1. Pfizer provides update on phase 3 study of investigational gene therapy for ambulatory boys with Duchenne muscular dystrophy. News release. Pfizer. June 12, 2024. Accessed June 19, 2024. https://www.pfizer.com/news/press-release/press-release-detail/pfizer-provides-update-phase-3-study-investigational-gene

2. Study of fordadistrogene movaparvovec in early stage Duchenne muscular dystrophy. ClinicalTrials.gov. Updated April 4, 2024. Accessed June 19, 2024. https://www.clinicaltrials.gov/study/NCT05429372

3. Update on Pfizers phase 2 gene therapy trial for Duchenne. Parent Project Muscular Dystrophy. May 7, 2024. Accessed June 19, 2024. https://www.parentprojectmd.org/update-on-pfizers-phase-2-gene-therapy-trial-for-duchenne/

4. Gene therapy for Duchenne muscular dystrophy. Childrens Hospital of Philadelphia. Accessed June 19, 2024. https://www.chop.edu/gene-therapy-duchenne-muscular-dystrophy

5. FDA approves first gene therapy for treatment of certain patients with Duchenne muscular dystrophy. News release. FDA. June 22, 2023. Accessed June 19, 2024. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treatment-certain-patients-duchenne-muscular-dystrophy

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Phase 3 CIFFREO Trial of DMD Gene Therapy Misses Primary End Point - AJMC.com Managed Markets Network

(CNN) The US Food and Drug Administration has given the green light for the first gene therapy that treats a rare form of muscular dystrophy to be used in most people who have the disease and a certain genetic mutation.

Last year, the drug Elevidys, from the biotech company Sarepta Therapeutics was approved to treat only children ages 4 and 5 with Duchenne muscular dystrophy, one of the most severe forms of inherited muscular dystrophies, who have a confirmed mutation in a gene called DMD that is associated with muscle strength.

The FDA announced Thursday that it had given traditional approval for Elevidys for ambulatory people 4 and older with a confirmed mutation in the DMD gene and accelerated approval for non-ambulatory people 4 and older with this mutation. Theres not enough data on safety to support its use in children under 4, the agency says.

Elevidys, given as a one-time intravenous infusion, costs about $3.2 million per patient, making it among the most expensive drugs in the world. Although eye-popping, such aprice tagisnt out of step with other one-time gene therapies, which have topped$3 million to 4 millionper patient in recent years.

Elevidys was previously approved under the FDAs accelerated approval pathway, which clears medicines for diseases where theyre urgently needed based on data suggesting that theyre likely to confer clinical benefits. The drug has been closely monitored since that approval, and in October, Sarepta Therapeutics released results from a confirmatory trial showing that the therapy missed its primary goal a measure of how well kids can move but was successful on a number of secondary measures.

The approval addressed an urgent unmet medical need and is an important advancement in the treatment of Duchenne muscular dystrophy, a devastating condition with limited treatment options, that leads to a progressive deterioration of an individuals health over time, Dr. Peter Marks, director of the FDAs Center for Biologics Evaluation and Research, said in a news release at the time.

It was the first time a therapy of this nature a one-time treatment that delivers a working copy of a gene to make up for one that leads to disease had been cleared under the accelerated approval framework. The move came after emotionaltestimonialsfrom families at an FDA advisory committee meeting.

Duchenne muscular dystrophy causes progressive muscle weakness that can rob children of their ability to walk by the time theyre teenagers, and many dont live well into their 30s. It primarily affects boys because of the way its inherited, affecting an estimated 1 in 3,300 boys.

The Muscular Dystrophy Association trusts the decision of the FDA, which weighs the risks and benefits of the drug, said Dr. Sharon Hesterlee, chief researcher at the association.

Ultimately, what we want is whats best for our patient community and thats balancing that risk-benefit ratio appropriately, she said.

Potential risks of Elevidys include increases in certain liver enzyme levels and acute serious liver injury. The most common side effects of the drug include vomiting, nausea, increased liver function tests and fever.

Yet a major benefit is that the gene therapy provides another option for people with Duchenne muscular dystrophy, and its administered just once.

There is no cure for Duchenne muscular dystrophy, and outside of Elevidys, treatments are limited. Otherapproachesmay include steroid medications,certaindrugs that change how the muscle cells read the mutated gene, physical therapy or surgery to correct spinal curvature, Hesterlee said.

Right now, themainstandard of care for Duchenne is corticosteroids, like prednisone,although there are some newer drugs available. These kids arestilloften on chronic doses ofsteroidsfor many, many years, she said, adding that the side effects of corticosteroids such as weight gain, behavioral issues and increased risk of bone breakage are not ideal.

Duchenne muscular dystrophy can be difficult to treat, she said, and having more treatment options that are proven to be effective remains important.

Muscle makes up a significant amount of your body mass. So when you have a disease like this, its really impacting a lot of tissue. So anything you do, youre looking at trying to bring back or stop a disease process thats really throughout the body, and its a disease thats progressive, so you lose more and more muscle over time, Hesterlee said.

Thats made it pretty challenging, but weve certainly learned a lot, she said. You cant overlook the fact that these boys are living so much longer and doing so much better. Even 20 years ago, they were dying in their teens, and many of them are now living into their 30s. Theyre going to college; they have girlfriends; some of them have gotten married. These are things that werent happening years ago. So weve made a tremendous amount of progress.

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FDA expands approval of first gene therapy for rare form of muscular dystrophy - KDRV

This is a developing story and will be updated with new information as it becomes available.

The FDA has approved Sarepta Therapeutics delandistrogene moxeparvovec-rokl (marketed as Elevidys), an adeno-associated virus (AAV) vector-based gene therapy for patients with Duchenne muscular dystrophy (DMD), for an expanded indication in the disease.1 The therapy is now approved for ambulatory patients (via traditional approval) and nonambulatory patients (via accelerated approval) with a confirmed mutation in the DMD gene who are 4 years of age or older and who do not have any deletion in exon 8 or exon 9 in the gene.

Representing many years of dedicated research, development, investment and creative energy, the expansion of the Elevidys label to treat Duchenne patients aged 4 and above, regardless of ambulatory status, is a defining moment for the Duchenne community," "Today also stands as a watershed occasion for the promise of gene therapy and a win for science, Doug Ingram, JD, the president and chief executive officer of Sarepta, said in a statement.1 At this pivotal moment, I want to give warm thanks to Drs. Jerry Mendell and Louise Rodino-Klapac for their dogged, 20-year pursuit of a gene therapy to treat this ruthless and life-robbing disease, to the FDA for following the scientific evidence to speed delivery of a therapy for a life-threatening rare disease to waiting patients, and to the many clinical investigators and courageous Duchenne families who have participated in the multiple studies that led to this important day.

Elevidys was originally granted FDA approval under an accelerated approval pathway for a more limited indication on June 22, 2023.2 That original decision limited the therapy's use to ambulatory patients aged 4 through 5 years withDMD and a confirmed mutation in theDMDgene, excluding patients with any deletion in exon 8 and/or exon 9. Today's decision, which was based on the agency's confirmation of functional benefits, additionally converts the accelerated approval to a traditional approval for patients who are ambulatory. Although it also expands eligibility to nonambulatory patients, the therapy remains under an accelerated approval for patients who are nonambulatory.1 Sarepta noted that ongoing approval for patients who are nonambulatory may depend on the results of ENVISION (Study SRP-9001-303), a phase 3 confirmatory clinical trial evaluating the gene therapy in patients with DMD who are nonambulatory or older and ambulatory.

