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Exome sequencing reveals genetic heterogeneity and clinically actionable findings in children with cerebral palsy - Nature.com

Recommendation and review posted by Bethany Smith

WATCH TIME: 6 minutes

"Geographic diversity is incredibly important in Parkinson research. Tasso's device helps reach patients in remote areas where access to phlebotomists may be limited."

PD GENEration: Mapping the Future of Parkinsons Disease,an international genetics study led by the Parkinsons Foundation, tests for mutations among select, clinically relevant genes to accelerate research, advance treatments, and improve care for patients with Parkinson disease (PD). The goal of the study is to make genetic testing accessible to patients with PD, empower those with the disease and their clinicians to know their genetic status, and identify clinical trials in which they might enroll. PD GENEration returns genetic findings to all participants through a genetic counseling session, bridging the knowledge gap between patients and clinicians to accelerate research collectively.

In recent news, the foundation announced a partnership with Tasso that would leverage the companys patient-centric, end-to-end sample collection and logistics platform for PD GENEration.1 Patients in the study will receive a kit containing a Tasso device that will collect a small blood sample in the comfort and convenience of their home. Online proctors will help guide participants through the collection process and provide support for a successful collection. After collection, patients can ship their sample in a pre-paid box to a lab for research analysis. Overall, the goal of this research is to screen the collected samples for mutations among select, clinically relevant PD genes.

James Beck, PhD, senior vice president and chief scientific officer of the Parkinsons Foundation, and Ben Casavant, PhD, CEO and cofounder of Tasso, recently sat down with NeurologyLive in an interview to discuss how the blood collection device can simplify the process of collecting a sample of blood compared with traditional methods. The duo also talked about the role of genetic analysis in Parkinson research, and how Tasso is contributing to it. Additionally, Beck and Casavant talked about the importance of geographic diversity in the context of Parkinson research and patient access to testing.

Click here to learn more about PD GENEration.

Excerpt from:
Using a Blood Collection Device to Advance Genetic Research in Parkinson Disease: James Beck, PhD & Ben ... - Neurology Live

Recommendation and review posted by Bethany Smith

A high-risk cancer genetics program at Erlanger is helping improve cancer treatment and screenings for patients in our community.

Actress Olivia Munn says a breast cancer risk assessment helped save her life. She announced last year that she had been diagnosed with Luminal B Cancer, which is an aggressive, fast-moving cancer.

A similar program is available at Erlanger.

McKenzie Smartt, Erlanger, NP-C says, Our cancer genetics program we can do genetic testing to test your DNA to see if you have any hereditary conditions that were passed down from your family that could possibly increase your risk for cancer.

Smartt says patients can also enroll in their high-risk program.

McKenzie Smartt says, Where if you do not have anything genetic you can still have an increased risk for cancer based off family history. So, we can manage the increased risk as well.

Smartt says everyones risk is different. While this program is for everyone, it is geared toward women.

McKenzie Smartt says Specifically, women over the age of 25. The guidelines now suggest women over 25 should have a risk assessment.

A lot of that has to do with the fact that 1 in 8 women will be diagnosed with breast cancer in their lifetime.

Smartt says they also are concerned about other cancers like colon cancer and prostate cancer.

McKenzie Smartt says We want to be proactive in the community and be able to identify those who are at risk, catch cancer early so we can begin treatment as quickly as possible.

For more details, click here.

Visit link:
EYE ON HEALTH: High risk and genetic testing being offered at local hospital - Local 3 News

Recommendation and review posted by Bethany Smith

GREENFIELD It took nearly 35 years, but the human remains found just off Route 78 in Warwick in 1989 have been identified as belonging to Constance (Holminski) Bassignani, who was 65 years old at the time of her murder.

The Northwestern District Attorneys Office held a press conference Thursday morning to announce that the victims identity was learned about eight months after her DNA was submitted to Othram, a Texas corporation that specializes in using forensic genetic genealogy to resolve unsolved murders, disappearances and identification of unidentified decedents or homicide victims.

According to the DAs office, Bassignani was born in Hawaii in 1924 and was living with her second husband, William Bassignani, in Woonsocket, Rhode Island, at the time of her death. William reportedly told family that she had moved back to Hawaii, though the DAs office stated investigators found no evidence of this. William, who died in 1993, is considered a person of interest in the case.

The State Police Detective Unit attached to DAs office collaborated with the State Police Crime Laboratory to submit the genetic material to Othram. This led to living potential relatives, who submitted their own DNA and confirmed the victims identity.

Were all very appreciative of the dedication, the hard work and the perseverance in this case, Northwestern District Attorney David Sullivan said. Seeking justice for the unknown victim has been their driving force from Day 1.

Bassignanis body was found on June 24, 1989, by a passing motorist. Her remains were found 10 to 20 feet off the roadway of Route 78, in a lightly wooded area near a small gravel pit about 1 miles south of the New Hampshire state line and about the same distance north of the entrance to Mount Grace State Forest. Sullivan said local and State Police responded to the scene, documenting evidence and collecting the remains.

First Assistant District Attorney Steven Gagne explained that identifying the victim is the first step in any homicide investigation.

From there, investigators can determine who the victims circle of family, friends and co-workers were and attempt to retrace their last known steps and contacts, he said. In this case, investigators were hampered from the start in their efforts to solve this homicide without an identification of the victim.

Article continues after...

Gagne said investigators have learned Bassignani got married and divorced in the 1940s before marrying William in 1945. Authorities have tracked down and spoken with the three grandchildren born to a son that has died, as well as a daughter who lives on the West Coast. Gagne said the DAs office was assisted by law enforcement in Washington state and in Hawaii.

So this investigation, which is now reinvigorated, has literally spanned half the globe, he said.

Gagne explained Bassignani and her husband lived in an apartment in Woonsocket, Rhode Island, approximately 80 miles from Warwick. The couple had previously lived in Franklin, Massachusetts. Sullivan said there is no known connection between the couple and Warwick, and it is unknown where the murder took place.

According to the DAs office, the victim was last seen alive on Memorial Day weekend in 1989. Gagne said William reportedly told relatives that his wife had decided to move back to Hawaii and that they would not be seeing or hearing from her again.

Gagne said he hopes this news provides some closure to the victims family, shedding some light on what may have been a looming cloud of doubt surrounding her disappearance for decades.

Retired Warwick Police Chief Brian Peters, who was at the departments helm in 1989, said it is refreshing to get some answers.

Its a big relief, he said. [We] never had anything like this happen in town, and hopefully never will [again].

Paul Marguet, the State Polices lead investigator on this case, said the victims grandchildren initially did not believe authorities, having long believed their grandfathers story about his wifes abrupt disappearance. But, he said, their DNA was used to confirm the truth.

Michael Vogen, an Othram representative, appeared via Zoom during Thursdays press conference and lauded the DAs office and the State Police before briefly speaking about his companys mission.

We were purpose-built to do just this, he said. Thats to generate human ID from forensic evidence.

Gagne previously told the Greenfield Recorder the decision was made to reach out to Othram due to the companys success in identifying the so-called Granby Girl as Patricia Ann Tucker, a 28-year-old woman found shot to death in 1978, but who went unidentified until a little over a year ago. Gerald Coleman, Tuckers husband when she died, is a person of interest in that murder. He died in state prison in 1996, and prosecutors say he never reported his wife as missing.

Othram also helped identify the Lady of the Dunes a 37-year-old woman found murdered in Provincetown on July 26, 1974 as Ruth Marie Terry. Her now-deceased husband, Guy Muldavin, was officially named as the killer in August 2023.

David Mittelman, founder and CEO of Othram, previously told the Recorder that his company tests DNA based on hundreds of thousands of markers, whereas the FBIs Combined DNA Index System (CODIS) uses 20. He also said victims are often not in CODIS because it was designed about 30 years ago to track the repeat offenses of known criminals. But Othram, which employs 60 people, can work from evidence generally considered unusable because it is too old or too degraded.

Gagne mentioned authorities hope to bring renewed attention to the Warwick case and trigger some new leads that have a domino effect that ends with additional answers. Anyone with information that might be helpful in this case is encouraged to call the State Police Detective Unit attached to the Northwestern District Attorneys Office at 413-512-5361. Messages can also be submitted anonymously through northwesternda.org.

Its like were trying to piece together an ancient puzzle here, Gagne told reporters, but any small piece would certainly help.

Reach Domenic Poli at: dpoli@recorder.com or 413-930-4120.

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1989 homicide victim found in Warwick ID'd through genetic testing, but some mysteries remain - The Recorder

Recommendation and review posted by Bethany Smith

WASHINGTON, May 2, 2024 /PRNewswire/ -- One of the biggest scams targeting Medicare beneficiaries in the last decade has been the genetic testing scam. Recently, the Senior Medicare Patrol (SMP) has seen an increase in genetic testing complaints. Across the nation, genetic testing company representatives are offering "free" genetic tests, also referred to as DNA screenings, cancer screenings, and hereditary testing, in exchange for the beneficiaries' Medicare numbers.

Beneficiaries are getting calls about genetic tests claiming that the results will help them avoid or detect diseases like cancer or Alzheimer's. The SMP has also received reports of genetic testing claims on Medicare statements when the beneficiaries never received any contact about genetic testing. It is incredibly important to review your Medicare statements and report these claims.

The genetic testing scam can be dangerous. "Scammers can steal people's medical identity and falsely bill Medicare (around $10,000 a claim), draining the Medicare program. Additionally, tests ordered under these circumstances could lead to confusion and inaccurate medical records," said Nicole Liebau, SMP Resource Center director.

The SMP recommends that Medicare beneficiaries:

The Senior Medicare Patrol (SMP) is ready to provide you with the information you need to PROTECT yourself from Medicare fraud, errors, and abuse; DETECT potential fraud, errors, and abuse; and REPORT your concerns. SMPs help educate and empower Medicare beneficiaries in the fight against health care fraud. Your SMP can help you with your questions, concerns, or complaints about potential fraud and abuse issues. It also provides information and educational presentations. To locate the local Senior Medicare Patrol, contact 1-877-808-2468 or go to http://www.smpresource.org.

SMP Resource Center Nicole Liebau 319-284-0702 [emailprotected]

SOURCE SMP Resource Center

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Double Helix Deception: The Return of Genetic Testing Scams in Medicare Fraud - PR Newswire

Recommendation and review posted by Bethany Smith

Finally, the moment arrived for my BRCA test a simple blood draw, almost anticlimactic for something that had the potential to be life-altering and life-saving. But how did I get here?

This moment had been something Id been building up in my head since 2017 when I saw The Bold Type and Jane Sloans personal journey as she navigated her mothers battle with breast cancer and her own subsequent decision to undergo genetic testing to look for BRCA genes. Now, here I was at my very own appointment in 2024. Sure, maybe it took me seven years but I was finally confronting, and struggling to manage, the overwhelming mix of anxiety, fear, grief, and empowerment I felt (but at least my therapist is fantastic). I was facing my own mortality squarely in the face and saying bring it.

So here I was, at a large hospital, as ready as I could be for my hour-long session where my family tree, personal history, and risks would be reviewed and evaluated. I was immediately struck by the warmth and empathy of my genetic counselor, whose name was Emily. She was kind and welcoming to both me and my partner, and never said any of the awkward veiled homophobia I usually hear in medical offices like is this your friend or worse, is this your sister. She was able to delicately balance the clinical aspects of genetics with the emotional weight of familial cancer history.

Most of my known family history and risk is on one half of my family, and the cancers that show up in my family tree can all be linked genetically. The counselor took all of this anecdotal information and plugged it into a mathematical formula. She calculated the risk that I would have a genetic mutation that is known to cause cancer. Even with only one half of my family history known to me, Emily confirmed my risk level was considered high enough to make me a strong candidate for testing.

It was strange hearing that out loud. I had already done my own research and knew that was likely to be the outcome, but having an expert confirm that I had a high risk level was both scary and exciting. I wanted to get the test, even if doing so seemed like asking a psychic when I was going to die.

Emily gave me information regarding my insurance coverage and financial support available through the genetic testing lab itself (not the hospital where I was doing the testing, but the organization that would actually analyze my genes). Fortunately, because of fairly recent health laws that require insurance to cover BRCA testing for high risk patients, my insurance would cover the test based on the fact that I was confirmed to be a good candidate.

Next it was time to consider how extensive I wanted the test to be, which required thinking about some of the most complex parts of the entire process: mastectomy, hormone therapy, hysterectomy, and more.

The genetic counselor was again fantastic and made sure that I didnt boil over like an out-of-control pot of water. She told me that the recommendations for medical intervention can vary based on the specific gene combination that is found. For example, a mastectomy might be a recommendation for a really high risk gene profile, but monitoring and hormone treatment might be the recommendation for a more moderate risk gene profile.

The question of whether or not to get a hysterectomy or mastectomy or even do hormone therapy is inextricably tied to notions of gender and what that means, personally and societally. I was first introduced to those conversations ten years ago, when Angelina Jolie revealed that she had gotten a mastectomy because of her own BRCA mutation. At the time it terrified me. It sounded like such an intense surgery and it was impossible for me, at the time, to separate breasts from femininity. The media confirmed this with countless op-eds at the time arguing whether or not Angelina Jolie was still a woman.

Of course, I no longer believe that breasts are necessary to be a woman. Trans women are women, even if they dont take hormones or get top surgery.I could still be a woman after a mastectomy. Tig Notaro, who I adore, went on stage shirtless r in her comedy special Boyish Girl Interrupted after a mastectomy because of breast cancer, and she identifies as a woman and a lesbian. Id also spent time as the primary caretaker for someone recovering from top surgery and had first hand knowledge about the bandages, drains, scars, and all of the details in between. Top surgery and a mastectomy arent exactly the same, but the surgeries have a lot in common, and my queer experience gave me a unique perspective to bear witness to the ways in which breast removal could be a joyful experience and not solely something done out of the fear and pain that comes with cancer. It helped me understand the surgery from a practical perspective, rather than the fear and tragedy that often accompanies a mastectomy in the case of cancer.

Hysterectomy and hormones are a little harder to comprehend, because they are not as visible and public. I also have endometriosis and the lesser-known adenomyosis, both of which mean that the lining of the uterus grows where they are not supposed to, meaning it is wildly painful and extraordinarily torturous to have a period. For me, a hysterectomy would address these conditions as well, making it feel easier to consider such a big surgery. Pair all of this with the BRCA statistics: A BRCA mutation can cause up to a 72% lifetime risk of developing cancer and a 44% lifetime risk of developing ovarian cancer. For those with BRCA who do develop cancer, it is much more likely to be aggressive and more likely to be deadly. I had already decided if I had high genetic risk, I wanted to take an aggressive approach to fighting and preventing it.

