Courtesy of Sarepta Therapeutics

In its second-quarter conference call, Sarepta Therapeutics indicated plans to accelerate its timeline for SRP-9001 (delandistrogene moxeparvovec), a gene therapy for Duchenne muscular dystrophy (DMD).

Sarepta currently has three antisense therapeutics for DMD on the market. The company's original plan was to not apply to regulators until 2023, but now, it plans to submit the drug to the U.S.Food and Drug Administration in the next few months, with plans to launch the drug in mid-2023.

In July, Sarepta and its partner on the drug, Roche, presented promising functional data at the 17th International Congress on Neuromuscular Diseases (ICNMD 2022) in Brussels. DMD is a rare genetic disease that results in progressive muscle degeneration and weakness. Caused by mutations in the dystrophin gene, which codes for muscle, the disease primarily occurs in boys and typically causes impaired pulmonary function, acute respiratory failure and death.

SRP-9001 is a gene transfer technology that delivers the therapy to muscle tissue for the targeted production of essential dystrophin. Sareptas other DMD therapies utilize gene skipping technology. In that, the therapy skips over the deleted part of the gene, resulting in a truncated but mostly functional dystrophin protein.

The data from Cohort 1 of Study SRP-9001-103, ENDEAVOR, demonstrated a 3.8-point improvement (unadjusted means) and 3.2-point improvement (least squared means) on the North Star Ambulatory Assessment (NSAA) 52 weeks after treatment compared to external control.

Doug Ingram, Sareptas CEO, launched right into the conference call by discussing the accelerated regulatory plans for SRP-9001, noting, we previously disclosed that we were engaging with the U.S. FDA about the possibility of submitting a Biologics License Application, or BLA, for the Accelerated Approval of SRP-9001 to treat Duchenne muscular dystrophy. We also cautioned numerous times that we would not change our base case assumption on the timing of approval unless we have strong conviction on the receptivity to an Accelerated Approval BLA by the FDA. As we announced last week, our discussions are now complete, and our base case assumption has indeed changed.

They expect to submit the BLA this fall. Its not clear yet if an advisory committee meeting will be called, but they think it would likely be in the spring of 2023, and, Assuming a successful review, we anticipate approval and launch in mid-2023, Ingram said.

Further, they are ramping up commercial preparations for what Ingram said will be the largest gene therapy launch in the United States.. He added, "That will include augmenting our commercial and medical affairs organizations, site readiness, and importantly, building sufficient inventory to serve the community at large without delay.

The company is also planning to launch its Pivotal EMBARK trial in 120 patients for the gene therapy. Ingram said that the demand has been intense for EMBARK and site initiation and enrollment ramped up enormously in the second quarter. Based on our current screen rates, we should be fully screened and enrolled in the next few weeks.

Because Accelerated Approvals, typically based on biomarker results rather than clinical benefit, require confirmatory trials, Ingram noted that the FDA and Congress have been encouraging companies to already have confirmatory in motion at the time of Accelerated Approval. Sarepta expects EMBARK to be its confirmatory trial, which is expected to be fully enrolled by the time the BLA is filed.

Companies focusing on DMD gene therapies have proceeded cautiously after a fatal case of myocarditis was observed in Pfizers gene therapy candidate. In May 2022, four companies, Pfizer, Sarepta, Genethon and Solid Biosciences, were all observing serious side effects in their gene therapy clinical trials for DMD. In an unprecedented move, they partnered to analyze the data along with independent experts and then presented it at the American Society of Gene and Cell Therapy Meeting.

Serious adverse events (SAEs) were seen in five patients across three trials, and appeared about three to seven weeks after the initial gene infusion. Muscle weakness and variable cardiac involvement were observed.

Researchers now believe the SAEs were related to a specific transgene/genotype-related class effect. They believe that the mechanism of the SAEs was a T-cell-mediated immune response to the transgene protein expressed by all of the therapies in a cross-reactive immune response, but it was determined by the patients genotype. The SAEs occur only in patients with specific genome deletions, including N-terminal epitopes, which are present in the transgene protein.

It's a good bet that due to the issues, the FDA will call for an advisory committee meeting.