Todays expansion of the Elevidys label represents the culmination of my 50-year pursuit of a treatment for Duchenne patients and, along with my colleague Dr. Louise Rodino-Klapac, a nearly 20-year effort to optimize and develop a gene therapy that could be safely and effectively delivered to muscle, Jerry Mendell, MD, the coinventor of Elevidys and the senior advisor for Medical Affairs at Sarepta added to the statement.1 The initial approval of Elevidys was a significant milestone, and the expanded indication means clinicians now have a treatment option for the great majority of boys and young men living with Duchenne. This expansion speaks to the success of the science, the evidence, and the improvements in the trajectory of the disease we have seen to date across studies.

Elevidy's original approval in 2023 was based upon data from the phase 1/2 SRP-9001-101 (NCT03375164) study, the phase 2 SRP-9001-102 study, and the phase 1 ENDEAVOR study (SRP-9001-103; NCT04626674).2 Changes in expression of microdystrophin, a surrogate end point, informed the approval. Furthermore, in the 20-patient cohort 1 of ENDEAVOR, announced in July 2022, findings showed that SRP-9001-treated patients improved 4 points from their pre-therapy baselines on the North Star Ambulatory Assessment (NSAA) compared with a propensity-weighted external control group (P < .0001) over 1 year.3 These patients demonstrated a 3.8-point (unadjusted means) and 3.2-point (least squared means) improvement that diverged from the natural history of DMD over time.

More recently, results from EMBARK, another phase 3 clinical trial (NCT05096221) evaluating Elevidys in DMD, were presented atthe American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting, held May 7 to 10, 2024, in Baltimore, Maryland, by Damon Asher, MS, PhD, senior director of global medical affairs at Sarepta Therapeutics.4 Notably, EMBARK failed its primary end point. However, key secondary endpoints of change in time to rise (TTR) and time to walk/run 10 meters (10MWR) had small but significant differences from placebo, with a least square mean (LSM) difference of -.64 seconds (standard error [SE], 0.21; P = .0025) on TTR and an LSM of -0.42 seconds (SE, 0.15; P = .0048) on 10MWR.5 A composite, prespecified global statistical test including NSAA, TTR, 10MWR, Stride Velocity 95th Centile, 100MWR, and ascend 4 steps test was statistically significant compared with placebo (P = .0044).4

What's really going to tell the story is as these patients are followed over longer periods of time," Asher told CGTLive during the conference. "Theres a lot of challenges in showing efficacy, even with something that may be fairly efficacious, over such a short period of time."

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FDA Approves Sarepta's DMD Gene Therapy Elevidys for Expanded Indication - CGTLive

Roche has announced a research collaboration and licensing agreement with biotech firm Ascidian Therapeutics to develop gene therapies for neurological diseases.

According to the terms of their agreement, Roche will pay Ascidian $42 million upfront for exclusive, target-specific rights to use Ascidian's RNA exon-editing technology to develop therapeutics for undisclosed neurological diseases, the firms said in a statement. Furthermore, Roche will pay Ascidian up to $1.8 billion in total as research, clinical, and commercial milestones are reached. Ascidian is also eligible to receive royalties on commercial sales worldwide for any therapies that are developed under the collaboration.

Under their arrangement, Ascidian will be responsible for conducting discovery and certain preclinical activities in collaboration with Roche, and Roche will be responsible for certain other preclinical activities, as well as further clinical development, manufacturing, and commercialization, Ascidian said. While Roche will have exclusive rights to the technology for the targeted diseases, Ascidian can pursue other disease targets outside of its agreement with Roche.

Boston-based Ascidian's exon-editing technology is designed to correct the RNA produced by damaged exons, the regions of DNA containing the blueprints to make proteins. Correcting the RNA produced by damaged exons greatly mitigates the risks associated with direct DNA editing and gene replacement, such as off-target edits, Ascidian said.

Original post:
Roche, Ascidian to develop gene therapies for neurological diseases - LabPulse

Market Overview and Report Coverage

Lentiviral vectors are a type of viral vector used in gene therapy to deliver genetic material into target cells. They are derived from lentiviruses, which are a type of retrovirus known for their ability to integrate their genetic material into the host cell's genome. This integration allows for stable and long-lasting gene expression, making lentiviral vectors an attractive option for gene therapy applications.

The Lentiviral Vector In Gene Therapy Market is expected to grow at a CAGR of 12.30% during the forecasted period. The current outlook for this market is promising, with increasing research and development activities in the field of gene therapy driving the demand for lentiviral vectors. Advances in genetic engineering and gene editing technologies have also expanded the potential applications of lentiviral vectors in treating a wide range of genetic disorders, cancers, and other diseases.

Moreover, the growing globalization of the pharmaceutical and biotechnology industries, along with increasing partnerships and collaborations between companies and research institutions, are further fueling the growth of the Lentiviral Vector In Gene Therapy Market. With ongoing technological advancements and a supportive regulatory environment, the future of lentiviral vectors in gene therapy looks bright, offering hope for patients with currently incurable genetic diseases.

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Market Segmentation 2024 to 2031

The Lentiviral Vector In Gene Therapy Market Analysis by types is segmented into:

Retrovirus (RV) Adenovirus (AdV) Adeno-associated Virus (AAV)

Lentiviral vectors are commonly used in gene therapy due to their ability to efficiently deliver genetic material into target cells. Retrovirus (RV) vectors are derived from retroviruses and integrate their genetic material into the host cell genome. Adenovirus (AdV) vectors are based on adenoviruses and provide high levels of gene expression in target cells. Adeno-associated Virus (AAV) vectors are derived from adeno-associated viruses and are known for their safety and ability to infect a wide range of cell types. These vectors play a crucial role in the gene therapy market for various applications.

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The Lentiviral Vector In Gene Therapy Market Industry Research by Application is segmented into:

Hospital Clinic Research Institution Others

The Lentiviral Vector in Gene Therapy market application is utilized in various sectors including hospitals, clinics, research institutions, and other medical facilities. This technology is being increasingly used in gene therapy to treat genetic disorders and other diseases. Hospitals and clinics are incorporating Lentiviral Vector technology to provide advanced treatment options to their patients. Research institutions are utilizing these vectors in their studies to explore potential therapeutic applications. Other markets also benefit from Lentiviral Vectors for various research and medical purposes.

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In terms of Region, the Lentiviral Vector In Gene Therapy Market Players available by Region are:

North America: United States Canada

Europe: Germany France U.K. Italy Russia

Asia-Pacific: China Japan South Korea India Australia China Taiwan Indonesia Thailand Malaysia

Latin America: Mexico Brazil Argentina Korea Colombia

Middle East & Africa: Turkey Saudi Arabia UAE Korea

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What are the Emerging Trends in the Global Lentiviral Vector In Gene Therapy market?

- CRISPR/Cas9 technology: Lentiviral vectors are being used in conjunction with CRISPR/Cas9 gene editing technology to more precisely target and edit genes for therapeutic purposes.

- Cell-specific targeting: Researchers are developing lentiviral vectors that can target specific cell types, improving the efficiency and safety of gene therapy treatments.

- Non-viral gene delivery systems: Emerging non-viral gene delivery systems are being developed as alternatives to lentiviral vectors, potentially disrupting the market.