In fact, my coming to this specific hospital was the result of a lot of research to confirm that things like hysterectomy would be an option. Navigating the healthcare system as a disabled LGBTQ individual in a post-Roe and post-Trump America is far from straightforward, and I knew that it was possible that religious hospitals and state laws could affect what treatment options are available to me. Having received affirming care at One Medical, known for its LGBTQ+-friendly environment and inclusive practices, I was taken aback when I was first referred to Providence hospitals. Despite Providences outward portrayal of inclusivity, deeper research revealed instances of discriminatory practices, such as denying transgender individuals necessary medical procedures or restricting access to birth control and abortion based on religious doctrine. Choosing a hospital system became a key part of my process and I had to do extra work to ensure comprehensive gynecologic care options and avoid discriminatory practices. I didnt want to risk being denied all of the possible treatment options.

Now, sitting with Emily, I felt confident that I made the right decision. I wouldnt have to worry about artificial restrictions on my healthcare. Instead I could just consider the range of genetic test options knowing that even if the test revealed a high genetic cancer risk, this hospital had a team who would give me all of the options.

Finally, it was time to decide how many genes I actually wanted to have tested. They could perform a targeted test focusing on specific cancer-linked mutations or a comprehensive panel. Emily noted that one reason to choose more targeted testing was because going with the full panel increased the odds of finding an unknown genetic mutation and that this could cause anxiety. Scientific understanding of genes and cancer is still evolving, and some genetic variations have been identified, but not studied enough to conclusively say whether or not they increase the risk for cancer. However, she assured me that the testing company would provide ongoing updates, ensuring I remained informed as the science developed. I decided to go with the full, comprehensive panel of eighty eight genes, choosing to confront the unknown with as much data as possible. Id prefer to have all available information that science could offer me.

(Plus, the cost for the test would be the same whether I tested all eighty eight genes or a much smaller number, and I love a good bargain. Eighty-eight genes for the price of two, yes please!)

It feels like after all of the build up the actual genetic test should take place around a campfire with chanting and handholding, but the reality was anticlimactic and impersonal. If anything, the test itself felt really awkward. The nurse struggled to take my blood even though I am usually told by medical professionals that I have good veins. It felt like some part of my body was determined to express the anxiety I still felt by physically fighting back against the test. Finally after several minutes of poking at me the blood began to flow, they collected two small tubes, packaged it in a box to send to the lab, and I was finished.

As I left the appointment, I carried a sense of relief tinged with apprehension, knowing that the next few weeks would surely make my anxiety boil over.

But what convinced me to do the test in the first place remains true: if I have the genetic mutations that increase my risk for multiple types of cancer, especially breast cancer, theyre already there whether or not I get the test.

Now, as I await the verdict of eighty-eight potential cancer-causing genes, I am buoyed by the legacy of LGBTQ+ people who came before and whose courage and honesty helped bring awareness, empathy, and less stigmatization to breast cancer and the gender-based shame that can come with it. There is Wanda Sykes, who opted for a double mastectomy after finding what she called stage 0 cancer because she wanted to reduce her chance of it spreading as much as possible. Melissa Etheridge, who destigmatized the connection between breast cancer and femininity by performing bald at the GRAMMY Awards shortly after completing chemotherapy. Robin Roberts and her partner Amber Laig,n who both have had breast cancer and shared their experiences via Robins platform as a host on Good Morning America. Angelina Jolie, and her pivotal role in bringing BRCA genes and treatment options into the national spotlight. Audre Lorde, who published The Cancer Journals and detailed her own struggles with and views of post-cancer femininity.

I also still think of Tig Notaro, who in the middle of a stand-up comedy special being recorded for broadcast boldly unbuttoned her shirt, revealing her post-mastectomy chest. She did the remaining half of her stand-up set like this, forcing viewers to confront their own discomfort with cancer, mortality, gender, and health all in one subtle but significant move.

Tig stands on a stage for 20 minutes, literally in a spotlight and on camera, shamelessly showing her nipple-less, slightly concave chest with obvious red surgery scars. The first time I watched it I felt uncomfortable, being forced to face medical and gender stigmas at the same time while feeling amazed at Tigs boldness. It felt like I was watching something public that was supposed to be kept hidden and private and it was inspiring to see that see the mystery, and with it the stigma, stripped away. Its what I aim to do with this series in some small, similar way. I think perhaps if the worst case scenario for my health and my gender presentation is that I have something in common with Tig Notaro then maybe thats not so bad.

What to Expect Whenis a series from Katie Reilly shedding light on cancer and the intersection of genetics, identity, and health.

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What to Expect While Awaiting the Results of a Life Changing Test - http://www.autostraddle.com

Recommendation and review posted by Bethany Smith

SAN FRANCISCO and KYOTO, Japan, April 18, 2024 /PRNewswire/ -- Shinobi Therapeutics, a biotechnology company developing a new class of immune evasive iPS cell therapies, today announced a partnership with Panasonic Holdings Corp and Kyoto University's Center for iPS Cell Research and Application (CiRA). Through this strategic collaboration, the organizations aim to engineer a novel manufacturing platform to produce iPS-T cell therapies more efficiently and at lower cost than is possible with currently available technology.

"To make promising iPS-T cell therapies accessible to the broader population, Panasonic is committed to developing a manufacturing platform that will produce safe cells for therapies at the lowest possible cost," said Yuki Kusumi, Representative Director and President of Panasonic Holdings Corporation. "Reducing the production time and cost of cell therapies must be done in a manner that does not compromise safety or efficacy, and we are thrilled to see the Japanese biotech and engineering communities coming together to make that happen."

Cell therapies have shown remarkable promise in treating blood cancers and other intractable diseases, but manufacturing costs render these therapies inaccessible to many patients around the world. Shinobi's iPS-T cell technology, built upon a decade's worth of iPSC research pioneered at CiRA by Shinobi co-founder Shin Kaneko using iPSCs originally created by Nobel laureate Shinya Yamanaka, will be used to support the creation of a closed-system manufacturing device created by Panasonic, opening up an entirely new paradigm for cell therapy production.

"Advancements in iPS cell production and Shinobi's genetic modification of iPSCs for immune evasion have made regenerative T cell therapy increasingly feasible," said Shin Kaneko, Co-Founder at Shinobi. "The automated cultivation device developed in this joint research will significantly accelerate this, contributing to the realization of a world where state-of-the-art regenerative killer T cell therapy can be provided for every patient."

The newly announced partnership will leverage Panasonic's manufacturing expertise to develop a new method of producing iPS-T cell therapies in a closed-system process. The first phase of the partnership will be completed in April 2025, when the companies expect to release the initial prototype.

"While cell therapies have the potential to transform patient care across a wide range of intractable diseases, we have a long road ahead to overcome the challenges in manufacturability and accessibility," said Dan Kemp, CEO at Shinobi. "We are fortunate to be working with the most renowned partners across the academic and industry landscape as we endeavor to put cell therapies within reach for all patients who need them."

About Shinobi TherapeuticsShinobi Therapeutics is a biotechnology company developing a new class of off-the-shelf immune evasive iPSC-derived cell therapies. Based on the research of scientific co-founders Shin Kaneko, M.D., Ph.D., at Kyoto University and Tobias Deuse, M.D., at University of California, San Francisco, Shinobi has created a new allogeneic CD8a iPS-T cell platform that demonstrates comprehensive immune evasion from all arms of the immune system. For more information, please visit http://www.shinobitx.com.

Media Contact [emailprotected]

SOURCE Shinobi Therapeutics

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Panasonic and Shinobi Therapeutics Partner to Develop Efficient and Cost-Effective iPS Cell Therapy Manufacturing ... - PR Newswire

Recommendation and review posted by Bethany Smith

Visiongain has published a new report entitled Cell Therapy Technologies Market Report 2024-2034: Forecasts by Product (Sera, Media, Reagent, Cell Engineering Product, Cell Culture Vessels, Equipment, Systems and Software, Others), by Cell Type (T-Cells, Stem Cells, Other Cells), by Process (Cell Processing, Cell Preservation, Distribution, and Handling, Process Monitoring and Quality Control), by End-users (Biopharmaceutical & Biotechnology Companies, CROs, Research Institutes and Cell Banks) AND Regional and Leading National Market Analysis PLUS Analysis of Leading Companies AND COVID-19 Impact and Recovery Pattern Analysis.

The cell therapy technologies market is estimated at US$7,041.3 million in 2024 and is projected to grow at a CAGR of 10.7% during the forecast period 2024-2034.

The rise in chronic diseases like cancer, cardiovascular issues, and autoimmune disorders has created a pressing need for effective treatments. Supportive regulatory frameworks have encouraged the development & commercialization of cell therapies. Additionally, increased awareness and acceptance of these therapies among healthcare professionals and patients are driving demand further. Advancements in cell therapies offer lucrative opportunities for market players. Companies are focusing on enhancing the efficacy & safety of these therapies to provide better disease management outcomes for patients.

Download Exclusive Sample of Report https://www.visiongain.com/report/cell-therapy-technologies-market-2024/#download_sampe_div

How has COVID-19 had a Significant Impact on the Cell Therapy Technologies Market?

The COVID-19 pandemic has affected the market for cell therapy technologies market significantly. The pandemic initially caused significant disruptions to the manufacturing and supply chains of numerous industries, including the biotechnology sector. As a result, there were delays in cell therapy clinical trials, regulatory approvals, and commercialization initiatives. Furthermore, the shift in healthcare resources towards the management of the pandemic led to a reduction in funding and attention for medical research unrelated to COVID-19, such as the development of cell therapies.

However, the pandemic also made clear how crucial cutting-edge medical innovations like cell therapies are to solving the world's health crises. Consequently, there has been a surge in interest and funding for the study and advancement of cell therapy as a means of treating not only COVID-19 but also other chronic illnesses and infectious diseases. Additionally, the pandemic's adoption of telemedicine and remote monitoring has sped up the acceptance of decentralised clinical trials, which could advance cell therapy technologies by lowering trial costs and increasing patient access. The COVID-19 pandemic has, in the long run, created opportunities for innovation, collaboration, and growth, even though it initially presented challenges to the cell therapy technology market. The cell therapy sector is positioned to have a significant impact on how healthcare and illness management are provided in the future, even as the globe struggles to cope with the pandemic's aftermath.

How will this Report Benefit you?

Visiongains 305-page report provides 109 tables and 173 charts/graphs. Our new study is suitable for anyone requiring commercial, in-depth analyses for the cell therapy technologies market, along with detailed segment analysis in the market. Our new study will help you evaluate the overall global and regional market for Cell Therapy Technologies. Get financial analysis of the overall market and different segments including product, cell type, process, end-users and capture higher market share. We believe that there are strong opportunities in this fast-growing cell therapy technologies 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?

Rise in Prevalence of Chronic & Degenerative Diseases

The healthcare sector faces numerous challenges from chronic illnesses like cancer, heart disease, neurological ailments, and autoimmune disorders. The management or cure of many disorders is frequently only partially successful with conventional therapeutic options.

With the ability to replace, regenerate, or repair damaged tissues or organs, cell therapy presents a viable substitute. Much emphasis has been paid to cell treatments' capacity to treat diseases at their root and encourage long-term healing.

Notable advancements in cell treatment technologies have been made over time to address degenerative and chronic illnesses. For example, developments in stem cell research have made it possible to identify and isolate several types of stem cells, each with a unique therapeutic potential. In order to create novel cell-based therapeutics, researchers are looking into the utilisation of hematopoietic stem cells, induced pluripotent stem cells, and mesenchymal stem cells.

Rigorous Efforts by Companies Towards Development of Proprietary & Supportive Technologies Anticipated to Boost Industry Growth

In regenerative medicine, cell therapy, which employs living cells to treat or cure diseases, has emerged as a promising area of study. Nevertheless, the efficacy of cell therapies is contingent upon the accessibility of cutting-edge technologies that facilitate the production, characterization, and transportation of cells.

Significant investments are being made by companies in the cell therapy industry in research and development of proprietary technologies that improve the safety, effectiveness, and scalability of cell therapies. The technologies in question comprise an extensive array of domains, such as tools for cell characterization, cell isolation and expansion techniques, and cryopreservation methods.

The advancement of cell culture systems is a primary area of emphasis. Organisations are currently engaged in the development and refinement of culture media, growth factors, and bioreactors that establish an optimal milieu for cellular proliferation while preserving the viability and functionality of the cells. The primary objectives of these proprietary culture systems are to increase cell yields, decrease production expenses, and facilitate the scalable production of cell therapies.

Considerable interest is being devoted to supportive technologies that affect cell isolation and purification. Innovative methods are being developed by businesses to isolate particular cell populations from complex mixtures, thereby ensuring the quality and purity of cells used in therapies. These technologies reduce the possibility of contamination or undesired cell populations while facilitating the efficient isolation of therapeutic cell types.

Cryopreservation technologies are indispensable for the transportation and long-term storage of cells. Organisations are presently preoccupied with the advancement of cryopreservation techniques that preserve the genetic stability, viability, and functionality of cells throughout the freezing and thawing processes.

These developments guarantee the presence of viable cells during therapy administration, notwithstanding the logistical obstacles that may arise from cell storage and transportation.

The development of proprietary and supportive technologies will therefore likely contribute to the expansion of the global market for cell therapy technologies.

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

Where are the Market Opportunities?

Emerging nations present a substantial potential for the progression and integration of cell therapy technologies. These countries are currently experiencing notable advancements in their healthcare systems, as significant financial resources are being allocated to accommodate the growth of their populations. Concurrent with this growth, developing nations are confronted with an increasing prevalence of chronic and non-communicable ailments as a result of urbanisation, alterations in lifestyles, and the ageing of their populations. Cell therapy technologies are of particular relevance in these regions due to the innovative solutions they offer to address these urgent medical needs.

Moreover, in comparison to developed countries, the execution of clinical trials in emerging economies frequently demonstrates greater cost-effectiveness, predominantly attributable to reduced labour and operational expenditures. The financial benefits associated with this incentive motivate pharmaceutical companies and research institutions to investigate and advance cell therapies in these areas. Furthermore, numerous developing nations provide favourable regulatory structures and incentives in order to promote the progress and acceptance of cutting-edge medical technologies, such as cell therapies. The convergence of these elements renders developing nations an optimal setting for the proliferation and integration of cell therapy technologies, holding the potential to yield substantial advantages for healthcare providers and patients.