Previous DMD therapies were designed for patients with specific mutations in the dystrophin gene. These are Exondys 51, Vyondys 53 and Amondys 45. This new therapy is expected to target the ambulatory patient population, which Sarepta indicates is about 50% of the market. The company also plans to launch the ENVISION Study 303 in the non-ambulatory patient population later this year.

The rest is here:
Sarepta to Expedite Timeline for Muscular Dystrophy Gene Therapy - BioSpace

Gene therapy research and development has been progressing the field of genetic disease by striving to provide an effective therapeutic approach to correcting the genes involved with specific conditions.

The mission of biotech company Odylia Therapeutics is to bring therapeutics to patients with rare diseases, regardless of prevalence or commercial opportunity.

Odylia is a nonprofit organization, setting it apart from traditional biotech and pharmaceutical companies. President and CSO, Ashley Winslow, PhD, explained that while the science and research functions the same way, the fundraising efforts are different and the organiztion utilizes its profits as an investment for furthering its mission.

We are very focused, as a nonprofit, on the treatments we're trying to develop, or how we're helping other groups, so everything comes back to the mission, she said.

The organization's current gene therapy pipeline is focused on 2 rare ocular diseases, according to Winslow. In this interview with HCPLive, she spoke about the genes each program is working with to address Leber congenital amaurosis (LCA) and Usher syndrome.

The transparent practices of a nonprofit like Odylia has opportunities that industry companies may not.

I really love that aspect of my job, Winslow said, because having worked in industry before, you don't get the opportunity to talk every day about what you do. As a nonprofit, we have to talk about it. It's not only inspiring, it's easy to get up every day and do what we do.

Education and awaress are escpecially critical when working with rare diseases.

We have to speak to it in order to really attract people to our mission, she said. And that's what we're trying to do through fundraising efforts is speak to what we've accomplished, what we're seeking to accomplish, where we are pushing boundaries.

Read more:
Nonprofit Research on Gene Therapy for Rare Ocular Diseases - MD Magazine

New York, Aug. 02, 2022 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Gene Therapy Market by Vectors, Indication, Delivery Method, Region - Global Forecast to 2027" - https://www.reportlinker.com/p05843076/?utm_source=GNW However,factors like high cost of gene therapy is restraining the growth of this market.

The cancer segment accounted for the highest growth ratein the gene therapy market, by indication, during the forecast periodIn 2021, cancer segment accounted for the highest growth rate. Growing disease burden of cancer across the globe coupled with rising demand for gene therapies to treat cancer will augment the segmental growth of cancer over the forecast period.

Asia Pacific: The fastest-growing region in the gene therapy marketThe Asia Pacific market is estimated to record the highest CAGR during the forecast period. The high growth rate of this market can be attributed to the improving healthcare expenditure in emerging economies, increasing product launches, and increasing incidence of cancer and neurological diseases.

The primary interviews conducted for this report can be categorized as follows: By Company Type: Tier 1- 32%, Tier 2- 44%, and Tier 3-24% By Designation: C-level (Managers) - 30%, D-level(CXOs, Directors)- 34%, and Others (Executives) - 36% By Region: North America -50%, Europe -32%, Asia-Pacific -10%, Rest of the World -8%

List of Companies Profiled in the Report: Biogen (US) Sarepta Therapeutics (US) Gilead Sciences, Inc. (US) Amgen, Inc. (US) Novartis AG (Switzerland) Orchard Therapeutics Plc (UK) Spark Therapeutics, Inc. (A Part Of ?F. Hoffmann-La Roche) (US) AGC Biologics (US) Anges, Inc. (Japan) Bluebird Bio, Inc. (US) Jazz Pharmaceuticals Plc (Ireland) Dynavax Technologies (US) Human Stem Cells Institute (Russia) SibionoGenetech Co., Ltd. (China) Shanghai Sunway Biotech Co., Ltd. (China) Uniqure N.V. (Netherland) Gensight Biologics S.A. (France) Celgene Corporation (A Bristol-Myers Squibb Company) (US) Cellectis (France) Sangamo Therapeutics (US) Mustang Bio (US) AGTC (Applied Genetic Technologies Corporation) (US) Poseida Therapeutics, Inc. (US)

Research Coverage:This report provides a detailed picture of the global gene therapy market.It aims at estimating the size and future growth potential of the market across different segments such as vectors, indication, delivery method, and region.