- Personalized medicine: Increased focus on personalized medicine is driving the development of lentiviral vectors tailored to individual patient needs.

- Regulatory advancements: Regulatory agencies are working to streamline the approval process for gene therapy products, spurring growth in the lentiviral vector market. These trends collectively indicate a positive growth trajectory for the Lentiviral Vector In Gene Therapy market as it continues to evolve and innovate.

Lentiviral Vector In Gene Therapy Market Competitive Analysis

One of the key players in the competitive Lentiviral Vector in Gene Therapy market is Thermo Fisher Scientific. Thermo Fisher Scientific is a global biotechnology company that offers a wide range of products and services for the life sciences industry. The company has a strong presence in the gene therapy market, offering high-quality Lentiviral vectors for gene delivery. Thermo Fisher Scientific has a proven track record of innovation and expertise in the development of gene therapy products.

Another major player in the market is Vigene Biosciences, a leading provider of viral vector products and services for gene therapy applications. Vigene Biosciences has a strong focus on quality and innovation, which has helped the company to establish itself as a key player in the gene therapy market. Vigene Biosciences has experienced significant growth in recent years, driven by the increasing demand for Lentiviral vectors in gene therapy research and applications.

OriGene Technologies is also a prominent player in the Lentiviral Vector in Gene Therapy market, offering a wide range of Lentiviral vectors and other gene therapy products. OriGene Technologies has a strong presence in the market, backed by its extensive experience and expertise in gene therapy research and development. The company has witnessed substantial market growth and has been able to capture a significant market share in the gene therapy market.

In terms of sales revenue, companies like Thermo Fisher Scientific and Vigene Biosciences have reported strong financial performance in recent years, reflecting the growing demand for Lentiviral vectors in gene therapy applications. These companies have continued to invest in research and development to expand their product offerings and stay ahead of the competition in the gene therapy market.

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Lentiviral Vector In Gene Therapy Market Forecasts, Market Trends and Impact Analysis (2024 - 2031) - openPR

The risk of secondary cancer after CAR-T therapy, pioneered at Penn, is less than regulators feared last year, an FDA official said Friday at cell and gene therapy conference in King of Prussia.

The FDA had announced in November that it was studying a handful of cases where patients developed lymphoma after being treated with chimeric antigen receptor T cell, CAR-T, therapies, hailed as a possible cure for some forms of blood cancer.

In January, the agency ordered the companies to put black box warnings on the six products that were part of the review.

We were pretty concerned about this when we first saw it last year, Peter Marks, who heads the FDAs Center for Biologics Evaluation and Research, said at the conference sponsored by the Sino-American Pharmaceutical Professionals Association Greater Philadelphia.

Most look to be secondary cancers affecting the T cells of patients who already have related cancers, he said.

Luckily, the majority of these, it looks like are just secondary T cell malignancies that are occurring in people who have T cell malignancies. Thats a known phenomenon, he said.

In a few cases, however, there are signs that patients developed secondary cancer in the type of white blood cells that were genetically modified as part of their treatment, Marks said. The risk is probably on the order of 1 in 10,000 people treated, he said.

That risk is orders of magnitude lower than the risk of malignancies from forms of chemotherapy that are given after cancer hasnt responded to standard treatments, Marks said during his opening keynote address at the conference, now in its third year.

About 500 people were registered for the 2024 @Philly Cell And Gene Therapy Annual Conference, a spokesperson for the conference organizers said.

READ MORE: Emily Whitehead was the first child cured of cancer with therapy from Penn. Shes back as a freshman.

Original post:
Philadelphia Cell and Gene Therapy Conference hosts FDA officials - The Philadelphia Inquirer

Richard DiGeorge and Jo Kaur, pictured with their son Riaan DiGeorge, founded the nonprofit Riaan Research Initiative to support UMass Chan scientists quest to cure Cockayne syndrome

UMass Chan Medical School has received $2.2 million from a nonprofit patient-advocacy organization to contract with Ohio-based Andelyn Biosciences, a contract development and manufacturing organization with extensive experience in gene therapy, to manufacture clinical grade AAV9-CSA vector to treat Cockayne syndrome.

The gift from Riaan Research Initiative brings the start of clinical trials for the fatal autosomal recessive disorder one step closer.

Riaan Research Initiatives historic contribution is the largest known donation ever made toward the development of a treatment for Cockayne syndrome, according to Riaan Research Initiative.

Cockayne syndrome primarily causes mutations in genes CSA (ERCC8) and CSB (ERCC6) and leads to a failure in DNA transcription and repair processes. Impacted children often present with significant growth failure, microcephaly, vision and hearing problems, and global developmental delays. Children with the most severe form of Cockayne syndrome have a life expectancy of five to seven years. There are no approved treatments.

Were thrilled to move on to the next step of this years-long partnership with UMass Chan and are eternally grateful to our donors who have opened the doors to a better world, said Jo Kaur, founder of Riaan Research Initiative and mother of Riaan, a 4-year-old boy diagnosed with Cockayne syndrome and the inspiration behind the research. We have strong evidence of the treatments success in the preclinical phase and look forward to our collaborators manufacturing a potentially life-saving drug that can actually be given to our children.

UMass Chan and Riaan Research Initiative launched this collaboration in 2021 when the organization funded the preclinical phase of this groundbreaking program. Now that research led by Miguel Sena-Esteves, PhD, associate professor of neurology and director of the Translational Institute for Molecular Therapeutics, and Rita Batista, PhD, instructor in neurology, has demonstrated the AAV9-CSA vector efficacy in an animal model, the team is ready to move forward with toxicology studies and clinical manufacturing.

Andelyn Biosciences has been selected to manufacture the plasmids and use its AAV Curator Suspension Platform to industrialize the process, performing analytical development and process optimization, followed by toxicology manufacturing and GMP (good manufacturing practice) production.

Earlier this year, the UMass Chan research team and Riaan Research Initiative received favorable feedback from the U.S. Food and Drug Administration on their pre-Investigational New Drug submission with plans for toxicology studies and manufacturing.

Riaan Research Initiative has been an amazing partner in our journey to develop an AAV9 gene therapy for Cockayne syndrome, Dr. Sena-Esteves said. Our dream of making a difference for Cockayne patients is coming closer every day, and signing the contract with Andelyn Biosciences to make the clinical material is a major step in that direction. Developing a gene therapy for fatal pediatric neurological diseases is challenging in many ways, but together with Riaan Research Initiative we have an outstanding team to bring our program to a first-in-human gene therapy clinical trial for Cockayne syndrome.

Original post:
UMass Chan receives $2.2 million to fund gene therapy for Cockayne syndrome - UMass Medical School

A Pfizer gene therapy for the rare muscle-wasting disease Duchenne muscular dystrophy did not help patients ability to walk or stand up in a pivotal clinical trial. The pharmaceutical giant is still weighing its next steps for the therapy, but analysts say these disappointing trial results, the latest in a series of setbacks, likely mark the end for this program.

The Phase 3 test enrolled boys ages 4 to 7 who still had the ability to walk. The trials main goal was to show an improvement in motor function compared to a placebo. Without providing specific figures, Pfizer said after Wednesdays market close that its gene therapy did not achieve this goal measured one year after treatment. Secondary goals include measuring how fast patients can run or walk for 10 meters and how quickly they can rise from the floor. On these measures, there was no significant difference between the gene therapy arm and the placebo group.