Competitive Landscape

The major players operating in the cell therapy technologies market are Thermo Fisher Scientific Inc., Novartis AG, Gilead Sciences, Inc., Merck KGaA, Danaher Corporation, Bristol-Myers Squibb Company, Sartorius AG, FUJIFILM Diosynth Biotechnologies, Lonza, GE Healthcare, Terumo BCT, Avantor, Inc., Bio-Techne Corporation, and Corning Incorporated among others. These major players operating in this market have adopted various strategies comprising M&A, investment in R&D, collaborations, partnerships, regional business expansion, and new product launch.

Recent Developments

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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 Therapy Technologies market is projected to grow at a CAGR of 10.7% by 2034: Visiongain - GlobeNewswire

Recommendation and review posted by Bethany Smith

NINA MOINI: The Minneapolis Saint Paul International Film Festival is currently under way. And tonight, a documentary will premiere called "Sequencing Hope." The film is directed by Lindsey Seavert and Maribeth Romslo. It follows an Alabama family who came to Minnesota to get their young daughter life-saving gene therapy for a rare disease. Let's listen to a clip from the trailer.

CELIA GRACE HAMLETT: Can you hold my hand?

[CHUCKLES]

They give me the medicine.

SUBJECT 1: There is lots of other gene therapy research on the horizon.

SUBJECT 2: That's got far-reaching consequences to move medicine forward.

SUBJECT 3: In my heart, I feel that the good Lord has something in store for Celia Grace.

SUBJECT 4: We just pray, Lord, for a miracle. We pray for a healing for Celia Grace.

SUBJECT 5: You know, when it comes to your kids, you're going to do whatever it takes to protect them.

SUBJECT 6: If it saves one child, then I feel like we have accomplished something.

NINA MOINI: Celia Grace Hamlett was four-years-old when she came to M Health Fairview Masonic Children's Hospital in 2021 and became the first person in the US to undergo the experimental gene therapy. Her family's in town for the film's premiere tonight and Celia Grace's dad, Gary, joins us now along with their doctor, Doctor Paul Orchard. Thank you both for being here.

GARY HAMLETT: Thank you.

PAUL ORCHARD: Thank you, Nina.

NINA MOINI: Yeah, and Gary, let me start with you if I might. Tell us about your daughter, Celia Grace. She's seven-years-old now and I understand she was diagnosed with this rare and often fatal genetic disorder, MLD, when she was diagnosed at three-years-old. What were her options at that point?

GARY HAMLETT: Well, at that point had one or had two options. One was bone marrow transplant or the other was gene therapy that was only being done in Milan, Italy.

NINA MOINI: Wow. Doctor Orchard, can you tell us all what MLD is?

PAUL ORCHARD: Certainly. Appreciate the opportunity to speak with you today. So metachromatic leukodystrophy is a rare inherited disorder. It's what we call a lysosomal disease. The lysosome is an organelle within cells that help break down materials that the cell is attempting to get rid of.

And there's a number of enzymes that are present in the lysosome that help accomplish that. Arylsulfatase A. is one of those. And in this circumstance, if you are unlucky enough to receive a mutation within the arylsulfatase gene from both mom and from dad, then you're affected with the disease.

But both parents who have one normal copy of the gene are absolutely fine. There's nothing to suggest that they have any sort of problem, but again, if you receive an abnormal copy from both parents then you see the disease. And in this situation, it's primarily a neurologic disorder. It occurs in kids as young as one or so in terms of manifestations of the disease, but it's progressive and lethal if there's no therapy.

NINA MOINI: Wow, that's just so much to take in, Gary. And you know, you mentioned having to maybe think about treatment over in Milan. How did you hear about the treatment for MLD that was right here in Minnesota?

GARY HAMLETT: Our doctor neurologist in Alabama, Doctor Matt, is the one that contacted us and said, what would y'all think if I told y'all y'all's daughter was going to make history books? At that point we said, what do you mean? And she said, well, your daughter may be the first child in the United States to receive gene therapy for MLD and it will be done at the Masonic Children's Hospital in Minneapolis under the care of Doctor Paul Orchard.

NINA MOINI: And then what did you think? I mean, were you going to have to pay for that?

GARY HAMLETT: Yes. At that point we didn't really care what it cost us being able to save our daughter's life. So our community started doing fundraisers to try to raise money to pay for this.

NINA MOINI: Wow, yeah. Doctor Orchard, can you explain how the gene therapy works and is it accessible to most people or is it just too costly?

PAUL ORCHARD: Well, the gene therapy clinical trials occurred in Europe, as Gary was alluding to, and the data was sufficiently positive that it was approved as therapy in the EU, essentially. So it's been licensed therapy there for several years, but none of the clinical trials have been done here in the US.

And because of the promise of this new therapy, we were gearing up to being able to offer this regardless, but there was the opportunity in this situation from Celia Grace's diagnosis to be able to intervene. So it's just become licensed therapy in the last month or so, as March 18th.

But prior to that and for Celia Grace, we had to petition the FDA to allow us to use it because it's still considered experimental therapy, and get all the approvals from all the various regulatory groups to be able to do that. So it took some time, but it opened the doors. And now we've treated a total of five patients with compassionate use therapy.

NINA MOINI: All right. Is it still pretty pricey, though? I understand it's among some of the priciest treatments.

PAUL ORCHARD: Yes, it is very expensive. So for the compassionate use treatment as an experimental therapy, the company actually donated the cell product, but it's millions of dollars now as licensed therapy.

NINA MOINI: Yeah. So still working to make it more accessible. Gary, you said something that really struck me in the trailer for the film. You said that you take care of people for a living. I understand you work in law enforcement, but you couldn't fix this for your daughter. And it seems like this film is really an exploration of your family's journey. Tell me how did that feel to feel sort of helpless in the moment, but then to see her go through this journey and be, I mean, cured?

GARY HAMLETT: We just felt very helpless, not knowing the outcome of it, how sick Grace was. Just thinking that we were going to lose our daughter. Possibly by the age of five-years-old.

NINA MOINI: Yeah.

GARY HAMLETT: And seeing her now as a normal seven-year-old, running, playing, is going to graduate in kindergarten, and it's just an amazing feeling.

NINA MOINI: Yeah, I'm sure. And so how is she doing? Tell us a little bit just about how she's getting around fine, and she's feeling well.

GARY HAMLETT: Oh, she is rambunctious, non-stop playing, running, doing her schoolwork. She is just like a typical seven-year-old little girl.

NINA MOINI: Yeah, and I understand some more patients are going to be undergoing that same treatment as Celia Grace, which is great news, Doctor Orchard.

PAUL ORCHARD: Yes, I hope it's going to be widely available. As you mentioned, the cost is going to be significant and attempting to determine how we're going to do this. The vast majority of these patients that we treat are obviously not from Minnesota.

And so being able to get insurance that's going to work across state lines and going to be sufficient for this is going to be a challenge. But that's one of the things that we're currently working on.

NINA MOINI: OK, and Gary, I'll leave the last question for you here. What do you hope people will take away from watching your family's story in this documentary?

GARY HAMLETT: The struggles of not knowing the outcome of your child. The struggles of possibly knowing that you will only have a few years with your child. And then knowing that there are people out there willing to help and willing to do anything possible to save your daughter or your son. I just can never repay everybody that along this journey for what they have done for my child.

NINA MOINI: Yeah, and it really sounds like it's some of the best parts of humanity and also some of the hardest struggles that anyone will go through. Thank you so much for sharing that journey and for being here, Gary. And to you as well, Doctor Orchard, thank you, and congratulations on this film reaching an audience today.

PAUL ORCHARD: Thank you.

GARY HAMLETT: Thank you so much.

NINA MOINI: Gary Hamlett is the father of Celia Grace Hamlett and Doctor Orchard is a pediatric blood and marrow transplant physician at M. Health Fairview. Both are featured in the documentary, "Sequencing Hope," which is premiering tonight at 7:00 PM. We'll have that information on our website, mprnews.org.

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Recommendation and review posted by Bethany Smith

Liam Shore is a third-year researcher at the University of Liverpool, in the Department of Philosophy. His research interests fall within the domain of ethics, notably on the ethics of digital and biotechnologies.

The Making of a Philosopher

Im a philosopher, but I havent always been one, so how does someone become a philosopher? And more fundamentally, why would anyone want to become one?

As a rare vocation, youd be forgiven for supposing that philosophers are an extinct species who once roamed the Athenian plazas during early antiquity, gesticulating poignantly and wearing togas. Well, happily they do exist today, sans the togas, largely unnoticed, behind the scenes on ethics boards, or engaging in fundamental first-principles critiques of.well.everything.

A question arises: if philosophers critique everything, how do they develop knowledge to criticise specialist areas? This becomes particularly poignant in an applied ethics context. My own personal journey, from Technologist to Philosopher, shows that one practically needs to be educated in two disciplines to become a bona fide philosopher.

When deciding what subjects to study, and what career to pursue, I was torn between multiple strong interests. In third place, Technology; in second place, Medicine; and in first place, Philosophy. In my case, I took the reverse path toward becoming a philosopher. Namely, I studied technology, worked in the biological sciences industry, and returned to academia with domain-specific expertise to enter into the philosophy sub-field of ethics. The beauty of philosophy for me, and the reason why I personally had the desire to pursue becoming a philosopher, is that philosophy, being able to critique everything, can powerfully converge disparate interests. It is this quality that made philosophy my first love, and so my PhD journey began, delving into the ethics of radical life extension.

Understanding Rejuvenation Biotechnologies

Recently, breakthroughs in rejuvenation biotechnologies, particularly those of the Strategies for Engineered Negligible Senescence (SENS) variant, have garnered little attention, and yet constitute steps towards a paradigm-altering event. SENS therapies, like maintaining classic cars to prolong their lifespan, seeks to do the same for our bodies as we age. SENS suggests that ageing is caused by the accumulation of cellular and molecular damage throughout the body over time, and advocates posit that by repairing or reversing this damage, it is possible to rejuvenate tissues and organs, thereby extending a persons healthy lifespan. Ultimately, by seeking to tackle age-related diseases at their root, via interventions such as stem cell therapies, the aim is to bring age-related diseases fully under comprehensive medical control. Overall, the eventual aim of SENS is to combine a panel of these therapies to combat all preceding causes of age-related diseases, and consequently, tackle ageing itself!

Although this sounds futuristic, there are therapies in various stages of development, with the furthest along being in clinical trials. Advocates claim that these therapies could, in due course, function well enough to rejuvenate a persons body to a youthful state. In effect, this is a process that, amongst other things, removes damage and replaces cells, enabling the body to regain a healthy condition. The outcome of extending good health is that it prolongs life, as it postpones the onset of age-related diseases until higher chronological ages. Accordingly, if someone repeatedly receives these therapies throughout life, this could constitute a potentially radical life-extending situation, as periods of poor health may be postponed repeatedly, allowing one to maintain optimal physiological functioning for longer, thereby delaying death itself!

A Case for Philosophical Inquiry The SENS approach to rejuvenation biotechnologies represents a bold vision for extending healthy lifespans and combating age-related diseases. However, realising this vision requires careful consideration of the ethical implications of extending human lifespan, making the SENS approach a question for philosophical research.

The most common ethical concerns for life-extending technologies are Health Equity i.e. fairness in health opportunities for all; Longevity/Population Dynamics i.e. understanding how long people live & how populations change; Environmental Impacts i.e. the effects of human activities on nature and Informed Consent/Autonomy i.e. respecting peoples right to make their own decisions.

Nevertheless, although important, these concerns dont engage with how this technology impacts what we find meaningful at the profoundest level as human beings. However, my research incorporates all the aforementioned ethical concerns and delves deeper into the realms of identity, purpose, and meaning in life, primarily through an existentialist lens.

Existentialism, as a philosophical theory, concerns itself with questions of: the nature of individual existence, authenticity of self, human freedom, and the search for purpose/meaning in life. It is via this prism that Im currently defining a taxonomy of values supported by radical life extension advocates, with this taxonomy categorising virtues like fairness, compassion, and autonomy, providing a structured framework for ethical analysis. In addition, Im exploring how a SENS-induced radically extended life may impact what we value. And next, I plan to explore whether the consequences of SENS therapies could result in mental ageing, in essence a feeling of listlessness, a sense of ennui, or a notion of world-weariness.

Overall, I hope that my research will deliver original insights to help us work towards a future where radically extended healthspans are possible, while fully prioritising and ensuring human well-being.

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Becoming an Expert: Exploring the Ethics of Radical Life Extension - News - University of Liverpool - News

Recommendation and review posted by Bethany Smith

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Genetically engineered B cells could produce super-antibodies to HIV

Researchers at Washington University School of Medicine in St. Louis have received a $6.2 million grant from the National Institutes of Health (NIH) to develop a gene therapy that would modify the immune systems B cells to spur them to produce broadly neutralizing antibodies against HIV. In theory, such an approach could control or eliminate the infection without need for ongoing antiretroviral therapy. Shown is the engineered adenovirus designed to deliver HIV superantibody genes into B cells.

HIV infections can be controlled with medication, but such therapy must continue throughout patients lives because no strategy exists to eliminate the virus from the body or control the infection without ongoing treatment.

With the aim of developing such a strategy, researchers at Washington University School of Medicine in St. Louis have received a $6.2 million grant from the National Institutes of Health (NIH) to develop a gene therapy that would modify the immune systems B cells to spur them to produce broadly neutralizing antibodies against HIV. In theory, such an approach could control or eliminate the infection without need for ongoing antiretroviral therapy.

Permanent ways to control or eliminate HIV infection remain elusive, and their development is a major goal of the field, said David T. Curiel, MD, PhD, the Distinguished Professor of Radiation Oncology. The idea of modifying B cells which naturally produce antibodies to ensure that they manufacture specific antibodies that are broadly effective at targeting HIV is an exciting strategy. We have brought together a great team with expertise in HIV, gene therapy, and animal models of infection to work toward this goal.

Curiels co-principal investigators are Michael R. Farzan, PhD, of Harvard Medical School and Boston Childrens Hospital, and Mauricio de Aguiar Martins, PhD, of the University of Florida.

Over the decades since HIV appeared, researchers have learned that about 1% of people with the virus are able to produce what might be considered superantibodies against the virus. Such individuals known as elite neutralizers can produce antibodies against multiple strains of HIV.