The report also includes an in-depth competitive analysis of the key market players along with their company profiles recent developments and key market strategies.

Key Benefits of Buying the Report:The report will help market leaders/new entrants by providing them with the closest approximations of the revenue numbers for the overall gene therapy market and its subsegments.It will also help stakeholders better understand the competitive landscape and gain more insights to better position their business and make suitable go-to-market strategies. This report will enable stakeholders to understand the markets pulse and provide them with information on the key market drivers, challenges,trends,and opportunities.Read the full report: https://www.reportlinker.com/p05843076/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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The global gene therapy market is valued at an estimated USD 7.3 billion in 2022 and is projected to reach USD 17.2 billion by 2027, at a CAGR of...

When you encounter a big challenge, it takes teamwork to face it. Speeding novel gene therapies to patients with rare diseases has certainly been a challenge so much so that the federal government is now leading an exciting new public-private partnership aimed at solving it.

The Food and Drug Administration (FDA) and National Institutes of Health (NIH) launched the Bespoke Gene Therapy Consortium (BGTC) last October. The five-year initiative, managed by the Foundation for the National Institutes of Health (FNIH), is focused on accelerating the development of gene therapies for rare diseases and includes many partners from industry, including my employer, Aldevron, and other life sciences companies at Danaher Corporation.

Participating in BGTC is both a personal and professional passion for me. Having lost my daughter in 2018 to Batten disease, a rare genetic disorder that could be a prime target for gene therapy, I understand the urgency of BGTCs mission.

Enabling and accelerating the development of genomic medicines like gene therapy are commitments Danaher and its companies have made. In my work, I often encounter families who are facing diagnoses of rare diseases and have precious little time to wait for breakthrough treatments to hit the market.

I understand their frustration. And I hope many more biopharma companies will join the effort to foster gene therapy development in Batten disease and other genetic disorders.

In 2006, my family met with a geneticist after we noticed that our daughter, Taylor, was losing her vision. She was diagnosed with CLN1 disease, a form of Batten disease thats caused by a mutation in the gene PPT1. This gene produces an enzyme thats critical to the functioning of lysosomes, the components of cells that digest nutrients. Defects in PPT1 prevent cells from breaking down fats and other harmful substances, causing a range of neurological disabilities.

The geneticist told us Taylor would become blind and lose her ability to walk, talk and swallow and shed have seizures. We looked at him and said, So what can we do? He told me there was nothing we could do, but I couldnt accept that she was about to turn eight years old. As it turned out, we couldnt do anything more than treat Taylors seizures and pain. There was nothing to get at the root cause of her disease.

We wanted to contribute to the development of treatments for children like Taylor, so we launched a public charity and have been supporting gene therapy research since 2013.

I have always been reticent to use the word cure, but I wanted something that would make life better for my daughter and nothing existed. You cant lose hope, even when you know your child is not going to survive.

There are about 7,000 rare diseases, most of which are caused by a single defective gene. Yet there are only two gene therapies approved to treat heritable rare diseases. There are FDA-approved treatments for several hundred rare diseases, but they treat symptoms rather than offer the opportunity for cures.

I see great potential for gene therapy. In addition to the two approved gene therapies, there are many strong candidates in clinical and preclinical testing for monogenic diseases. The gene therapy our public charity supported in 2013 is now being developed by a biotech company and a clinical trial is on the horizon.

But we need more gene therapies in the pipeline, because when its your child, its not an ultra-rare disease, Its an illness that needs an answer.

The BGCT falls under the umbrella of the FDAs and NIHs Accelerating Medicines Partnership programme, which focuses on building a better understanding of biological pathways that could be targets for new treatments.

BGCT will fund research focused on improving the most common delivery vehicle used in gene therapy, the adeno-associated virus (AAV). It will also develop analytics aimed at improving AAV manufacturing, conduct clinical trials of gene therapies for rare diseases and test methods for streamlining regulatory processes, so new gene therapies can get to patients faster.

The fund could help address many of the challenges facing gene therapy developers today. For example, AAVs are difficult to develop and to manufacture at commercial scale, intensifying the need for novel and efficient solutions. By conducting several gene therapy clinical trials, each using a different AAV, the BGTC will gain a deep understanding of whats needed to improve viral vector manufacturing and shorten the path from animal studies to clinical development.