We are extremely disappointed that these results did not demonstrate the relative improvement in motor function that we had hoped, Dan Levy, Pfizers development head for Duchenne muscular dystrophy, said in a prepared statement. We plan to share more detailed results from the study at upcoming medical and patient advocacy meetings, with the goal of ensuring that learnings from this trial can help improve future clinical research and development of treatment options that can improve care for boys living with Duchenne muscular dystrophy.

Duchenne is an inherited disorder that results in the inability to produce normal versions of dystrophin, a protein key to muscle function. Patients develop progressively worsening muscle weakness that robs them of their ability to walk. The muscle weakness eventually affects the lungs and the heart, becoming fatal.

[Paragraph updated to correct the study on dosing pause.] The Pfizer gene therapy, fordadistrogene movaparvovec, uses an engineered virus to deliver to muscle cells a mini-version of the gene that codes for dystrophin. Its clinical development path has had prior setbacks. A patient death in 2021 led to a clinical hold on tests of the therapy. Pfizer was later cleared to resume clinical trials after implementing additional safety measures. But last month, Pfizer disclosed a patient death in a Phase 2 study evaluating its Duchenne gene therapy in boys ages 2 to 3. Dosing in that study is complete. Pfizer said dosing in the crossover arm of the Phase 3 study has paused as the company continues to gather information to understand what caused the patient death in the Phase 2 test. In the latest results reported Wednesday, Pfizer said the gene therapys safety profile was manageable and adverse effects were mostly mild to moderate.

The developments unfolding for Pfizers gene therapy come as the FDA weighs whether to award full approval to Elevidys, a Duchenne gene therapy developed by Sarepta Therapeutics. Nearly a year ago, the Sarepta therapy won accelerated approval for Duchenne patients ages 4 and 5. But last fall, the company reported the failure of the Phase 3 study meant to confirm the therapys benefit and support expansion to a wider range of patients. That pivotal study evaluated patients with the same measures used for Pfizers pivotal study.

Leerink Partners analyst Joseph Schwartz draws distinctions between the two gene therapy programs. Though both failed in Phase 3, Sareptas therapy showed statistically significant improvement according to its trials secondary measures, which are more sensitive in detecting benefit, he said in a Thursday research note. Furthermore, Sareptas gene therapy does not have the safety questions overhanging the Pfizer gene therapy.

Thus, with no efficacy signals and a less-than-pristine safety profile, we see this readout as the final nail in the coffin for the program and think it is unlikely to move forward, Schwartz said.

William Blair analyst Tim Lugo said in a research note that his firm did not view Pfizers gene therapy as a real competitive threat to Sarepta due to the safety concerns throughout its development. He echoed Schwartzs comments about the Sarepta therapys ability to hit the secondary goals of its study.

We believe overall the totality of the data generated to date support the efficacy of Elevidys, the current 4- and 5-year-old label, a conversion to full approval, and a broader expansion to include older boys and into non-ambulatory patients, Lugo said. However, we believe expansion into non-ambulatory patients is more of a stretch, and we would not be surprised if these patients are excluded from the expanded label.

Sareptas Elevidys faces a June 21 target date for an FDA decision.

Photo: Dominick Reuter/AFP, via Getty Images

Read the original:
Pfizer & Sarepta Gene Therapies Both Failed Phase 3, But Analysts Expect Sarepta Will Win Approval - MedCity News

Expanding on his earlier podcast discussion with EPR, Dr Arun Upadhyay, Chief Scientific Officer and Head of Research & Development at Ocugen, discusses the companys promising modifier gene therapy candidates for ophthalmic disorders.

OCU400 is a modifier gene therapy aimed at treating retinitis pigmentosa and Leber congenital amaurosis (LCA).

In April 2024, Ocugen received US Food and Drug Administration (FDA) clearance to initiate the Phase III liMeliGhT clinical trial for OCU400 for retinitis pigmentosa. Shortly thereafter, the European Medicines Agency (EMA) reviewed the study design, endpoints, and planned statistical analysis, and deemed the US-based trial acceptable for a Marketing Authorisation Application (MAA). In December 2023, the FDA granted OCU400 Regenerative Medicine Advanced Therapy (RMAT) designation.

The Phase III study will include 150 participants75 with the RHO gene mutation and 75 that are gene-agnostic. In each arm, participants will be randomised 2:1 to the treatment group (2.5 x 1010 vg/eye of OCU400) and the untreated control group.

Ocugen plans to expand the OCU400 clinical trial in the second half of 2024 to include patients with LCA, contingent on favourable results from the Phase I/II study and alignment with regulatory agencies.

EPR Podcast 24 Developing modifier gene therapy Ocugen

Unlike conventional methodsmodifier gene therapyemphasises the importance of the broader biological system, potentially leading to more effective treatments.

The gene-agnostic mechanism of action for OCU400 provides hope for a larger retinitis pigmentosa patient population and demonstrates the potential to expand the range of indications for which modifier gene therapy could apply.

Unlike conventional methods that typically focus on replacing a mutated gene with a functional copy, modifier gene therapy modifies gene expression using master gene regulators. These master regulators work in a gene-agnostic way and open the possibility for the treatment of diseases caused by different gene mutations.

Modifier gene therapy triggers epigenetic mechanisms to restore homeostasis in the cellular environment and thereby structural and functional improvement in affected cells.

This pragmatic approach emphasises the importance of the broader biological system, potentially leading to more effective treatments.

OCU410 is a modifier gene therapy for the treatment of geographic atrophy, an advanced stage of dry age-related macular degeneration (dAMD). It utilises an adeno-associated virus (AAV) delivery platform for the retinal delivery of the RAR-related orphan receptor A (RORA).

The RORA protein plays a crucial role in stress and metabolism, reducing lipofuscin deposits and oxidative stress, and demonstrates anti-inflammatory properties as well as inhibiting the complement system in in vitro and in vivo studies.

Ocugen is currently enrolling patients in the Phase I/II ArMaDa clinical trial to assess the safety and efficacy of OCU410 for GA secondary to dAMD.

The ArMaDa clinical trial will assess the safety and efficacy of unilateral subretinal administration of OCU410 in subjects with GA and will be conducted in two phases.

OCU410STis a modifier gene therapy utilising an AAV delivery platform (AAV5) for the retinal delivery of the RORA gene for treating the genetic eye disorder Stargardt disease.

The GARDian clinical trial will assess the safety and efficacy of unilateral subretinal administration of OCU410ST in participants and will be conducted in two phases.

In May 2024, Ocugen announced the second cohort (medium dose) completed dosing in the dose-escalation phase. To date, six patients with Stargardt disease have been dosed in the Phase I/II clinical trial. An additional three patients will be dosed with the high dose (cohort 3) in the dose-escalation phase.

Manufacturing of AAV vectors for gene therapy presents several significant challenges, such as:

By leveraging new technologies, [gene therapy] manufacturers can overcome existing challenges, streamline production workflows, and accelerate the development and commercialisation

There has been advancement in many areas related to gene therapy manufacturing, which has played a crucial role in accelerating gene therapy manufacturing by improving efficiency across the production process. By leveraging new technologies, manufacturers can overcome existing challenges, streamline production workflows, and accelerate the development and commercialisation.