Some people naturally have antibodies that can bind and destroy or deactivate very diverse strains of HIV, and we now have the ability to build those types of antibodies in the lab, said Paul Boucher, a doctoral student in Curiels lab. But just giving other patients these superantibodies is not an ideal solution, because these proteins would stay in the body only temporarily. Instead, our approach is to genetically modify the cells responsible for making antibodies the immune systems B cells so they can always produce superantibodies against HIV whenever they may need to.

Such engineered B cells could create, in theory, a state of permanent vaccination against the virus. Even if such a gene therapy doesnt fully clear HIV from the body, the strategy could allow the amount of virus in the body to be controlled, keeping it at a minimal level and creating a functional cure, according to the researchers.

The strategy involves modifying a different type of virus, called adenovirus. When used in gene therapy, such viruses are genetically disabled so they cant cause disease. The researchers then could engineer the adenovirus to carry the gene responsible for manufacturing broadly neutralizing antibodies to HIV. In the same viral vector, they also could include genes responsible for manufacturing the CRISPR/Cas9 gene editing proteins. In this way, the gene therapy delivery vehicle would carry into the body both the antibody gene that will be edited into the B cell genome and the genes to build the molecular tools to carry out that editing.

Using a three-part targeting strategy, the researchers would design the adenovirus to deliver its genetic payload only to B cells, avoiding other cell types. They have developed ways to modify the virus so that it is targeted directly to a protein that is expressed on the surface of B cells and no other cell types. The researchers can further restrict the targeting by using genetic methods to ensure that the CRISPR/Cas9 proteins can only be manufactured when their genes are delivered into B cells. Finally, they have developed strategies to modify the adenovirus in a way that stops its natural tendency to accumulate in the liver.

This strategy to modify B cells is distinct from another adenoviral gene therapy approach to HIV treatment that is currently in clinical trials led by principal investigator Rachel M. Presti, MD, PhD, a professor of medicine in the Division of Infectious Diseases at Washington University School of Medicine. HIV is difficult to eliminate from the body because the virus integrates its genome into the DNA of the infected individuals T cells. The strategy currently in clinical trials is focused on using precise targeting of the CRISPR/Cas9 gene editing proteins to excise the virus from the genomes of all of a patients infected T cells. This strategy is being tested in a first-in-human, phase 1 clinical trial to determine its safety and preliminary efficacy at various doses.

Curiel said engineered B cells are ripe for developing new therapies to treat a wide variety of diseases. In November, a genetically engineered B cell therapy was administered to a patient for the first time at the University of Minnesota Medical Center. In that case, the therapy was designed to treat mucopolysaccharidosis type 1, a life-threatening condition in which the body lacks an enzyme necessary to break down large sugar molecules inside cells.

Gene therapy with engineered B cells is an exciting new area of research, Curiel said. We look forward to combining our expertise in adenovirus gene therapy, HIV infection and preclinical models of disease to realize our plan for developing an HIV therapy that we hope can permanently control the infection.

This work is supported by the National Institute of Allergy and Infectious Diseases (NIAID) of the National Institutes of Health (NIH), grant number 1R01-AI174270-01A1. This content is solely the responsibility of the authors and does not necessarily represent the official views of the NIH.

About Washington University School of Medicine

WashU Medicine is a global leader in academic medicine, including biomedical research, patient care and educational programs with 2,900 faculty. Its National Institutes of Health (NIH) research funding portfolio is the second largest among U.S. medical schools and has grown 56% in the last seven years. Together with institutional investment, WashU Medicine commits well over $1 billion annually to basic and clinical research innovation and training. Its faculty practice is consistently within the top five in the country, with more than 1,900 faculty physicians practicing at 130 locations and who are also the medical staffs of Barnes-Jewish and St. Louis Childrens hospitals of BJC HealthCare. WashU Medicine has a storied history in MD/PhD training, recently dedicated $100 million to scholarships and curriculum renewal for its medical students, and is home to top-notch training programs in every medical subspecialty as well as physical therapy, occupational therapy, and audiology and communications sciences.

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$6.2 million to help develop gene therapy for HIV Washington University School of Medicine in St. Louis - Washington University School of Medicine in...

Recommendation and review posted by Bethany Smith

A type of gene therapy called CAR-T that has extended survival for thousands of patients with leukemia and other blood cancers is being adapted at UC San Francisco to treat people with glioblastoma, the most common and deadly adult brain tumor.

This new more powerful version of CAR-T employs a novel technology developed at UCSF called synthetic notch (synNotch) that both protects healthy tissue from damage and enables the treatment to work more effectively.

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Approximately 12,000 Americans are diagnosed each year, with an average survival of just 15 months.

UCSF opened enrollment this week for a clinical trial that is using the technology for the first time in people. A second trial, also at UCSF, is slated for 2025.

Approximately 12,000 Americans are diagnosed each year with glioblastoma. Patients survive on average for just 15 months after their diagnosis, and new treatments are urgently needed.

This project is a prime example of bench-to-bed translation within UCSF, representing the strengths in basic and clinical science, said Hideho Okada, MD, PhD, a physician-scientist and director of the UCSF Brain Tumor Immunotherapy Center. We have a truly home-grown project here.

Okada has received up to $11 million for the first trial from the California Institute for Regenerative Medicine (CIRM), which funds stem cell and gene therapy research for incurable diseases and disorders through all stages of clinical trial development.

Initial funding for the second trial is provided by the National Cancer Institute Specialized Programs of Research Excellence (NCI SPORE).

We hope that the treatment will prolong lives for patients with glioblastoma, said Okada, who is a professor of neurosurgery at UCSF and a member of the Weill Institute for Neurosciences. However, the primary goal of the current phase 1 study is to ensure safety and characterize any toxicities.

When tested in mice, Okada said the therapy provided a robust and long-lasting result that was more remarkable than anything he had encountered during 30 years of brain tumor research.

The CIRM-funded trial will be led by principal investigator Jennifer Clarke, MD, MPH. It is open to patients with newly diagnosed glioblastoma, who have completed standard-of-care treatment. Tumors must have a mutation found in approximately 20% of glioblastomas, and that can be identified by the UCSF500 cancer gene panel test.

The second study will be open to glioblastoma patients whether or not they have the mutation.

CAR-T refers to chimeric antigen receptor T-cells, which are cancer-killing immune cells that have been extracted from the patient and genetically modified to recognize and destroy antigens that appear on the surface of cancer cells. These supercharged CAR-T cells are then infused back into the body to attack tumor cells.

For many patients with leukemia and other blood cancers, CAR-T has demonstrated long-term remission, but the approach hasnt worked against brain tumors. Glioblastoma cells are more diverse than blood cancer cells, and they can evade CAR-T. Many of the antigens made by the tumors are also found in healthy tissue, leaving them open to attack.

To overcome these obstacles, Okada drew from the synNotch system developed by Wendell Lim, PhD, director of the UCSF Cell Design Institute and professor in the UCSF Department of Cellular and Molecular Pharmacology.

The technology allowed scientists to program CAR-T cells to target specific antigens on tumor cells, without touching those found in healthy tissue. They also do not succumb to T-cell exhaustion, a common problem with CAR-T therapies, because they are more metabolically stable and use less energy to fight cancer longer.

Weve created a system that is flexible and thorough and addresses the major concerns weve had about using CAR-T cells against solid tumors, Lim said. These cells act like computers: integrating multiple units of information and making complex decisions.

About the California Institute for Regenerative Medicine (CIRM): AtCIRM, we never forget that we were created by the people of California to accelerate stem cell treatments to patients with unmet medical needs, and act with a sense of urgency to succeed in that mission. To meet this challenge, our team of highly trained and experienced professionals actively partners with both academia and industry in a hands-on, entrepreneurial environment to fast track the development of todays most promising stem cell technologies. With $5.5 billion in funding and more than 150 active stem cell programs in our portfolio,CIRMis one of the worlds largest institutions dedicated to helping people by bringing the future of cellular medicine closer to reality.

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Gene Therapy Is Halting Cancer. Can It Work Against Brain Tumors? - UC San Francisco

Recommendation and review posted by Bethany Smith

Gene therapy research is booming. Since the U.S. Food and Drug Administration (FDA) issued its first approval for a gene therapyin 2017, oncology researchers have been breaking barriers in gene therapy trials, followed by an explosion in mRNA research during the COVID pandemic. Today, this trailblazing science is providing new ways to approach rare diseases and new hope when other investigational interventions have failed. In fact, themajorityof approved gene therapies are for rare diseases 14 are currently in Phase III trials for 10 rare diseases and 45 gene therapies are in early stages of development to treat 30 rare diseases. We see great potential for gene therapies, said Leslie Johnston, senior vice president of biotech delivery for Parexel. As more products are approved, it will gain traction and more companies will look to expand their therapies into other therapeutic indications. This progress presents tremendous potential to change more patients lives across many different diseases. This could be gene therapys moment. But to fully seize it, the industry must clear some complex hurdles. Gene therapies pose several unique challenges for clinical research, including ethical and safety considerations, regulatory hurdles, precarious logistics, and potentially staggering costs. These challenges may already be having ramifications: New U.S. patients treated with gene therapies approved or in development areexpected to fallby one-third from 2025 to 2034. The key to clearing these hurdles? Cooperation between sponsors, sites, regulators, patients, and other stakeholders is essential to expediting the advancement of life-saving gene therapies. Regulators should address risks without limiting innovation Gene therapy trials are strictly regulated and rightly so, due to the novel nature of the intervention and the potential long-term consequences. Gene therapy interventions also carry inherent safety risks, including the potential for unintended genetic changes or adverse immune reactions. Ensuring patient safety requires rigorous monitoring and adherence to strict protocols. However, obtaining regulatory approval under these conditions is time consuming and resource intensive. To avoid hampering scientific progress, regulators should aim to ensure that requirements are appropriately rigorous without being unmanageably onerous. Thankfully, the FDA is paying close attention to gene therapy and has demonstrated a desire to work with drug developers toward the success and approval of these treatments. Dr. Peter Marks, Director of the Center for Biologics Evaluation and Research (CBER) at the FDA, has expressed his hope for an exponential, if not logarithmic, increase in gene therapy approvals. There is a lot of excitement that this could potentially make a big difference for the treatment of human disease, said Dr. Marks in hisremarksto the National Press Forum last November. The FDA is going beyond mere rhapsodizing and taking action to accelerate gene therapy. Last year, the agencylaunched a pilot programcalled Support for Clinical Trials Advancing Rare Disease Therapeutics, or START. This program is designed to accelerate the development and approval process for treatments targeting rare diseases by providing regulatory guidance, assistance, and incentives to sponsors conducting clinical trials in this field. The program represents an important step forward in fostering innovation and collaboration between regulatory bodies and sponsors. In addition, the FDA is working toharmonize global requirementsfor the review of gene therapies. Encouraging and facilitating international cooperation and harmonization of regulatory standards including mutual recognition agreements and shared regulatory pathways for multinational clinical trials can help streamline gene therapy development globally and help bring innovations to patients faster. Even with this progress, regulators should continue to help accelerate gene therapy research by streamlining regulatory pathways specifically tailored to gene therapies. This means providing clear guidance on requirements for preclinical and clinical development, fostering collaboration between stakeholders to share knowledge and best practices, and offering expedited review processes for gene therapy products aimed at treating serious or life-threatening diseases. With a staggering2,500 cell and gene therapyinvestigational new drug applications (INDs) on file, the FDA approved justfivecell and gene therapies in 2023. Dr. Marks hassuggestedthat accelerated approval, which has successfully advanced cancer and HIV/AIDS treatments, may be the most appropriate path for this new category of treatments. But, regulators also need to commit to proactively partner with developers to understand the patient population and the risks and benefits of each new therapy. Likewise, researchers, industry stakeholders, and patient advocacy groups should engage with regulators to help them understand the unique challenges and opportunities in the field of gene therapy. This can help regulators adapt regulatory frameworks to ensure patient safety while expediting the development and approval of promising treatments. Sites and sponsors must be prepared Of course, sites and sponsors also have a crucial part to play in advancing this promising field of medicine. Clinical trial sites should enhance their capacity to conduct gene therapy trials safely and effectively and sponsors should do their part to assist sites in these efforts. By working closely with clinicians and regulators, sponsors can ensure that the trial development process aligns with clinical needs and regulatory standards. Sponsors should have a thorough understanding of FDA requirements pertaining to design, preclinical testing, and long-term follow-up. Better alignment from the outset will lead to more efficient trial designs, faster regulatory approvals, and ultimately quicker patient access to therapies. For example, sponsors working with mRNA and other genetically engineered therapies in North America not only have to go through institutional review board (IRB) review, they also have to navigate additional requirements from the U.S. National Institutes of Health (NIH) Office of Science PolicyGuidelines for Research Involving Recombinant or Synthetic Nucleic Acid Molecules(NIH Guidelines). These requirements usually involve an additional biosafety risk assessment review from an institutional biosafety committee (IBC) in addition to IRB review. NIH Guidelines apply for any research involving recombinant or synthetic nucleic acids (e.g. genetically engineered materials) that receives NIH support or takes place at sites that have received NIH support for such research. Even when there is zero NIH support, IBC review is considered a best practice. IBC review and inspection helps sites ensure they are fully prepared by identifying areas for improved biosafety protections and calling out gaps in current standard operating procedures (SOPs). Proactive coordination and integration of these separate review processes can speed trial timelines and help sponsors consistently address any potential concerns or issues. Sites can also be better prepared by pre-registering an IBC. The NIH takes six to eight weeks or more to approve a new registration, in addition to IBC review time so by registering an IBC before they even have a trial, sites can save a month or more in startup time over a site that waited to register. Clinical trial sites looking to host gene therapy studies must be prepared in other ways, as well, both in terms of knowledge and infrastructure. Gene therapy studies require specialized infrastructure for manufacturing, storing, and administering genetic material to adhere to strict biosafety guidelines. Something as simple as having an upholstered chair in the infusion room which would pose an unacceptable contamination risk if genetic materials were to spill would require the site to rethink their current processes. Rigorous training is also key due to the added risk of spreading genetic material to caregivers and others in close contact with patients. Research staff must be specially trained to handle, deliver, and dispose of this material safely. Of course, these measures can seem intimidating for sites that are already cost-constrained. Large academic medical centers with more resources and experience are more likely to be well-positioned for these studies. For instance, they may already have conducted bench, animal, and/or agricultural research with genetic engineering or have the funding to make any needed adjustments such as purchasing special equipment. But to maximize the potential number of sites where this research can be conducted and therefore reach more potential participants sponsors might consider providing help in the form of financial assistance, training curricula, SOP guidance, and more to smaller sites seeking to conduct gene therapy research. Logistical complexities depending on the investigational medicine and therapeutic area are among the most complicated challenges in gene therapy trials, added Johnston. From collecting the specimen from the patient, modifying it, storing it, transporting it, and then returning it back to the patient all comes with tremendously unique logistical challenges and requires equally unique equipment, technology, and expertise. And it can be cost-prohibitive. Patients must be fully on board Of course, the most essential stakeholder in any clinical trial is the patient. In gene therapy research, which can be particularly demanding, patients must have a complete understanding of and commitment to their involvement. Understanding the potential risks and benefits can help patients make informed decisions and navigate the study process. First, it's crucial for patients to adhere strictly to the protocol provided by the clinical trial team, including following medication schedules, maintaining specific hygiene practices, and attending all study visits. They should strive to maintain optimal health to enhance the body's response to gene therapy. And to avoid delays, patients should maintain open and honest communication with the clinical trial team, reporting any changes in symptoms, side effects, or general health as soon as they occur. Trial participants also need to be in it for the long haul. Because gene therapy interventions aim to produce lasting effects, even cures, they typically require long-term patient follow-up to assess efficacy and safety. But they may also need to have incredible patience. Johnston explained, There are many complexities that can impact study progress. For example, unpredictable logistical challenges like a weather event or vehicle accident could delay a temperature-sensitive delivery to a site, or data review outcomes could require an indeterminate pause period. Patience and agility are must-haves, but it is difficult for patients potentially depending on this new therapy to save or change their lives. Lastly, the industry cannot forget the patient. Involving patients and patient advocacy groups in the regulatory process can help ensure that the development of gene therapies is aligned with patient needs and priorities, as well as shed light on risk-benefit perspectives from a patients viewpoint. The more these perspectives are considered from the beginning, the greater the chance of a trials success. Rita Naman, co-founder of the Mighty Milo Foundation, emphasizes the need for a more collaborative and patient-centered approach to gene therapy development. "For ultra-rare diseases likeSPAX5, gene therapy offers a glimmer of hope where traditional treatments do not. But logistical hurdles make these therapies expensive and inaccessible, explained Naman. Closer collaboration with patients, industry, and regulators could streamline these processes, drive costs down, and speed trials. Patients like my son, and their caregivers, plus advocacy groups should be invited into the earliest discussions to prevent false starts or missed milestones in gene therapy development especially as the patients priorities dont always line up with the sponsors. In the fight for gene therapy breakthroughs, cooperation is key. The road to operationalizing gene therapy clinical trials is laced with land mines and potholes. To capture the full potential of novel gene therapy research, a new level of collaboration between sponsors, CROs, sites, oversight committees, regulatory bodies, and patients is paramount. Patients want access to novel gene treatments, and they want it fast. Sponsors want to deliver but fight logistical and financial obstacles. Regulators want to ensure safety first, especially considering such new, promising science, concluded Johnston. These three goals may seem conflicting at times, so we need to strike a balance of safety and speed, so patients dont miss their only potential treatment opportunity. A seasoned industry veteran with more than 25 years of experience, James Riddle is senior vice president of global review operations at Advarra. Riddles expertise includes large program management and growth, operational processes, development and implementation of technology solutions, and management of large Human Research Protection Programs (HRPP), Biosafety programs (IBC) and Institutional Animal Care and Use programs (IACUC). Riddle has directed numerous clients in achieving Part 11 compliance and meeting computer system validation requirements.