It will also look closely at the challenge of streamlining regulatory requirements for the approval of gene therapies. This will include standardising approaches to preclinical studies. Its important to develop standards so developers have a playbook to follow, and they dont get delayed trying to answer questions that have already been answered. These standards will create efficiencies, which in turn will speed more gene therapies to the patients who need them.

The BGTC is bringing government agencies, companies, and non-profits together to tackle the many challenges of developing gene therapies. I hope that with the support of all of the participants, we will be able to accelerate innovative gene therapies to so many patients who have rare diseases.

Sharon King is manager of Advocacy and Community Engagement at Aldevron. Sharon and her family founded Taylors Tale, a public charity supporting gene therapy. Go to taylorstale.org

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Group therapy - PharmaTimes - PharmaTimes

Wilmington, Delaware, United States, Transparency Market Research Inc.: Gene therapy is one of the best treatment options for most chronic diseases. It involves inserting a functional copy of a gene into a defective cell. Gene therapy is useful in the treatment of cancers, inherited disorders, cardiovascular diseases, and infectious pathogen neurological disorders.

Read Report Overview https://www.transparencymarketresearch.com/viral-vectors-manufacturing-market.html

Viral or non-viral vector methods are used in efficient transfer of therapeutic gene into the target cells. Viral vectors used in gene therapy include adenovirus, lentivirus, retrovirus, and adeno-associated viral (AAV). Non-viral vectors generally depend on delivery of plasmid DNA.

Development of quality vectors in terms of formulation, physical size, cost, and delivery function is quite challenging. To minimize this problem, manufacturers use various approaches such as development of cell line culture, current good manufacturing practices, cell culture system, and expression systems that are used in the development of vectors. This is projected to boost the growth of the global viral vectors manufacturing market.

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Additionally, increase in the number of gene therapy candidates due to rapid development of diseases and rise in funding for gene therapies are expected to fuel the growth of the global viral vectors manufacturing market. The Alliance for Cancer Gene Therapy (ACGT) is a public charity foundation in the U.S. which funds for advancement in cancer gene therapies from laboratory to clinical trials. However, high cost of gene therapies and possible mutagenesis restrain the market.

The global viral vectors manufacturing market can be segmented based on type, disease, application, and region. In terms of type, the global market can be divided into adenoviral vectors, retroviral vectors, adeno-associated viral vectors, and others. The retroviral vectors segment dominated the global viral vectors manufacturing market due to ease of application in major target diseases such as cancer and genetic disorders. Based on disease, the global viral vectors manufacturing market can be classified into cancers, infectious diseases, genetic disorders, and other diseases.

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The genetic disorders segment is anticipated to dominate the market due to increase in research activities on various genetic disorders such as sickle cell anemia, hemophilia A and B, and Huntingtons disease, and a strong gene therapy pipeline in the last phase of drug development. In terms of application, the global market can be bifurcated into gene therapy and vaccinology. The gene therapy segment is expected to account for the largest share of the market due to increase in the number of gene therapy clinical trials conducted for chronic diseases such as cancer, cardiovascular diseases, and neurodegenerative diseases globally.

Geographically, the global viral vectors manufacturing market can be segmented into North America, Europe, Latin America, Asia Pacific, and Middle East & Africa. Each region can be divide into specific countries/sub-regions such as the U.S., Canada, the U.K., Germany, Brazil, China, India, and GCC Countries. North America dominated the global viral vectors manufacturing market because of increase in research activities, large number of regenerative medicine companies, rise in prevalence of target diseases, and availability of funds. Asia Pacific is expected to be the most attractive market during the forecast period due to increase in health awareness and demand for advanced medical technology.

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Key players operating in the global viral vectors manufacturing market are Lonza, Merck, Oxford BioMedica, CGT Catapult, Cobra Biologics, uniQure, FUJIFILM Diosynth Biotechnologies, Kaneka Eurogentec, and Spark Therapeutics, among others. These players adopt various strategies such as collaborations, agreements, partnerships, and launch of new products to gain competitive advantage in the market.

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Viral Vectors Manufacturing Market: Increase in the Number of Gene Therapy Candidates due to Rapid Development of Diseases to Drive the Market -...