Some of these improvements include:

Biopharmaceuticals, Clinical Development, Clinical Trials, Data Analysis, DNA, Drug Development, Drug Safety, Gene therapy, Industry Insight, Research & Development (R&D), Technology, Therapeutics

Read more:
Developing and manufacturing modifier gene therapy - European Pharmaceutical Review

Pictured: A scientist works behind an FDA sign/Taylor Tieden for BioSpace

The FDA approved 55 new drugs and 34 cell and gene therapies in 2023.But its not always good news that companies have to deliver to their stakeholders; the year also had its fair share of Complete Response Letters.

As we embark on 2024,BioSpaceis committed to keeping you up-to-date on all the FDAs actions in thisFDA Decision Tracker.

June 10

Product: Ipsen and Genfits Iqirvo

Indication: Primary biliary cholangitis

Monday, the FDA approved the first new drug in nearly a decade for primary biliary cholangitis: Ipsen and Genfits Iqirvo. A rare liver disease, PBC affects around 100,000 people in the U.S. and can lead to liver failure.

Iqirvo is intended to be used in combination with ursodeoxycholic acid (UDCA) in adult patients who have an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA.

The companies won accelerated approval for Iqirvo based on a reduction of alkaline phosphatase, a biochemical marker often used as a surrogate endpoint in PBC studies. Treatment with the drug demonstrated statistically significant improvements in biochemical response compared to UDCA alone, Christelle Huguet, executive vice president and head of research and development at Ipsen, said in a press release. An improvement in survival or prevention of liver decompensation events has not yet been shown, and the companies may need to run a confirmatory trial to verify Iqirvos clinical benefit.

June 10

Product: Almiralls Klisyri

Indication: Actinic keratosis

Dermatology company Almirall secured expanded approval of Klisyri for larger actinic keratosis-affected areas of the face or scalp. Klisyri can now be used to treat lesions up to 100 cm2 caused by the pre-cancerous dermatological condition, after safety and tolerability profiles were consistent with original pivotal trial results.

The new authorization for Klisyri, a microtubule inhibitor ointment, increases dosing for surface area treatment from up to 25 cm2 to up to 100 cm2, according to the companys press release.

In the same press release, Almirall Chief Scientific Officer Karl Ziegelbauer called the expanded approval a significant step forward for both patients and treating dermatologists, adding that the latter are looking for ways to treat the entire affected area to help prevent further lesion progression.

June 7

Product: Gerons Rytelo

Indication: Myelodysplastic syndromes

Geron Corporation kicked off the weekend on a high note as the FDA approval of its telomerase inhibitor Rytelo for myelodysplastic syndromes (MDS)a group of blood cancerssent the companys stock soaring more than 30%. Rytelo is specifically approved for MDS patients with transfusion-dependent anemia who do not respond to or are ineligible for the standard-of-care treatment, erythropoiesis-stimulating agents.

The approvalGerons first after 34 years in businesswas supported by data from the Phase III IMerge trial, in which patients on Rytelo had significantly higher rates of red blood cell transfusion independence over placebo for at least 24 weeks28% in the treatment arm versus 3% on placebo. For those who responded, this was sustained for a median of 1.5 years.

June 7

Product: GSKs Arexvy

Indication: Respiratory syncytial virus

People ages 5059 at an increased risk of severe outcomes from respiratory syncytial virus have a new preventative option after the FDA greenlit GSKs RSV vaccine Arexvy for this subgroup on Friday. Arexvy is indicated for the prevention of lower respiratory tract disease associated with RSV.

Fridays label expansionwhich was backed by strong immunogenicity and safety data in this populationextends the market reach for Arexvy, which became the first vaccine for RSV in May 2023, at that point intended for adults 60 and above.

GSK is also evaluating the vaccine for use in people 18-49 at increased risk of severe disease, and immunocompromised patients 18 and older.

May 31

Product: Modernas mRESVIA

Indication: Respiratory syncytial virus

Moderna has a second product on the market after the FDA approved mRESVIAformerly mRNA-1345to protect adults 60 years and older fromrespiratory syncytial virus (RSV). In a press release, Moderna CEO Stphane Bancel touted the strength and versatility of the companys mRNA platform, adding that the approval also marks the first time an mRNA vaccine has been approved for a disease other than COVID-19.

mRESVIA won approval based on the Phase III ConquerRSV trial, a global study of around 37,000 adults aged 60 or older in 22 countries, in which it displayed an efficacy rate of 83.7% against RSV lower respiratory tract disease. No serious safety concerns were identified in the trial.

May 30

Product: BMSs Breyanzi

Indication: Mantle Cell Lymphoma

After winning approval earlier this month in follicular lymphoma, Bristol Myers Squibbs Breyanzi got the FDA nod for another indication on Thursday: relapsed or refractory mantle cell lymphoma (MCL). Specifically, Breyanzi is approved for patients with MCL who have received at least two prior lines of systemic therapy, including a Bruton tyrosine kinase inhibitor.

The approval is backed by the results of the MCL cohort of TRANSCEND NHL 001, where treatment with Breyanzi elicited a 67.6% complete response rate in the target patient population.

Thursdays approval marks the fourth indication for Breyanzi, making it the CAR T cell therapy available to treat the broadest array of B-cell malignancies, according to BMSs press release.

May 29

Product: Eli Lillys Retevmo

Indication: RET-altered pediatric cancers

Eli Lilly won accelerated approval Wednesday for Retevmo to treat pediatric patients two years and older with RET-positive thyroid cancers and other solid tumors that carry the mutation. Retevmo is the first drug in the class available for children under 12 years of age, Pharmaphorum reported.

Retevmo is specifically indicated for advanced or metastatic medullary thyroid cancer with a RET mutation, advanced or metastatic thyroid cancer with a RET gene fusion untreatable with radioactive iodine therapy, and locally advanced or metastatic solid tumors with a RET gene fusion that have progressed after prior systemic treatment or have no treatment options, according to the publication.

The new approval for Retevmo, which was previously authorized to treat patients 12 and older with RET-positive thyroid cancers, is based on a single-arm study that showed an overall response rate of 48%, with a median duration of response not reached after 12 months of follow-up.

May 29

Product: Tris Pharmas Onyda XR

Indication: Attention deficit hyperactivity disorder

Wednesday, the FDA greenlit Tris Pharmas Onyda XR as the first non-stimulant medication for attention deficit hyperactivity disorder (ADHD) with a liquid formulation and nighttime dosing, according to the company. Onyda XR is a reformulation of clonidine hydrochloride, which was first approved by the FDA in 1974 to treat high blood pressure. Clonidine was approved for ADHD in 2010 under the brand name Kapvay, which is owned by Shionogi.

Onyda XR leverages Tris LiquiXR platform, producing a smooth, extended-release profile, per the biotech.

Approved for patients six years and older, Tris expects to have Onyda XR available in U.S. pharmacies by the second half of 2024.

May 29

Product: Tevas Austedo XR

Indication: Tardive dyskinesia and Huntingtons disease chorea

People with tardive dyskinesia and Huntingtons disease chorea have a streamlined treatment option after the FDA approved a new one-pill-a-day version of Tevas Austedo XR. The newly approved formulation offers more flexibility with the most once-daily doses of any vesicular monoamine transporter 2 (VMAT2) inhibitor, for these conditions, according to Tevas press release. Austedo XR comes in four tablet strengths: 30, 36, 42 and 48 mg.

Austedo XR, a once-daily extended-release formulation, was first approved in February 2023.