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Gene Therapy is Having its Moment: Can the Clinical Research Ecosystem Seize It? - Contract Pharma

Recommendation and review posted by Bethany Smith

One of their biggest successes uses gene therapy to treat a rare genetic disorder called aromatic L-amino acid decarboxylase (AADC) deficiency.

Children with AADC deficient are missing the enzyme that produces dopamine and serotonin in the central nervous system. This affects pathways in the brain responsible for motor function and emotions.

As a result, these children cant coordinate the movements of their head, face and neck. They often dont reach normal childhood milestones, such as sitting up or walking by themselves.

Along with her mother, Arcelia Ramirez, they traveled 800 miles from their home near Omaha, Neb., so that Delilah could have this life-changing gene therapy surgery at Ohio State Wexner Medical Center.

But now, Delilah has changed so much for the better. On her 9th birthday, she blew out a candle on her cupcake on purpose. This was the first time she had ever blown out a birthday candle.

She's like a different kid. Her sleeping is a lot better. She can walk now, she can self-feed, said Arcelia Ramirez. When she started using a fork, that was a reason to celebrate. When she started using a straw, that was a reason to celebrate. Walking was a big, big milestone for her that we just celebrated.

So we are bringing in a correctly spelled sequence of the gene, said Bankiewicz, who is also chief scientific officer at the Ohio State Gene Therapy Institute.

This helps ensure we put the genetic material in exactly the right place, so the brain will start making dopamine and serotonin again, said Elder, who also is a professor of neurological surgery. This

therapy is designed to approach both parts of the brain that control movements and emotions.

This breakthrough in treating patients with AADC was decades in the making.

It requires a use of the technology and devices that we had to develop and establish over the years to do these surgeries very precisely, very carefully and then do it safely, Bankiewicz said. The issue of, Is it going to work? It's no longer being questioned. It works.

In addition to expanding this method to central nervous system diseases such as Alzheimers, Parkinsons, Multiple System Atrophy and Huntingtons disease, Elder and Bankiewicz are also trying to edit genetic mutations in other neurological disorders, including brain tumors.

We are not treating a gene that causes Parkinson's or Alzheimer's, Bankiewicz said. We're using this technology to deliver a therapeutic that we believe will, in a positive way, affect the progression of the disease.

# # #

Media Contact: Eileen Scahill, Wexner Medical Center Media Relations, Eileen.Scahill@osumc.edu

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The future of gene therapy has arrived, and it's changing lives - Wexner Medical Center - The Ohio State University

Recommendation and review posted by Bethany Smith

The global gene therapy market size was valued at USD 8.75 billion in 2023 and is poised to grow from USD 10.47 billion in 2024 to USD 52.40 billion by 2033, growing at a CAGR of 19.6% in the forecast period (2024-2033).

Gene therapy is a technique that uses a gene to treat, prevent or cure a disease or medical disorder. Often, gene therapy works by adding new copies of a gene that is broken, or by replacing a defective or missing gene in a patients cells with a healthy version of that gene. Both inherited genetic diseases (e.g., hemophilia and sickle cell disease) and acquired disorders (e.g., leukemia) have been treated with gene therapy.

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The development of the market is owing to an increase in the number of gene therapy-based discoveries, increasing investment in this sector, and rising approval of gene therapy products. According to the WHO, 10 to 20 new cell and gene therapies are expected to be approved each year by 2025.

Continuous developments in recombinant DNA technology are anticipated to enhance the efficiency of gene therapy in the coming years. Hence, ongoing progresses in recombinant DNA technology are anticipated to expand the number of ongoing clinical trials for gene therapy. Primarily, these advancements are taking place in the context of various gene-editing tools and expression systems to augment the R&D for products. The advent of CRISPR/Cas9 nuclease, ZFN, and TALEN allows easy & precise genome editing. As a result, in recent times, the gene-editing space has witnessed a substantial number of research activities, which, in turn, is expected to influence the growth of the gene therapy market.

The growth of the gene therapy market is expected to be majorly benefitted from the increasing prevalence of cancer. The ongoing increase in cancer patients and related death per year emphasizes the essential for the development of robust treatment solutions. In 2020, there were around 18.1 million new cases of cancer worldwide. 9.3 million of these cases involved men, while 8.8 million involved women. Continuing developments in tumor genetic studies have delivered substantial information about cancer-related molecular signatures, which in turn, is expected to support ongoing clinical trials for cancer therapeutics.

With rising demand for robust disease treatment therapies, companies have focused their efforts to accelerate R&D for effective genetic therapies that target the cause of disease at a genomic level. . Furthermore, the U.S. FDA provides constant support for innovations in this sector via a number of policies with regard to product manufacturing. In January 2020, the agency released six final guidelines on the manufacturing and clinical development of safe and efficient products.

Furthermore, facility expansion for cell and gene therapies is one of the major factors driving the gene therapy market growth. Several in-house facilities and CDMOs for gene therapy manufacturing have begun investing to enhance their production capacity, which, in turn, is anticipated to create lucrative opportunities for market players. For instance, in April 2022, the FDA approved commercial licensure approval to Novartis for its Durham, N.C. site. This approval permits the 170,000 square-foot facility to make, test, and issue commercial Zolgensma, as well as manufacture therapy products for current & upcoming clinical trials.

Cell and Gene Therapy Market :https://www.biospace.com/article/releases/u-s-cell-and-gene-therapy-clinical-trial-services-industry-is-rising-rapidly/

Gene Therapy Market Report Highlights

U.S. Gene Therapy Market Size in U.S. 2024 to 2033

The U.S. gene therapy market size was estimated at USD 3.19 billion in 2023 and is projected to surpass around USD 18.50 billion by 2033 at a CAGR of 19.22 % from 2024 to 2033.

North America dominated the market in 2023 with the largest revenue share of 65.12% in 2023. This region is expected to become the largest routine manufacturer of gene therapy in terms of the number of approvals and revenue generated during the forecast period. Increasing investments in R&D from large and small companies in the development of ideal therapy drugs are anticipated to further boost the market.

Furthermore, the increasing number of investments by the governments and the growing prevalence of targeted diseases are the factors fueling the market. According to the Spinal Muscular Atrophy Foundation, in 2020, around 10,000 to 25,000 children and adults in the U.S. were affected by spinal muscular atrophy, making it a fairly common disease among rare diseases.

Europe is estimated to be the fastest-growing regional segment from 2024 to 2030. This is attributed to its large population with unmet medical needs and increasing demand for novel technologies in the treatment of rare but increasingly prevalent diseases. Asia Pacific market for commercial application of genetic therapies is anticipated to witness significant growth in the forecast period, which can be attributed to the easy availability of resources, local presence of major companies, and increased investment, by the governments.

UK Gene Therapy Market

The UK gene therapy market is anticipated to witness accelerated growth over the forecast period, due to increased investments by various big companies and governments, including the NHS & research laboratories. For instance, in March 2022, the UK government invested USD 326.45 million to accelerate healthcare research and manufacturing. Under this investment, additional $80 million of the fund will help companies at the forefront of invention with their commercial-scale manufacturing investments in areas like gene and cell therapies, as well as improved diagnostic technologies, among others. Various mergers & partnerships between manufacturers, universities, and other government bodies are expected to boost the market over the forecast period.

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What is gene therapy used for?

Most gene therapies are still in the clinical trial phase. Clinical trials play an important role in finding treatments that are safe and effective. Clinical trials are investigating gene therapy for the treatment ofcancer,macular degenerationand other eye diseases, certaingenetic conditionsandHIV/AIDS.

The U.S. Food and Drug Administration (FDA) has approved two gene therapies for use in the U.S.:

Is gene therapy safe?

The first gene therapy trial was run more than thirty years ago. The earliest studies showed that gene therapy could have very serious health risks, such as toxicity, inflammation, and cancer. Since then, researchers have studied the mechanisms and developed improved techniques that are less likely to cause dangerous immune reactions or cancer. Because gene therapy techniques are relatively new, some risks may be unpredictable; however, medical researchers, institutions, and regulatory agencies are working to ensure that gene therapy research, clinical trials, and approved treatments are as safe as possible.

Comprehensive federal laws, regulations, and guidelines help protect people who participate in research studies (called clinical trials). The U.S. Food and Drug Administration (FDA) regulates all gene therapy products in the United States and oversees research in this area. Researchers who wish to test an approach in a clinical trial must first obtain permission from the FDA. The FDA has the authority to reject or suspend clinical trials that are suspected of being unsafe for participants.

The National Institutes of Health (NIH) also plays an important role in ensuring the safety of gene therapy research. NIH provides guidelines for investigators and institutions (such as universities and hospitals) to follow when conducting clinical trials with gene therapy. These guidelines state that clinical trials at institutions receiving NIH funding for this type of research must be registered with the NIH Office of Biotechnology Activities. The protocol, or plan, for each clinical trial is then reviewed by the NIH Recombinant DNA Advisory Committee (RAC) to determine whether it raises medical, ethical, or safety issues that warrant further discussion at a RAC public meeting.

An Institutional Review Board (IRB) and an Institutional Biosafety Committee (IBC) must approve each gene therapy clinical trial before it can be carried out. An IRB is a committee of scientific and medical advisors and consumers that reviews all research within an institution. An IBC is a group that reviews and approves an institution's potentially hazardous research studies. Multiple levels of evaluation and oversight ensure that safety concerns are a top priority in the planning and carrying out of gene therapy research.

The clinical trial process occurs in three phases. Phase I studies determine if a treatment is safe for people and identify its side effects. Phase II studies determine if the treatment is effective, meaning whether it works. Phase III studies compare the new treatment to the current treatments available. Doctors want to know whether the new treatment works better or has fewer side effects than the standard treatment. The FDA reviews the results of the clinical trial. If it determines that the benefits of the new treatment outweigh the side effects, it approves the therapy, and doctors can use it to treat a disorder.

What are CAR T cell therapy, RNA therapy, and other genetic therapies?

Several treatments have been developed that involve genetic material but are typically not considered gene therapy. Some of these methods alter DNA for a slightly different use than gene therapy. Others do not alter genes themselves, but they change whether or how a genes instructions are carried out to make proteins.

Cell-based gene therapy

CAR T cell therapy (or chimeric antigen receptor T cell therapy) is an example of cell-based gene therapy. This type of treatment combines the technologies of gene therapy and cell therapy. Cell therapy introduces cells to the body that have a particular function to help treat a disease. In cell-based gene therapy, the cells have been genetically altered to give them the special function. CAR T cell therapy introduces a gene to a persons T cells, which are a type of immune cell. This gene provides instructions for making a protein, called the chimeric antigen receptor (CAR), that attaches to cancer cells. The modified immune cells can specifically attack cancer cells.

RNA therapy

Several techniques, called RNA therapies, use pieces of RNA, which is a type of genetic material similar to DNA, to help treat a disorder. In many of these techniques, the pieces of RNA interact with a molecule calledmessenger RNA(or mRNA for short). In cells, mRNA uses the information in genes to create a blueprint for making proteins. By interacting with mRNA, these therapies influence how much protein is produced from a gene, which can compensate for the effects of a genetic alteration. Examples of these RNA therapies include antisense oligonucleotide (ASO), small interfering RNA (siRNA), and microRNA (miRNA) therapies. An RNA therapy called RNA aptamer therapy introduces small pieces of RNA that attach directly to proteins to alter their function.