The combined group will offer process development expertise and cGMP manufacturing for a broad range of autologous and allogeneic cell therapies, with unparalleled expertise in gene editing and industry-leading induced Pluripotent Stem Cell (iPSC) capabilities.

In particular, the group will benefit from significantly expanded capacity: with process and analytical development laboratories and cGMP manufacturing facilities in Edinburgh, Scotland, and in Hopkinton, Massachusetts.

As a CDMO focused on cell-based therapies, Lykan has a 64,000 sq. ft. cell therapy manufacturing facility and innovation/development laboratories, with 16 cGMP processing suites to be running by the end of 2022.

Further laboratory and cGMP capacity expansion in Scotland is planned to build on cell and gene therapy specialist RoslinCTs existing 40,000 sq. ft facilities, including 8 cGMP suites.

The two companies note that demand for high-quality development and manufacturing capacity is increasing across the world: adding that its combination can shorten development and manufacturing timelines for advanced therapy sponsors.

This is thanks to GMP manufacturing capability that stretches all the way from clinical through commercial, meaning there is no need for a tech transfer to a commercial CDMO during the project, the companies told us.

The combined expertise also means the newly formed group is better placed to serve development challenges (for example, the RoslinCT iPSC platform and expertise can be leveraged to accelerate development timelines).

And with a manufacturing footprint on both sides of the Atlantic, this can bring manufacturing closer to the patient without the need to bring in other CDMOs.

Asked what the main strength of the new combined company will be, the group highlights its ability to partner with sponsors to take a therapeutic all the way from development through to commercial manufacture.

Both businesses are set up for commercial GMP manufacture: and while neither currently manufacture any commercialized product, they expect to begin this next year on the back of a successful clinical trial run by RoslinCT with a long-term undisclosed customer.

RoslinCT CEO Peter Coleman and Lykan President & CEO Patrick Lucy will remain in their current roles: leading a combined global workforce of 300 employees.

Peter Coleman, CEO of RoslinCT said:This combination puts us in a strong position as a leading global CDMO in the process development and manufacturing of advanced cell therapies, and we look forward to working with our new colleagues at Lykan to fuel future growth and meet the increasing demand for innovative therapies.

Patrick Lucy, President & CEO of Lykan Bioscience, added:We are delighted to combine with RoslinCT to better serve the growing demand for manufacturing capacity and expand the range of innovative services we can provide our partners to support the development of advanced cell and gene therapies.

Global Healthcare Opportunities, a European specialist investor in global healthcare, announced its investment in RoslinCT earlier this year. As part of the business combination, GHO is making a majority investment in Lykan and is backing the funding of the combined entity.

WindRose Health Investors, previously the majority owner of Lykan Bioscience, has reinvested in the new combined group along with Lykan Management.

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RoslinCT and Lykan Bioscience combine to create advanced cell therapy CDMO - BioPharma-Reporter.com

Sickle cell disease (SCD) is a group of genetic conditions that affect the function of hemoglobin. Health experts are exploring gene therapy as a potentially new treatment to manipulate gene expression. With this technique, it may be possible to restore the shape of red blood cells (RBCs) and eliminate SCD complications.

SCD is a group of genetic RBC disorders that affects roughly 100,000 people in the United States. At present, most treatments aim to reduce symptoms and complications. However, advances in gene therapy may offer a new curative approach.

In this article, we will discuss gene therapy and its potential role in treating sickle cell disease.

SCD is an inherited RBC condition. A person with SCD has a gene alteration in the hemoglobin beta (HBB) gene present on chromosome 11. Hemoglobin is an iron-rich protein in RBCs that gives them their shape and helps transport oxygen. Healthy RBCs are round and flexible, allowing them to travel through blood vessels.

However, in people with SCD, RBCs are rigid and shaped like a C or sickle, which is how the condition gets its name. As these C-shaped cells are misshapen, the body breaks them down more quickly than healthy RBCs. They can also become stuck and interrupt blood flow, which can cause pain and infections.

There are several types of SCD, depending on what genes biological parents pass on. The most common types include:

Gene therapy refers to a medical approach that aims to treat genetic conditions. This technique modifies gene expression to prevent gene changes from causing symptoms of a disease. Different mechanisms are available and may include:

Gene therapy aims to treat genetic diseases by providing cells with a new set of instructions to change how they function, with the aim of correcting the condition. SCD results from alterations in the HBB gene, which produces the protein beta-globin. By adding a new version of this gene, it may be possible to prevent RBCs from developing a sickle shape.