May 28

Product: Amgens Bkemv

Indication: Paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome

AstraZenecas rare disease drug Soliris now has a biosimilar on the market after the FDA greenlit Amgens Bkemv to treat paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). Bkemv was granted the FDAs interchangeability designation, which allows it to be used in place of the branded reference product without needing to change the prescription.

Like Soliris, Bkemv carries a boxed warning for meningococcal infections, which according to its label can be serious and life-threatening. Thus, it is only available through a restricted Risk Evaluation and Mitigation Strategies program.

May 16

Product: Amgens Imdelltra

Indication: Small cell lung cancer

Amgen secured approval Thursday for its first-in-class bi-specific T-cell engager, Imdelltra, for extensive-stage small cell lung cancer (SCLC). With the FDA nod, Imdelltra becomes the first bispecific T-cell engager therapy for advanced SCLC.

The accelerated approval was based on a Phase II study of 99 patients in the target population, where Imdelltra led to an overall response rate of 40% and a median duration of response of 9.7 months. Imdelltras label contains a boxed warning for serious or life-threatening cytokine release syndrome and neurologic toxicity, including immune effector cell-associated neurotoxicity syndrome, according to the FDAs press release.

May 15

Product: BMSs Breyanzi

Indication: Follicular lymphoma

Bristol Myers Squibbs Breyanzi is now approved for the treatment of relapsed or refractory follicular lymphoma after the FDA granted a label expansion under its accelerated approval pathway. The approval was backed by data from the Phase II TRANSCEND FL study in which treatment with the CAR T cell therapy led to a 95.7% overall response rate, with a complete response rate of 73.4%.

Breyanzi, which first won approval in February 2021 for relapsed or refractory large B cell lymphoma, is also authorized to treat small lymphocytic leukemia and chronic lymphocytic leukemia. By May 31, the FDA is expected to decide whether to grant approval for the therapy in refractory mantle cell lymphoma.

May 14

Product: Dynavaxs Heplisav-B

Indication: Hepatitis B patients undergoing hemodialysis

The FDA declined to approve the supplemental Biologics License Application for Dynavax Technologies hepatitis B vaccine in patients undergoing hemodialysis, deeming the safety and efficacy data submitted by the company insufficient.

In its Complete Response Letter, the regulator said the data was insufficient because a third-party clinical site operator destroyed data source documents for about half of the subjects enrolled in the vaccines trial, according to Reuters.

While the vaccine, Heplisav-B, initially won approval for the prevention of hepatitis B in 2017, its path to the market was rocky, with two previous rejections in 2013 and 2016 for unresolved safety concerns, per Reuters.

May 1

Product: Boehringer Ingelheims Cyltezo

Indication: Rheumatoid arthritis, Crohns disease, ulcerative colitis and more

Theres another new biosimilar option to AbbVies blockbuster antirheumatic Humira. Wednesday, the FDA greenlit a high-concentration and citrate-free version of Boehringer Ingelheims Cyltezo, which was originally approved in October 2021. The newly approved dose is 100 mg/mL and is sold at a 5% discount to the branded reference product.

Cyltezo is indicated for all the same conditions as Humira, including moderate-to-severe rheumatoid arthritis, Crohns disease and ulcerative colitis. Wednesdays approval is backed by data from the Phase I VOLTAIRE-HCLF study, which compared the bioavailability of the high- and low-concentration (50 mg/mL) formulation of Cyltezo in 200 healthy volunteers.

April 30

Product: Neurocrine Biosciences Ingrezza

Indication: Huntingtons disease

A more convenient version of Neurocrine Biosciences Ingrezza will be hitting the market to treat tardive dyskinesia and chorea in Huntingtons disease after the FDA closed out April by approving a sprinkle capsule formulation of the drug.

Like the original capsule version, which was approved in 2017 for tardive dyskinesia and in 2023 for chorea in Huntingtons, Ingrezzas sprinkle formulation comes in 40-mg, 60-mg and 80-mg doses but is designed to be opened and sprinkled on soft foods. This format could be more accessible for patients who have trouble swallowing whole capsules, according to the Neurocrines announcement, which also noted that a survey of Huntingtons patients with chorea and their caregivers showed that 62% had difficulty swallowing due to their involuntary movements.

April 29

Product: Pfizer and Genmabs Tivdak

Indication: Cervical cancer

The FDA has converted the accelerated approval of Pfizer and Genmabs Tivdak into a full nod for recurrent or metastatic cervical cancer that has progressed on or after chemotherapy.

The antibody-drug conjugate (ADC), which was originally developed under a partnership between Seagen and Genmab, was granted accelerated approval in September 2021 based on a 24% objective response rate seen in the Phase II innovaTV 204 trial.

In the Phase III innovaTV 301 study, which enrolled more than 500 patients, Tivdak significantly boosted survival versus chemotherapy. An October 2023 readout showed the ADC cut the risk of death by 30% in patients with recurrent or metastatic cervical cancer; it also reduced the risk of death or worsening disease by 33% versus chemotherapy. No new safety signals were observed.

April 29

Product: X4 Pharmaceuticals Xolremdi

Indication: WHIM Syndrome

The FDAapprovedX4 Pharmaceuticals Xolremdi Monday as the first targeted treatment for WHIM syndrome, an ultra-rare immunodeficiency disease named for its four characteristics: warts, hypogammaglobulinemia, infections and myelokathexis.

Myelokathexis is a congential disorder of the white blood cells, and Xolremdi, an oral CXCR4 antagonist, is designed to mobilize white blood cells such as neutrophils, lymphocytes and monocytes from the bone marrow into the blood to improve immune deficiencies.

In the Phase III 4WHIM trial, Xolremdi showed a 60% reduction in annualized infection rate compared to placebo; trial participants had less than one infection per year compared with 4.5 for the placebo group. Patients saw an even greater reduction with additional time on treatment.

Its an exciting time for personalized medicine, and I think WHIM is going to be a poster child for rare diseases and the ability where were at now in modern medicine to design therapies to treat underlying genetic disorders, Teresa Tarrant, an associate professor at Duke Universitys School of Medicine and lead investigator of the 4WHIM trial, told BioSpace prior to Xolremdis approval.

April 26

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Patients

We screened 316 participants for eligibility (Fig. 1). Five pediatric patients (two girls and three boys) with bilateral congenital hearing loss caused by biallelic OTOF mutations were enrolled from 14 July 2023 to 15 November 2023 (Fig. 1 and Table 1). Details of Sanger sequencing results and OTOF variant interpretation in patients are provided in Extended Data Fig. 1 and Extended Data Table 1. The average auditory brainstem response (ABR) threshold was >95dB in all patients at baseline (Table 1). None of the patients received cochlear implants before the trial. A dose of 1.51012vector genomes (vg) AAV1-hOTOF per ear, selected on the basis of the previous unilateral study11, was subsequently injected into the bilateral cochleae of the patient through the round window during a one-time operation. We have completed a 26-week assessment in patients 1, 2 and 3, and a 13-week assessment in patients 4 and 5. The study is ongoing.

Five patients were enrolled to receive binaural gene therapy and were evaluated for the primary endpoint. CI, cochlear implant.