Epigenetic therapy

Another gene-related therapy, called epigenetic therapy, affectsepigenetic changesin cells. Epigenetic changes are specific modifications (often called tags) attached to DNA that control whether genes are turned on or off. Abnormal patterns of epigenetic modifications alter gene activity and, subsequently, protein production. Epigenetic therapies are used to correct epigenetic errors that underlie genetic disorders.

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Vector Insights

The AAV segment shows a significant revenue contribution of 22.9% in 2023. Several biopharma companies are offering their viral vector platform for the development of AAV-based gene therapy product. For instance, in September 2016, Lonza signed an exclusive agreement with Massachusetts Eye and Ear to support its novel Anc-AAV gene therapy platform for development and commercialization of next-generation gene therapies based on their AAV platform. Similarly, RegenxBio had made an agreement with companies AveXis & Biogen in March 2014 and May 2016, respectively, which would allow both companies to use RegenxBios AAV vector platform for development of gene therapy molecules. Furthermore, in May 2021, Biogen Inc. and Capsigen Inc. entered into a strategic research partnership to engineer novel AAV capsids that have the possibility to deliver transformative gene therapies, which can address the fundamental genetic causes of numerous neuromuscular and CNS disorders. In July 2021, the U.S. Department of Commerces National Institute of Standards and Technology (NIST), National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL), and United States Pharmacopeia (USP) announced a collaboration to evaluate analytical methods and develop standards for AAV. As part of this partnership, NIST and USP will be conducting an interlaboratory study in which several laboratories will measure these serious quality attributes, and their results will be linked and examined. This collaboration will support the development of new promising gene therapies that will significantly advance peoples lives.

Indication Insights

The spinal muscular atrophy (SMA) segment dominated the market in 2023. Although SMA is a rare disorder, it is one of the most common fatal inherited diseases of infancy. The development of Zolgensma (AVXS-101), has proven its effectiveness in treating SMA and altering the phenotype of the illness. The FDA approved Novartis' Zolgensma approval in May 2019, which is aimed at treating the underlying cause of SMA. As of now, Zolgensma is the only gene treatment in this field to have been approved. The approval of this gene therapy is evidence of the growing use of therapies to treat serious hereditary illnesses like SMA.

The Beta-Thalassemia Major/SCD segment is anticipated to register the fastest CAGR over the forecast period. Gene therapy for SCD and -thalassemia is based on transplantation of gene-modified hematopoietic stem cells. Clinical and preclinical studies have shown the efficacy and safety of this therapeutic modality. However, several other factors, such as suboptimal gene expression levels & gene transfer efficiency, limited stem-cell dose and quality, and toxicity of myeloablative regimens are still hampering its efficacy. Despite these challenges, in June 2019, bluebird Bios Zynteglo (formerly LentiGlobin) received conditional approval in Europe for the treatment of -thalassemia and is expected to receive U.S. FDA approval in August 2022. Moreover, the product has already received Orphan Drug status by the U.S. FDA for treatment of patients with sickle cell disease (SCD). Furthermore, in April 2021, Vertex Pharmaceuticals and CRISPR Therapeutics amended partnership for the development, production, and commercialization of CTX001 in sickle beta thalassemia and cell disease. These achievements in this segment are anticipated to significantly boost the adoption of the product in this segment.

Route of Administration Insights

The intravenous segment dominated the global gene therapy market in 2023. Large number of approved products along with strong pipeline for IV candidates is the major reason for the segment dominance. The segment is also expected to emerge as the most lucrative over the forecast period.

Recent Developments

Some of the prominent players in the Gene therapy market include:

Segments Covered in the Report

This report forecasts revenue growth at global, regional, and country levels and provides an analysis of the latest industry trends in each of the sub-segments from 2021 to 2033. For this study, Nova one advisor, Inc. has segmented the global gene therapy market.

Indication

Vector Type

Route of Administration

By Region

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Gene Therapy Market Size Poised to Surge USD 52.40 Billion by 2033 - BioSpace

Recommendation and review posted by Bethany Smith

The global cancer gene therapy market size was accounted for USD 2.95 billion in 2023 and it is increasing around USD 18.11 billion by 2033 with a CAGR of 19.9% from 2024 to 2033, according to a new report by Nova One Advisor.

Cancer Gene Therapy Market Overview

Cancer is a group of diseases that involve abnormal cell growth which can spread to respective parts of the body. Cancer can spread throughout the human body.Gene therapyis a kind of treatment in which the genes that are not normal or are missing in the patients cells are replaced with normal genes. Cancer gene therapy is a technique for treating cancers where the therapeutic DNA is introduced in the gene of the individual suffering from cancer.

Due to a high success rate in preclinical as well asclinical trials, cancer gene therapy is gaining high popularity all over the world. There are numerous techniques utilized in cancer gene therapy. In one of the gene therapy techniques, either the mutated gene is replaced with a healthy gene, or the gene is inactivated if its function is abnormal. In a newly developed technique, new genes can be introduced in the body of the patient to help fight against cancer cells.

Further, the ongoing extensive research and development (R&D) strategies implemented bybiopharmaceuticalfirms for producing novel therapeutic drugs are driving the market growth notably.

The market players can aim towards expansions, collaborations, joint ventures, acquisitions, and partnerships to advance capabilities in gene therapy. This would help in yielding effective therapeutic drugs for treating different kinds of cancers. In April 2022, GSK plc announced the acquisition of Sierra Oncology for 1.6 billion ($1.9 billion). This acquisition would help GSK plc in enhancing its capabilities with respect to targeted therapies for treating rare forms of cancer.

Biotechnologyfirms are evaluating novel gene therapy vectors for increasing levels of protein production/gene expression, reducing immunogenicity, and improving durability.

The top cancers in terms of the count of new cases in 2020 all over the world were Lung Cancer (2,206,771 cases),Breast Cancer(2,261,419 cases),Prostate Cancer(1,414,259 cases), Colorectal Cancer (1,931,590 cases), Stomach Cancer (1,089,103 cases), and Liver cancer (905,677 cases). In 2018, there were around 134,632 new cancer cases and 89,042 cancer-related fatalities. Breast and liver cancers were among the most common tumors in terms of incidence and mortality. The high prevalence of breast cancer cases enhances the scope for CRISPR/Cas9-based gene editing for breast cancer therapy and VISA-claudin4-BikDD gene therapy.

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Key Takeaways:

Cancer Gene Therapy Market Size in U.S. 2024 to 2033

The U.S. cancer gene therapy market size was valued at USD 1.25 billion in 2023 and is anticipated to reach around USD 7.94 billion by 2033, growing at a CAGR of 20.31% from 2024 to 2033.

North America accounted for the largest share of over 61.15% in 2023. This is attributed to the conducive environment facilitated by the government and the National Cancer Institute that supports research and development activities to enhance cancer therapeutics. Further, the presence of key market players in the region, their research efforts in devising gene therapy for cancer treatment, and collaborative efforts among market players to enhance research are boosting the market growth in the region. For instance, in August 2022, Merck & Co., Inc., collaborated with Orna Therapeutics Inc., for discovery, development, and commercialization of multiple programs, inclusive of utilization of mRNA for cancer gene therapy.

Europe is estimated to be the fastest-growing region over the forecast period due to increase in research funding for novel therapeutics by government bodies and increasing demand for novel therapeutics that could help combat the growing incidence of cancer cases across the region. Moreover, The European Unions Horizon Europe Mission on Cancer was launched in September 2023 so as to offer funds to a broad spectrum of activities that are intended to lower Europes cancer burden by accelerating research and innovation in cancer therapeutics. The mission is anticipated to help over 3 million cancer survivors by the year 2033.

The cancer gene therapy market in the Asia Pacific (APAC) region is segmented into India, China, Japan, South Korea, and the rest of the Asia Pacific (APAC) region. China dominated the Asia Pacific region followed by Japan and India in 2023.

The Latin America, Middle East, and African (LAMEA) cancer gene therapy market is segmented into North Africa, South Africa, Saudi Arabia, Brazil, Argentina, and the Rest of LAMEA. The Middle East and the Latin America region are anticipated to have notable growth in the cancer gene therapy market during the forecast period. Brazil held the largest share in the LAMEA region in 2023. Due to low literacy, uncertainty, and civil war in African countries, the cancer gene therapy market in Africa is expected to grow at a comparatively slow rate.

What are the importance of Cancer Gene Therapy?

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Types of gene therapy for cancer

Gene therapy aims tocontrolthe altered genesor genetic mutationsof a cancertoprevent the cancers growth.This approach to using our own cells and genes to treat cancer is called somatic gene therapy.Thistype of gene therapydoes not impact germ-line cells in the reproductive system, meaning none of the genetic changescan bepassedon to otherfamily members.

There are four types of somatic gene therapy: gene editing; gene replacement; gene addition; and gene inhibition.

Gene editing is correcting the cells gene to fix the imbalance by snipping out the faulty part of the gene and changing the cancers DNA. This type of gene therapy may correct the alteration rather than trying to remove it. Gene replacement is just that: replacing the faulty or nonworking gene with a healthy copy of it. This type of gene therapy is another form of trying to fix the genetic change rather than trying to remove it.

Gene addition is adding novel genetic code to a different cell usually an immune system fighter cell to help it combat the protein linked to the damaged gene. CAR T-cell therapy is an example of gene addition. This form of gene therapy isnt adding a copy of an already-existing gene but rather an entirely new gene usually with the intent of killing the cancer cell via the immune system. Doctors may also add a new gene directly to the cancer cell that causes the cancer cell to commit apoptosis (kill itself).

Gene inhibition simply shuts down the faulty gene. This can either kill the cell or prevent it from acting in a cancerous manner, such as growing and replicating exponentially.

Steps of gene therapy

Gene therapy is a new and potentially curative approach to treating cancer, but researchers still have so much to learn. While the steps below may seem straightforward, each part of the process requires years of study to develop the technologies.

Researchers must first identify the gene and protein linked to the cancer. The next steps are:

Steps of CAR T-cell therapy

CAR T-cell therapy has a slightly different process than more direct forms of gene therapy. CAR T cells are lab-generated fighter cells with specific, anti-cancer genetic code. Adding this genetic code is the gene therapy component of CAR T-cell therapy. CAR stands for chimeric antigen receptor, which is the new genetic code added to the T cells.

There are six CAR T-cell therapy agents approved by the U.S. Food and Drug Administration for different blood cancers. These approvals validate CAR T cells as an effective form of cancer gene therapy to improve patient life expectancy.

Doctors first draw blood from a patient and separate the T cells, which are white blood cells leading the immune systems defense against viruses, diseases and more unwanted intruders. T cells aim to protect the body from cancer, but theyre often ineffective at doing so.

The process of drawing blood from patients and separating the T cells is called apheresis.

After removing T cells from the body, the steps of CAR T-cell therapy are:

A similar process occurs for CAR NK-cell therapy. Scientists create chimeric antigen receptors to strengthen natural killer (NK) cells, another white blood cell of the immune system.

How long does CAR T-cell therapy take?

There are six CAR T-cell therapies approved for types of three blood cancers: myeloma, leukemia and lymphoma. CAR T-cell therapy infusions can take place in an inpatient or outpatient care setting, but the patient must be closely monitored at all times.

CAR T-cell therapy can lead to side effects, most notable cytokine release syndrome.

The entire CAR T-cell process lasts approximately one month, not including the recovery time after treatment:

For the first seven days after receiving the CAR T-cell infusion, patients must remain under medical supervision. For weeks 2-4 of the post-infusion timeline, patients must remain within a short drive of their medical facility to respond to any issues.

The total recovery period from CAR T-cell therapy is usually 2-3 months following infusion, according to the Dana-Farber Cancer Institute.

There are several studies for CAR T-cell therapies for cancer. Participating in a clinical trial helps advance cell and gene therapy research and can advance much-needed therapies to more patients in need.

Therapy Insights

Gene induced immunotherapy dominated the market with a revenue share of over 41.9% in 2023. The dominance of the segment can be attributed to research studies aiming to lower the proliferation of various types of cancer by strengthening the immune system. Many gene therapies for cancers are designed on the basis of immunotherapy elements. For instance, PROVENGE (by Dendreon Corporation) is an autologous cellular immunotherapy designed to stimulate a subjects immune system against prostate cancer.

Oncolytic virotherapy is expected to grow at the fastest rate over the forecast period owing to the favorable outcomes and the level of efficacy offered by oncolytic virotherapy. Oncolytic viruses can combat cancer cells without disturbing the healthy cells in vicinity by stimulating natural killer cells. Moreover, there are lucrative research grants for the research on oncolytic virotherapy. For instance, in July 2022, the researchers at the Center for Nuclear Receptors and Cell Signaling at the University of Houston received a USD 1.8 million grant from the National Institutes of Health to work on oncolytic virotherapy.

End-use Insights

Biopharmaceutical companies led the market with a revenue share of over 50.0% in 2023. This is attributed to the increasing global prevalence of different types of cancers owing to various hereditary, environmental, and lifestyle risk factors. Moreover, the market is driven by increasing adoption of elemental gene therapy options by biopharmaceutical giants to design cancer therapeutic regimes. Many novel therapeutic drugs are under different phases of trials and firms are striving to market them in different regions across the globe. For instance, in January 2020, bluebird bio, Inc. launches its drug, ZYNTEGLO in Germany to be used as a one-time gene therapy solution for patients aged 12 years and above.

The biopharmaceutical companies segment is projected to grow at the fastest rate over the forecast period. The increasing global prevalence of malignant tumors is a key factor driving the market. Moreover, an increased interest in oncology therapeutics research and development is resulting in a rise in the number of FDA approvals of gene therapy drugs. For instance, there are 6 FDA-approved cancer gene therapy drugs with Tecratus, Abcema, and Kymriah being the recent approvals.

Recent Developments:

Some of the prominent players in the Cancer gene therapy market include:

Segments Covered in the Report

This report forecasts revenue growth at global, regional, and country levels and provides an analysis of the latest industry trends in each of the sub-segments from 2023 to 2033. For this study, Nova one advisor, Inc. has segmented the global cancer gene therapy market.