To begin this therapy, doctors will first collect stem cells either from a persons bone marrow or a blood sample using a medication called plerixafor. This drug helps move stem cells from the bone marrow into the bloodstream. The doctors will modify these stem cells outside of the body.

Doctors then need a carrier to transport the new genetic material into the stem cells they call this carrier a vector. Usually, the vector will be a virus that health experts have modified to make harmless and instead carry the new genes. Similar to how viruses replicate by injecting genetic material into living cells, the modified viruses insert the new genes into stem cells.

Before a person can receive these new stem cells, they first undergo a procedure called conditioning. Conditioning uses chemotherapy to create space in the bone marrow for the new stem cells. A person will then receive a blood infusion containing the new stem cells.

Researchers are also investigating a technique known as gene editing to treat SCD. Gene editing works by adding, removing, or altering genetic material to change how cells work. One of the main approaches for treating SCD is to encourage the production of fetal hemoglobin (HbF). This type of hemoglobin is present in a fetus but becomes suppressed as the child ages. Unlike adult hemoglobin, the altered sickle cell gene does not affect HbF.

Gene editing aims to stop the suppression of HbF by targeting a gene called BCL11A. By suppressing this gene, the body can resume producing HbF and, as a result, have healthy RBCs. This type of gene therapy involves similar steps of collection, a vector, and an infusion. However, instead of delivering new genetic material, the vector transports a gene editing technology called CRISPR/Cas9 to interrupt the BCL11A gene.

Although SCD will still be able to affect some of the RBCs in the body after this treatment, research estimates that a 20% level of HbF in the bloodstream can be enough to improve SCD symptoms.

New advances in gene therapy have led to several studies and trials that show promising results for the potential treatment of SCD.

A 2022 study investigated the effectiveness of a gene therapy for SCD called LentiGlobin and found that a one-time treatment led to a sustained increase in nonsickling hemoglobin in participants blood. This treatment led to a reduction in hemolysis and resolution of severe vaso-occulusive events. This refers to a type of sickle cell crisis, where sickled RBCs block blood flow and cause severe complications.

A 2019 study notes a successful trial using a modified vector to transfer a healthy HbF gene to people with SCD. In both cases of using this technique, individuals saw improvements in the SCD symptoms.

Similarly, a 2021 study notes a successful trial where CRISPR/Cas9 gene editing techniques were successful in targeting the BCL11A gene. After treatment, the individual had higher levels of HbF and no vaso-occlusive episodes.

Although gene therapy research for SCD is ongoing, early trials seem to be yielding positive results. The technique can potentially increase the level of healthy, functioning hemoglobin and reduce severe pain crises.

A potential risk from gene therapy comes from the need for a person to undergo chemotherapy beforehand to prepare their body for new stem cells. Potential side effects of chemotherapy may include:

There have also been cases of participants developing leukemia and myelodysplastic syndrome after gene therapy for their SCD. However, the research is inconclusive as to whether this occurs due to the chemotherapy conditioning, the gene therapy itself, or because people with SCD may have a higher risk of these cancers.

Since health experts tailor gene therapy specifically for each individual using their own cells, it is a time-consuming and expensive procedure. This high cost may significantly limit its availability for many people.

A further limitation is that since research into new gene therapy techniques is still new, healthcare professionals do not fully understand the long-term effects and safety of these treatments.

The only current curative treatment available for SCD is a bone marrow transplant. This procedure works similarly to gene therapy, but the healthy RBCs come from the bone marrow of a compatible donor.

Other treatments aim to manage symptoms, lower the frequency of sickle cell crises, and reduce the risk of complications. Such treatments may include:

Sickle cell disease is a group of genetic conditions that affects hemoglobin. These genetic alterations result in health complications, as red blood cells cannot function correctly. Gene therapy is a potentially curative treatment that aims to encourage the production of more healthy RBCs to alleviate symptoms.

Although trials are still ongoing and the long-term effects are still unclear, promising results show the potential for gene therapy as a treatment option.

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Can gene therapy help treat sickle cell? - Medical News Today