Source data

The primary endpoint was dose-limiting toxicity, defined as hematologic toxicity grade 4, nonhematologic toxicity grade 3 or aural toxicity grade 2 within 6weeks. The grade was assessed according to Common Terminology Criteria for Adverse Events Version 5.0 (CTCAE V5.0). The dose of 1.51012vg AAV1-hOTOF was selected for bilateral treatment based on the results of the unilateral study that tested different doses11. No dose-limiting toxicity happened in five patients receiving binaural gene therapy with a dose of 1.51012vg AAV1-hOTOF per ear.

Efficacy outcomes include auditory function and speech perception. ABR, auditory steady-state response (ASSR), distortion product otoacoustic emission (DPOAE), and related questionnaires and tests were used to evaluate the auditory function, speech perception and sound source localization in patients.

At baseline, the average ABR threshold in the right (left) ear was >95dB (>95dB) in all five patients. In patient 1, the average ABR threshold in the right (left) ear was restored to 65dB (68dB) at 4weeks, 63dB (63dB) at 6weeks, 63dB (63dB) at 13weeks and 58dB (58dB) at 26weeks; the average ASSR threshold in the right (left) ear was 103dB (103dB) at baseline, and was restored to 48dB (63dB) at 4weeks, 53dB (58dB) at 6weeks, 53dB (58dB) at 13weeks and 53dB (58dB) at 26weeks (Fig. 2a). In patient 2, the average ABR threshold in the right (left) ear was >95dB (>95dB) at 4weeks, >85dB (>95dB) at 6weeks, 83dB (88dB) at 13weeks and 75dB (85dB) at 26weeks; the average ASSR threshold in the right (left) ear was 88dB (83dB) at 4weeks, 73dB (85dB) at 6weeks, 61dB (64dB) at 13weeks and 60dB (60dB) at 26weeks, compared with 79dB (81dB) at baseline (Fig. 2b). In patient 3, the average ABR threshold in the right (left) ear was restored to 63dB (63dB) at 4weeks, 63dB (60dB) at 6weeks, 60dB (58dB) at 13weeks and 55dB (50dB) at 26weeks; the average ASSR threshold in the right (left) ear was restored to 58dB (63dB) at 4weeks, 60dB (65dB) at 6weeks, 63dB (60dB) at 13weeks and 53dB (53dB) at 26weeks, compared with 100dB (100dB) at baseline (Fig. 2c). In patient 4, the average ABR threshold in the right (left) ear was >95dB (>95dB) at 4weeks, >90dB (>95dB) at 6weeks and 75dB (78dB) at 13weeks; the average ASSR threshold in the right (left) ear was restored to 95dB (95dB) at 4weeks, 85dB (85dB) at 6weeks and 63dB (60dB) at 13weeks, compared with 106dB (106dB) at baseline (Fig. 2d). In patient 5, the average ABR threshold in the right (left) ear was restored to 68dB (75dB) at 4weeks, 70dB (68dB) at 6weeks and 63dB (63dB) at 13weeks; the average ASSR threshold in the right (left) ear was restored to 68dB (71dB) at 4weeks, 60dB (65dB) at 6weeks and 60dB (63dB) at 13weeks, compared with 85dB (88dB) at baseline (Fig. 2e).

ae, The ABR and ASSR thresholds of patients 1 (a), 2 (b), 3 (c), 4 (d) and 5 (e). The arrows indicate no response even at the maximum sound intensity level. Arrows pointing left and downward, right ear; arrows pointing right and downward, left ear.

In both ears of patients 13, the signal-to-noise ratio (SNR) of DPOAE decreased at most frequencies at 4weeks and gradually recovered at the later follow-up (Extended Data Fig. 2ac). In patient 4, the SNR was stable at some frequencies at 4weeks, decreased to some extent at later follow-up and has not recovered at 13weeks (Extended Data Fig. 2d). In patient 5, the SNR decreased at some frequencies at 6weeks and recovered to some degree at 13weeks (Extended Data Fig. 2e).

In patient 1, the Meaningful Auditory Integration Scale (MAIS) and Categories of Auditory Performance (CAP) scores were 1 and 0, respectively, at baseline, and 28 and 4, respectively, at 26 weeks; the Speech Intelligibility Rating (SIR) and Meaningful Use of Speech Scale (MUSS) scores were 1 and 0, respectively, at baseline, and 1 and 7, respectively, at 26 weeks. The Speech of the Speech, Spatial, and Other Qualities of Hearing Scale for Parents (SSQ-P), the Spatial of the SSQ-P and the Other Qualities of the SSQ-P scores were 0.3, 0 and 0, respectively, at baseline, and were improved to 7.8, 2.8 and 5.0, respectively, at 26weeks (Table 2). In a quiet environment, the perception of monosyllable, disyllable and sentence was all 0% at baseline and 2.0%, 1.4% and 0%, respectively, at 26weeks after treatment; ambient sound, tone, initial and final was all 0% at baseline, and 31.3%, 31.3%, 20.8% and 20.8%, respectively, at 26weeks (Extended Data Table 2). For sound source localization tests, the bilateral root mean square error (RMSE) was 92.81.1 at baseline and 40.01.7 at 26weeks; when one ear was covered, the unilateral RMSE (75.51.0) at 26weeks was worse (Extended Data Table 2). In Supplementary Video 1, patient 1 could not hear at baseline and could recognize sound 4weeks and 6weeks after injection. At 13weeks, she could speak syllables such as a, ba (father), i, u, s and ma (mother). She was able to complete the sound localization test well at 13weeks.

In patient 2, the InfantToddler MAIS (IT-MAIS) and CAP scores were 0 and 0, respectively, at baseline, and 35 and 5, respectively, at 26 weeks; the SIR and MUSS scores were 1 and 0, respectively, at baseline, and 2 and 9, respectively, at 26 weeks; the Speech of the SSQ-P, the Spatial of the SSQ-P and the Other Qualities of the SSQ-P scores were all 0 at baseline and 6.7, 5.3 and 8.5, respectively, at 26weeks (Table 2). In Supplementary Video 2, patient 2 could not respond to sound and music at baseline, but he was able to turn to the sound source when his name was called from the left and right of his backward side 6weeks after injection. He could dance to the music and complete some simple instructions at 15weeks, and he could say some simple words, for example, ayi (aunt) and bai (bye), and communicate with others at 26weeks.

In patient 3, the IT-MAIS or MAIS, and CAP, scores were all 0 at baseline, and 35 and 5, respectively, at 26weeks; the SIR and MUSS scores were 1 and 0, respectively, at baseline, and 2 and 15, respectively, at 26weeks; the Speech of the SSQ-P, the Spatial of the SSQ-P and the Other Qualities of the SSQ-P scores were all 0 at baseline, and 7.3, 8.0 and 8.5, respectively, at 26 weeks (Table 2). In Supplementary Video 3, patient 3 had no response to sound and music at baseline, but he could turn back when his name was called 3weeks after injection. At 13weeks, he was able to move his body and dance when he heard the music. He was able to say some simple words at 26weeks, such as baba (father), nainai (grandmother) and yeye (grandfather).

In patient 4, the MAIS and CAP scores were 2 and 0, respectively, at baseline, and 16 and 4, respectively, at 13weeks; the SIR and MUSS scores were 1 and 2, respectively, at baseline, and 1 and 7, respectively, at 13weeks; the Speech of the SSQ-P, the Spatial of the SSQ-P and the Other Qualities of the SSQ-P scores were 0.3, 0 and 0, respectively, at baseline, and 3.6, 5.8 and 4.5, respectively, at 13weeks (Table 2). In Supplementary Video 4, patient 4 had no response to sound at baseline, but she could turn back when her name was called 4weeks after injection. She could complete some instructions at 13weeks, and she could say simple words at 20weeks, for example, baba (father), mama (mother) and nainai (grandmother).