Therapy

End-use

By Region

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Excerpt from:
Cancer Gene Therapy Industry is Rising Rapidly Up to USD 18.11 Bn by 2033 - BioSpace

Recommendation and review posted by Bethany Smith

-First efficacy signals demonstrated for a gene therapy under development for Oculopharyngeal Muscular Dystrophy (OPMD) which affects ~15,000 patients worldwide-

- BB-301 facilitated improvements across multiple measures of swallowing function in the first Phase 1b/2a clinical study subject as compared to pretreatment assessments conducted during the observational natural history portion of the study-

-Virtual R&D Day being held today at 9:00 am EDT, details below-

HAYWARD, Calif., April 18, 2024 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDAQ: BNTC) (Benitec or Company), a clinical-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on its proprietary Silence and Replace DNA-directed RNA interference (ddRNAi) platform, today announces positive interim clinical data from the 90-day timepoint following the administration of BB-301 to the studys first subject (Subject 1) treated in the BB-301 Phase 1b/2a single-arm, open-label, sequential, dose-escalation cohort study (NCT06185673) in Oculopharyngeal Muscular Dystrophy (OPMD). BB-301 has been granted Orphan Drug designation by the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP).

To date, no clinical studies have systematically demonstrated a clinical improvement in OPMD patients across both objective and subjective measures of swallowing. We are, therefore, pleased to report positive interim clinical data from multiple radiographic measures as well as subject-reported outcome measures from the first subject treated with BB-301, said Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec. We are highly encouraged by these early clinical trial results and for the hope that they may offer to patients and caregivers, and we look forward to reporting additional results and continuing to treat patients as they enter the dosing portion of the study from the Natural History observational lead-in period.

BB-301 Interim Clinical Study Results:

During the OPMD Natural History Study, which represents the pre-dose observational period for each subject, Subject 1 experienced progressive worsening of dysphagia as demonstrated by the results of the videofluoroscopic swallowing studies (VFSS), the cold water timed drinking test, and the key subject-reported outcome measure (the Sydney Swallow Questionnaire). Videofluoroscopic swallowing studies represent the gold standard analytical method for the quantitative assessment of dysphagia (swallowing difficulty) in the clinical setting.

At the 90-day timepoint following the administration of BB-301, Subject 1 demonstrated improvements in key videofluoroscopic assessments which correlated with the observation of similar improvement in the key subject-reported outcome measure as compared to the average values for the respective assessments completed during the pre-dose observational period (as summarized in Figure 1). Notably, the results of many assessments completed at the 90-day timepoint demonstrated improvements over the initial measurements assessed at the subjects first visit for the natural history observational study which occurred more than 12 months prior to the 90-day assessment.

The most significant VFSS improvements at Day 90 were observed for swallowing tasks centered on the evaluation of pharyngeal constrictor muscle function and swallowing efficiency in the context of the consumption of thin liquids, solid foods and thick, non-solid foods (e.g., yogurt or pudding) (Figure 1). The VFSS improvements correlated with an improvement in the key subject-reported outcome measure the Sydney Swallow Questionnaire, indicating an improvement in swallowing function as reported by Subject 1 (Figure 1).

Figure 1: Improvement in All Outcomes at 90-Days Post-BB-301 Injection*

*Company data on file

Regarding the BB-301 safety profile observed to date, no Serious Adverse Events have been observed for the two subjects that have received BB-301. Transient Grade 2 Gastroesophageal Reflux Disease or GERD (i.e., acid reflux or heartburn) was observed for the two subjects that received BB-301. For both subjects, the GERD resolved following the completion of a short course of common prescription medications approved for the treatment of GERD.

OPMD is a rare progressive muscle-wasting disease caused by a mutation in the poly(A)-binding protein nuclear 1 (PABPN1) gene, for which there is currently no effective drug therapy. The disease is characterized by swallowing difficulties (dysphagia), limb weakness and eyelid drooping (ptosis). Dysphagia worsens over time and can lead to chronic choking, regurgitation, aspiration pneumonia, and in severe cases, death. Available clinical and surgical interventions are limited in scope and effectiveness and do not address the underlying progressive muscle weakness.

Virtual R&D Event Information: This live virtual R&D Event, featuring two OPMD key opinion leaders, will be held at 9:00 AM EDT today, April 18th, 2024 and can be accessed here. The event replay will be placed on the News & Events tab on the Investor page of the Benitec website.

About BB-301

BB-301 is a novel, modified AAV9 capsid expressing a unique, single bifunctional construct promoting co-expression of both codon-optimized Poly-A Binding Protein Nuclear-1 (PABPN1) and two small inhibitory RNAs (siRNAs) against mutant PABPN1. The two siRNAs are modeled into microRNA backbones to silence expression of faulty mutant PABPN1, while allowing expression of the codon-optimized PABPN1 to replace the mutant with a functional version of the protein. We believe the silence and replace mechanism of BB-301 is uniquely positioned for the treatment of OPMD by halting mutant expression while providing a functional replacement protein.

About Benitec Biopharma, Inc.

Benitec Biopharma Inc. (Benitec or the Company) is a clinical-stage biotechnology company focused on the advancement of novel genetic medicines with headquarters in Hayward, California. The proprietary Silence and Replace DNA-directed RNA interference platform combines RNA interference, or RNAi, with gene therapy to create medicines that simultaneously facilitate sustained silencing of disease-causing genes and concomitant delivery of wildtype replacement genes following a single administration of the therapeutic construct. The Company is developing Silence and Replace-based therapeutics for chronic and life-threatening human conditions including Oculopharyngeal Muscular Dystrophy (OPMD). A comprehensive overview of the Company can be found on Benitecs website at http://www.benitec.com.

Forward Looking StatementsExcept for the historical information set forth herein, the matters set forth in this press release include forward-looking statements, including statements regarding Benitecs plans to develop and potentially commercialize its product candidates, the timing of completion of pre-clinical and clinical trials, the timing of the availability of data from our clinical trials, the timing and sufficiency of patient enrollment and dosing in clinical trials, the timing of expected regulatory filings, the clinical utility and potential attributes and benefits of ddRNAi and Benitecs product candidates, the intellectual property position, and other forward-looking statements.

These forward-looking statements are based on the Companys current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; determinations made by the FDA and other governmental authorities; the Companys ability to protect and enforce its patents and other intellectual property rights; the Companys dependence on its relationships with its collaboration partners and other third parties; the efficacy or safety of the Companys products and the products of the Companys collaboration partners; the acceptance of the Companys products and the products of the Companys collaboration partners in the marketplace; market competition; sales, marketing, manufacturing and distribution requirements; greater than expected expenses; expenses relating to litigation or strategic activities; the Companys ability to satisfy its capital needs through increasing its revenue and obtaining additional financing, given market conditions and other factors, including our capital structure; our ability to continue as a going concern; the length of time over which the Company expects its cash and cash equivalents to be sufficient to execute on its business plan; the impact of the COVID-19 pandemic, the disease caused by the SARS-CoV-2 virus and similar events, which may adversely impact the Companys business and pre-clinical and clinical trials; the impact of local, regional, and national and international economic conditions and events; and other risks detailed from time to time in the Companys reports filed with the Securities and Exchange Commission. The Company disclaims any intent or obligation to update these forward-looking statements.

Investor Relations Contact: Irina Koffler LifeSci Advisors, LLC (917) 734-7387 ikoffler@lifesciadvisors.com

A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/a47d2f41-3feb-49a7-a58f-62e5b0dd4332

Originally posted here:
Benitec Biopharma Reports Positive Interim Clinical Trial Data for First OPMD Subject Treated with BB-301 in Phase 1b ... - GlobeNewswire

Recommendation and review posted by Bethany Smith

Generative A.I. technologies can write poetry and computer programs or create images of teddy bears and videos of cartoon characters that look like something from a Hollywood movie.

Now, new A.I. technology is generating blueprints for microscopic biological mechanisms that can edit your DNA, pointing to a future when scientists can battle illness and diseases with even greater precision and speed than they can today.

Described in a research paper published on Monday by a Berkeley, Calif., startup called Profluent, the technology is based on the same methods that drive ChatGPT, the online chatbot that launched the A.I. boom after its release in 2022. The company is expected to present the paper next month at the annual meeting of the American Society of Gene and Cell Therapy.

Much as ChatGPT learns to generate language by analyzing Wikipedia articles, books and chat logs, Profluents technology creates new gene editors after analyzing enormous amounts of biological data, including microscopic mechanisms that scientists already use to edit human DNA.

These gene editors are based on Nobel Prize-winning methods involving biological mechanisms called CRISPR. Technology based on CRISPR is already changing how scientists study and fight illness and disease, providing a way of altering genes that cause hereditary conditions, such as sickle cell anemia and blindness.

Originally posted here:
Generative A.I. Arrives in the Gene Editing World of CRISPR - The New York Times

Recommendation and review posted by Bethany Smith

The ambitious idea of using CRISPR to cure genetic diseases before birth is one step closer to reality. Scientists reported on Monday that they used a form of the technology known as base editing to alter the DNA of laboratory monkeys in the womb, substantially reducing the levels of a toxic protein that causes a fatal liver disease before the animals had even been born.

The research, by a team at the University of Pennsylvania and the Childrens Hospital of Philadelphia (CHOP), will be presented next month at the annual meeting of the American Society of Gene and Cell Therapy, potentially paving the way for human trials.

But arguably the bigger deal, said study co-leader William Peranteau, is that CRISPR base-editing machinery, packaged in lipid nanoparticles, made it into a number of organs beyond the liver, including the heart, kidney, diaphragm, and skeletal muscles. We were surprised to see that we were able to achieve moderate editing in some of these organs, which traditionally have been more difficult to access.

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Read more from the original source:
In a scientific first, researchers use CRISPR base editing to treat liver disease in fetal monkeys - STAT

Recommendation and review posted by Bethany Smith

ZUG, Switzerland and BOSTON, April 22, 2024 (GLOBE NEWSWIRE) -- CRISPR Therapeutics(Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced an oral presentation highlighting the Company's lipid nanoparticle (LNP) approach for ocular editing will be presented at the American Society of Gene & Cell Therapy (ASGCT) 2024 Annual Meeting, taking place May 7 11, 2024, in Baltimore, MD and virtually.

The abstract describes our proprietary capabilities to deliver to and edit genes in the eye, opening a potential new focus area. Multiple LNPs as well as modified gRNAs and mRNAs were screened to achieve maximal editing in vivo. These optimized components have been applied to target myocilin (MYOC). Mutations of MYOC in trabecular meshwork cells have been linked to severe glaucomatous conditions. In human primary trabecular meshwork cells, up to 95% MYOC editing and 85% protein knockdown were seen. This novel approach aims to facilitate glaucoma treatment using transient expression of editing machinery targeting MYOC.

Title: Development of an In Vivo Non-Viral Ocular Editing Platform and Application to Potential Treatments for Glaucoma Session Type: In-Person Oral Presentation Session Title: Ophthalmic and Auditory: Delivery Innovations Abstract Number:87 Location: Room 318 323 Session Date and Time: Wednesday, May 8, 2024, 1:30 p.m. 3:15 p.m. ET

The accepted abstract is available online on the ASGCT website. The data are embargoed until 6:00 a.m. ET on the presentation day, Wednesday May 8, 2024. A copy of the presentation will be available at http://www.crisprtx.com once the presentation concludes.

About CRISPR Therapeutics Since its inception over a decade ago, CRISPR Therapeutics has transformed from a research-stage company advancing programs in the field of gene editing, to a company that recently celebrated the historic approval of the first-ever CRISPR-based therapy and has a diverse portfolio of product candidates across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine, cardiovascular, autoimmune, and rare diseases. CRISPR Therapeutics advanced the first-ever CRISPR/Cas9 gene-edited therapy into the clinic in 2018 to investigate the treatment of sickle cell disease or transfusion-dependent beta thalassemia, and beginning in late 2023, CASGEVY (exagamglogene autotemcel) was approved in some countries to treat eligible patients with either of those conditions. The Nobel Prize-winning CRISPR science has revolutionized biomedical research and represents a powerful, clinically validated approach with the potential to create a new class of potentially transformative medicines. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic partnerships with leading companies including Bayer and Vertex Pharmaceuticals. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Boston, Massachusetts and San Francisco, California, and business offices in London, United Kingdom. To learn more, visit http://www.crisprtx.com.

CRISPR Therapeutics Forward-Looking StatementThis press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding CRISPR Therapeutics expectations about any or all of the following: (i) its ongoing and/or planned preclinical studies, clinical trials and pipeline products and programs, including, without limitation, the status of such studies and trials, potential expansion into new indications and expectations regarding data generally (including expected timing of data releases) as well as the data in the above-described abstract and any associated poster and the data that is being presented as described above; (ii) the safety, efficacy and clinical progress of its various clinical and preclinical programs including the program described in the oral presentation and poster; (iii) the data that will be generated by ongoing and planned preclinical studies and/or clinical trials, and the ability to use that data for the design and initiation of further preclinical studies and/or clinical trials; and (iv) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. AlthoughCRISPR Therapeuticsbelieves that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the efficacy and safety results from ongoing pre-clinical studies and/or clinical trials will not continue or be repeated in ongoing or planned pre-clinical studies and/or clinical trials or may not support regulatory submissions;pre-clinical study and/or clinical trial results may not be favorable or support further development; one or more of its product candidate programs will not proceed as planned for technical, scientific or commercial reasons; future competitive or other market factors may adversely affect the commercial potential for its product candidates; uncertainties inherent in the initiation and completion of preclinical studies for its product candidates and whether results from such studies will be predictive of future results of future studies or clinical trials; uncertainties about regulatory approvals to conduct trials or to market products; uncertainties regarding the intellectual property protection for its technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics most recent annual report on Form 10-K and in any other subsequent filings made byCRISPR Therapeuticswith theU.S. Securities and Exchange Commission, which are available on theSEC'swebsite atwww.sec.gov.CRISPR Therapeuticsdisclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

Investor Contact: Susan Kim +1-617-307-7503 susan.kim@crisprtx.com

Media Contact: Rachel Eides +1-617-315-4493 rachel.eides@crisprtx.com

Read the original:
CRISPR Therapeutics to Present Oral Presentation at the American Society of Gene & Cell Therapy (ASGCT) 2024 ... - GlobeNewswire

Recommendation and review posted by Bethany Smith

CRISPR technologys initial applications were primarily focused on targeted genome editing, but its use has expanded into cell line engineering, which holds promises of groundbreaking advancements, insists Promega'sPhilip Hargreaves

CRISPR technology has come a long way since its discovery as a natural defence mechanism in bacteria. Scientists have harnessed its potential to precisely edit genes, paving the way for groundbreaking advancements in genetic engineering. Initially, CRISPR was primarily used for editing specific genes within organisms, but its scope has expanded rapidly to include the manipulation of entire cell lines. This ability to edit cell lines using CRISPR technology has unlocked a myriad of possibilities.