In patient 5, the IT-MAIS or MAIS, and CAP, scores were 2 and 0, respectively, at baseline, and 29 and 4, respectively, at 13weeks; the SIR and MUSS scores were 1 and 0, respectively, at baseline, and 2 and 7, respectively, at 13weeks; the Speech of the SSQ-P, the Spatial of the SSQ-P and the Other Qualities of the SSQ-P scores were 0.2, 0 and 0, respectively, at baseline, and 7.6, 7.2 and 6.6, respectively, at 13weeks (Table 2).

To minimize the potential inflammatory response, dexamethasone was used intravenously for 8days starting from 3days before AAV1-hOTOF bilateral injection. No serious adverse event (AE) occurred. A total of 36 AEs occurred (Table 3), including emesis (patient 1), fever (patient 2), increased lymphocyte counts (patients 14), decreased lymphocyte counts (patient 3), decreased neutrophil counts (patient 2), decreased hemoglobin levels (patients 2 and 3), increased triglyceride levels (patient 2), increased cholesterol levels (patients 25), transient reduction in fibrinogen levels (patient 3), increased creatine phosphokinase levels (patient 2), decreased haptoglobin levels (patients 1 and 5), increased lactate dehydrogenase levels (patients 25), hyperglycemia (patient 5), proteinuria (patient 1) and hematuresis (patients 1 and 4). All 36 AEs were grade 1 or 2. The most common AEs were increased lymphocyte counts (6 out of 36) and increased cholesterol levels (6 out of 36), followed by increased lactate dehydrogenase levels (5 out of 36). In patient 1, emesis occurred at 2h after injection and was resolved with symptomatic treatment within 1day. In patient 2, fever (highest temperature, 38.7C) occurred at 18days and 29days after injection, with mild cough and increased lymphocyte counts, but no evidence of pneumonia or other concomitant symptoms.

In addition, the structure of the ears was observed by computed tomography and magnetic resonance imaging, showing the normality of the ear structure after injection (Extended Data Figs. 3 and 4).

Neutralizing antibodies against AAV1 were increased in all patients at 6weeks after treatment (Extended Data Table 3). Vector DNA in the blood was not detectable in any patient at 7days after treatment (Extended Data Table 3). Interferon gamma (IFN-) enzyme-linked immunosorbent spot (ELISpot) responses to AAV1 capsid peptide pools with peripheral blood mononuclear cells (PBMCs) drawn from each patient at 6weeks after AAV1-hOTOF binaural gene therapy were negative (Extended Data Fig. 5)

See the original post:
Bilateral gene therapy in children with autosomal recessive deafness 9: single-arm trial results - Nature.com

Inspiration sparked by shaving

Stephan co-founded Tidal Therapeutics to help commercialize his immune-cell programming nanoparticles (acquired by Sanofi in 2022). His lab needed a new direction, and inspiration struck one morning as he watched his freshly sprayed shaving foam expand in his palm.

I thought, Let's explore foam, Stephan said. Maybe we can make a formulation of foam that is not like the foam in in our shaving foam, but something that is biocompatible, to deliver therapeutics.

The froth had properties that would be attractive in a drug-delivery vehicle. Its volume started small, but puffed up. The foam stayed where it was sprayed, and didnt slide away. These characteristics could help get a therapeutic into contact with more critical cells while also ensuring that it didnt slip away.

Trying new substances or approaches that come from things in everyday life that you wouldn't necessarily associate in medical applications is sometimes a really interesting way to drive down costs and deliver drugs more easily, Fitzgerald said. But it was definitely a little bit out of my wheelhouse.

Foam-as-medical-delivery method isnt without precedent, Stephan noted. Foam-based delivery already enhances certain applications like delivery of hemorrhoid medication and intra-uterine imaging.

But could foam enhance gene therapy?

To create a bio-compatible foam, Stephan and his team initially took inspiration from the food industry.

Cocktails, ice cream, yogurt: they know how to make things foamy, he said.

Stephan Lab members, including staff scientist Sirka Stephan, PhD, started experimenting with ingredients available from the pantry store, he said.

Importantly, these materials are dead cheap, Stephan said. Theyre available for pennies. Theyre manufactured at large scale, and because theyre already used for pharmaceutical applications like coating tablets theyre available pharmaceutical-grade.

The scientists formulated a solution of methylcellulose (a food binder) and xanthan gum (a food thickener) that, when aerated using two lab syringes, bubbles into an easy-to-apply froth.

But did it have the potential to improve gene therapy?

We started with a lot of hypotheses in terms of, could foam potentially concentrate our gene therapy, keep it more localized, and help it stay in the tissue where we wanted to adhere? Fitzgerald said. My job was to do the experiments to prove the hypotheses.

Foam has certain properties that make it an attractive drug-delivery vehicle. Its more than tightly packed air bubbles: In a foam, the bubbles are separated and surrounded by incredibly thin layers of continuous liquid, called lamellae. Active ingredients become highly concentrated in these lamellae, which allows foam to deliver highly concentrated doses of medicine to large areas, even if the total dose is small.

View original post here:
Taking a frothy risk to advance gene therapy - Fred Hutchinson Cancer Center

The first clinical trial to administer gene therapy to both ears in one person has restored hearing function to 5 children born with a form of inherited deafness, astounding the research team..

Two of the children even gained an ability to appreciate music.

The success of the new approach is detailed in a new study published in Nature Medicine. The workbuilds on the first phase of the trial, published earlier this year, in which children were treated in a single ear.

The results from these studies are astounding, says study co-senior author Zheng-Yi Chen, an associate scientist in the Eaton-Peabody Laboratories at Massachusetts Eye and Ear in the US.

We continue to see the hearing ability of treated children dramatically progress and the new study shows added benefits of the gene therapy when administrated to both ears, including the ability for sound source localisation and improvements in speech recognition in noisy environments.

Lead author Yilai Shu, an attending doctor in otolaryngologyand an investigator at Eye and Ear, Nose, Throat hospital of Fudan University, China, says: restoring hearing in both ears of children who are born deaf can maximise the benefits of hearing recovery.

These new results show this approach holds great promise and warrants larger international trials.

The children involved in the study were born with DFNB9, which accounts for between 2 and 8% of hereditary deafness. It is caused by mutations in the OTOF gene which prevent the production of functioning otoferlin protein, which can significantly reduce sound transmission from the inner ear hair cells to hearing nerves.

Through minimally invasive surgery, Shu injected adeno-associated virus (AAV) engineered to carry and deliver functioning copies of the human OTOF transgene, into the childrens inner ears.

All 5 children were observed over a 13 or 26-week period and showed hearing recovery in both ears, with dramatic improvements in speech perception and sound localisation.

During follow-up, 36 adverse events were observed, the most common being increased white blood cell counts and increased cholesterol levels. However, there were no dose-limiting toxicity or serious adverse events.

The trial is continuing, and participant are stillbeing monitored.

The authors say their results show that the gene therapy is feasible, safe, and efficacious.

Read the original post:
Gene therapy restores hearing to children with inherited deafness - Cosmos