For many years researchers and drug developers have used immortalised cell lines (derived from humans but engineered to have stable properties over many divisions) to aid their work. These cell lines typically have the gene and/or protein target of interest expressed within them. CRISPR methodology has allowed quicker and more optimal manipulation of these cell lines.

As of now, CRISPR cell line technology is being used extensively for medical and agricultural applications. In medicine, researchers are employing CRISPR to engineer cell lines for therapeutic purposes, intending to treat diseases at the genetic level. For instance, CRISPR has been used to edit the genes of immune cells, enabling them to better target and eliminate cancer cells.

The Future Landscape

Looking ahead, the future of CRISPR cell line technology holds even more transformative possibilities. One of the key areas of exploration is regenerative medicine, where researchers aim to harness CRISPR to engineer cells for tissue repair and organ regeneration. The ability to precisely edit and manipulate cell lines could open new avenues for treating degenerative diseases and injuries, potentially revolutionising the field of transplantation.

The ability to precisely edit and manipulate cell lines could open new avenues for treating degenerative diseases and injuries, potentially revolutionising the field of transplantation

CRISPR technology is further poised to play a crucial role in the development of novel therapies for genetic disorders. The ability to edit problematic genes could pave the way for more effective treatments and even cures for conditions that were once considered incurable, such as Alzheimers.

Despite the remarkable progress in CRISPR technology, challenges and roadblocks still need to be addressed. Off-target effects, unintended mutations, and the potential for unpredictable consequences remain significant hurdles in developing and applying CRISPR-based therapies. Researchers are actively working to enhance the precision and safety of CRISPR technology to mitigate these risks.

CRISPR-engineered cell lines

One way to use CRISPR engineering to generate cell lines, which Promega is successfully offering, is to incorporate HiBiT technology. This 11 amino acid peptide can be fused to a target protein and serves as a luminescent tag. HiBiT can be integrated by knocking in the tag to the endogenous locus of the target using CRISPR gene editing, to help create a truer picture of protein behaviour and regulation in their natural cellular environment. HiBiT has a dynamic range spanning nine logs, which allows for the detection of extremely small quantities of tagged proteins.

In terms of applications, HiBiT's versatility is unmatched, as quantitative assays can be performed in both endpoint and live-cell formats, without the need for target-specific antibodies. From measuring target protein abundance to studying targeted protein degradation, protein secretion and receptor recycling, HiBiT opens up a myriad of possibilities in biomedical research. Its role in drug discovery is particularly noteworthy, enabling more precise and efficient screening of drug effects on cellular proteins.

The accessibility and affordability of CRISPR technology also need to be addressed to ensure that its benefits are not confined to a privileged few. Wide-scale adoption and integration of CRISPR cell line technology into various sectors will require collaborative efforts from researchers, policymakers, and the private sector. To aid in this endeavour, Promega now offers a comprehensive selection of pre-built CRISPR-edited cell line pools and clones, including HiBiT fusions.

Wide-scale adoption and integration of CRISPR cell line technology into various sectors will require collaborative efforts from researchers, policymakers, and the private sector

This development opens doors for researchers and developers as it reduces the costs involved in developing cell lines from scratch. Not only will this save budgets, but it also saves time. This means drug development times can be reduced by as much as 12 months, leading to vital medications being available much sooner.

The future of CRISPR cell line technology is undoubtedly exciting and promises transformative advancements in medicine, agriculture, and beyond. As scientists continue to unravel the mysteries of genetic engineering, there is a delicate balance between innovation and ethical stewardship to consider. But with efforts to maximise the technologys potential to make it more accessible and affordable, the future is bright.

Philip Hargreaves Ph.D, is director of strategic marketing & business development at Promega

Pic: Brano

More here:
The evolution of CRISPR cell line technology - Lab News

Recommendation and review posted by Bethany Smith

SNIPR Biome

SNIPR Biome receives funding from CARB-X to support advancement of CRISPR-medicine SNIPR001 into clinical trials in haematological cancer patients

Phase 1b/2a trial will evaluate SNIPR001 for the prevention of E.coli infections in patients undergoing hematopoietic stem cell transplantation

Copenhagen, April 22 2024: SNIPR Biome ApS (SNIPR), the company pioneering the development of precision medicines using CRISPR technology for microbial gene therapy, announces today that it has received $5.48 million from Combating Antibiotic-Resistant Bacteria Biopharmaceutical Accelerator (CARB-X) to co-fund a Phase 1b/2a clinical trial in hematological cancer patients.

The trial will evaluate SNIPR001, the first CRISPR-armed phage therapeutic that specifically targets E. coli in the gut, for the prevention of E. coli bloodstream infections in hematological cancer patients who are undergoing hematopoietic stem-cell transplantation (HSCT) and are colonized with Fluoroquinolone Resistant (FQR) E. coli. Fluoroquinolone is recommended in the US for prophylaxis of bacterial infections and febrile neutropenia in hematological cancer patients at high risk of neutropenia.

Despite the significant advances in hematologic cancer therapy over the past decade, infectious complications, and antimicrobial resistance (AMR) continue to pose significant threats to patients and clinical outcomes1. Currently, there are no approved therapies for the prevention of bloodstream infections (BSIs) in hematological cancer patients. SNIPR Biome is developing SNIPR001 to address this urgent unmet need to combat infections in hematological cancer patients.

Preclinical data published in Nature Biotechnology described SNIPR001s ability to selectively target and remove antibiotic-resistant E. coli strains in the gut, potentially offering a safe treatment which preserves the rest of the gut microbiome. This was supported by interim Phase 1 data published in 2023, which showed that oral dosing of SNIPR001 over seven days across three dosing levels in 24 healthy individuals was well tolerated. Furthermore, SNIPR001 could be recovered in faeces from treated individuals in a dose-dependent manner, and treatment with SNIPR001 numerically lowered gut E. coli levels.

Anticipated to begin later this year, the randomized, double-blinded Phase 1b/2a trial will investigate the safety, tolerability, pharmacokinetics, and pharmacodynamics of orally administrated SNIPR001 in 24 patients. It will be conducted at up to 10 sites across Europe and the United States.

CARB-X, a global non-profit partnership dedicated to supporting early-stage antibacterial research and development to address the rising threat of drug-resistant bacteria, has been a long-term collaborator with SNIPR in this field. The funding announced today enables SNIPR to move SNIPR001 into Phase 1b/2a clinical trials and will serve as a cornerstone for a further significant fundraise to enable the Company to continue development of its pipeline of CRISPR-based AMR and gut-directed gene therapies.

Story continues

Dr Christian Grndahl, Co-founder and CEO of SNIPR Biome, commented: Antibiotic resistance is one of healthcares biggest problems today, affecting treatment efficacy and survival among patients who are often already very sick. We are using our knowledge of gene editing and synthetic biology to create highly specific, designer bacteria and phage to disrupt, edit or add genes, and deliver these precision medicines in a carefully targeted way. We are pleased to be continuing our partnership with CARB-X who share our commitment to developing therapies for vulnerable patients.

Erin Duffy PhD, Chief of Research & Development, CARB-X, said: Having underscored safety for SNIPR001 in healthy subjects, SNIPR Biome is now focusing on demonstrating proof-of-mechanism for this novel product, with our support.We are keen to establish a link between gut decolonization and prevention of infection as a novel approach to antimicrobial resistance, and SNIPR001 offers the possibility of doing so.

CARB-X funding for this research is supported by the Biomedical Advanced Research and Development Authority under agreement number: 75A50122C00028, and by awards from Wellcome (WT224842), and Germanys Federal Ministry of Education and Research (BMBF). The content of this press release is solely the responsibility of the authors and does not necessarily represent the official views of CARB-X or any of its funders.

Ends

About SNIPR001 SNIPR001, a CRISPR-armed phage therapeutic that specifically targets E. coli in the gut, is designed to prevent infections from spreading into the bloodstream and represents a promising advancement against antibiotic-resistant pathogens. The pre-clinical studies of SNIPR001 published in Nature Biotechnology2 demonstrated the products activity against multi-drug resistant strains of E. coli and its specificity towards E. coli with no off-target effects toward any of the tested non-E. coli strains. SNIPR successfully completed a Phase 1 trial in the US, also funded by CARB-X, demonstrating safety of SNIPR001 and target engagement with E. coli in the gut of healthy subjects without disturbing the overall gut microbiome (NCT05277350), supporting its potential as a safe and effective preventative therapy for bloodstream infections in hematological cancer patients. SNIPR001 has been granted a Fast-Track designation for the indication Prophylaxis of bloodstream E. coli infections in patients with hematological malignancy at risk of neutropenia from the US Food and Drug Administration (FDA). SNIPR001 is also being developed to directly treat active E. coli infections.

About SNIPR BIOME SNIPR Biome is a Danish clinical-stage biotech company pioneering the development of precision medicines using CRISPR technology for microbial gene therapy. We are pioneering a novel use of CRISPR/Cas technology to better treat and prevent human diseases through precision killing of bacteria or gene modification. SNIPR Biome was the first company to orally dose humans with a CRISPR therapeutic and the first company to have been granted US and European patents for the use of CRISPR for targeting microbiomes. SNIPR technology is used in collaborations with Novo Nordisk A/S, CARB-X, SPRIN-D, and MD Anderson Cancer Center. For more information, visit http://www.sniprbiome.com and follow us on LinkedIn and X.

About CARB-X

CARB-X (Combating Antibiotic-Resistant Bacteria Biopharmaceutical Accelerator) is a global non-profit partnership dedicated to supporting early-stage antibacterial research and development to address the rising threat of drug-resistant bacteria. CARB-X supports innovative therapeutics, preventatives and rapid diagnostics. CARB-X is led by Boston University and funded by a consortium of governments and foundations. CARB-X funds only projects that target drug-resistant bacteria highlighted on the CDCs Antibiotic Resistant Threats list, or the Priority Bacterial Pathogens list published by the WHO, with a priority on those pathogens deemed Serious or Urgent on the CDC list or Critical or High on the WHO list. https://carb-x.org/ | X (formerly Twitter) @CARB_X

Contact ICR Consilium Tracy Cheung, Chris Welsh, Davide Salvi SNIPR@consilium-comms.com

SNIPR Biome Dr Christian Grndahl, Co-founder and CEO contact@sniprbiome.com http://www.sniprbiome.com

1 So M. Determining the Optimal Use of Antibiotics in Hematopoietic Stem Cell Transplant Recipients. JAMA Netw Open. 2023 Jun 1;6(6):e2317101 2 Gencay, Y.E., Jasinskyt, D., Robert, C. et al. Engineered phage with antibacterial CRISPRCas selectively reduce E. coli burden in mice. Nat Biotechnol (2023). https://doi.org/10.1038/s41587-023-01759-y

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SNIPR Biome receives funding from CARB-X to support advancement of CRISPR-medicine SNIPR001 into clinical ... - Yahoo Finance

Recommendation and review posted by Bethany Smith

Crispr Therapeutics AG (NASDAQ:CRSP)is 2.8% lower at $56.35 this afternoon, continuing a pullback from a Feb. 22, more than two-year high of $91.10. Over the last month, CRSP has erased 20.8% and now sports a 9.6% year-to-date deficit.

For those looking to buy in on the dip, however, the recent pullback puts Crispr Therapeutics stock within one standard deviation of its 320-day moving average, a trendline with historically bullish implications. According to Schaeffer's Senior Quantitative Analyst Rocky White, the equity saw two similar signals in the past three years, after which it was higher one month later each time, averaging an impressive 13.3% gain. A move of similar magnitude would put the shares at roughly $63.85.

Crispr Therapeutics stock's 14-day relative strength index (RSI) of 18.2 is deep in "oversold" territory, which is typically indicative of a short-term bounce. Plus, short interest represents 17.6% of the stock's available float, and would take eight days to cover at CRSP's average pace of trading.

Plus, itsSchaeffer's Volatility Scorecard(SVS) stands at a high 86 out of 100, indicating the stock exceededoptiontraders' volatility expectations in the past 12 month -- a boon for premium buyers.

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Bullish Trendline Has Never Failed Crispr Therapeutics Stock - Yahoo Finance

Recommendation and review posted by Bethany Smith

Researchers have engineered a Cas enzyme to enhance activity against RNA viruses, resulting in a system that completely blocked the replication of various SARS-CoV-2 strains.

The research, details of which were published in Cell Discovery, was prompted by the need to overcome a barrier to the use of the Cas13d enzyme as an antiviral against human RNA viruses. Studies have shown that CRISPR/Cas13 systems are programmable tools for manipulating RNAs and Cas13d is the most active subtype of the enzyme in mammalian cells, making it the most promising antiviral candidate.

However, the activity of Cas13d is largely restricted to the nucleus. Most RNA viruses only replicate in the cytosol, where Cas13d is barely active in mammalian cells and as such are protected from the antiviral effects enabled by the enzyme, wrote the study's authors, led by Christoph Gruber of the Technical University of Munich (Cell Discov, April 12, 2024, Vol. 10 [1], pp. 1-4).

The problem led Gruber and other scientists from Helmholtz Munich and the Technical University of Munich to investigate why Cas13d is largely restricted to the nucleus and explore ways to bring the enzyme into contact with replicating RNA viruses in mammalian cells.

The research revealed that the enzyme has little activity in the cytosol because the RNA that guides the CRISPR-Cas complex to specific target sequences is found in the nucleus. That finding led the scientists to explore ways to move CRISPR RNAs (crRNAs) from the nucleus to the cytosol.

Through screening and optimization of various designs of shuttling proteins, the researchers developed a system that transfers nuclear crRNAs into the cytosol. The enzyme -- nucleocytoplasmic shuttling Cas13d or Cas13d-NCS for short -- moves nuclear crRNAs to the cytosol, where the protein/crRNA complex binds and degrades complementary target RNAs.

The researchers hypothesized that Cas13d-NCS more adeptly degrades viral cytosolic RNAs than standard Cas13d and tested that idea using a self-replicating RNA from the Venezuelan equine encephalitis RNA virus. The tests showed the engineered approach "targets solely cytosolic RNA with greater efficiency compared to the current Cas13d system," the authors wrote.

To assess antiviral efficacy, the scientists targeted SARS-CoV-2, the RNA virus that causes COVID-19. The assessment showed Cas13d-NCS can completely block the replication of various SARS-CoV-2 strains, they wrote.

"Targeting conserved but weakly expressed viral-coding sequences resulted in relatively weak inhibition, whereas targeting the ubiquitous 3'UTR with a single crRNA resulted in complete inhibition of viral replication," Gruber et al concluded.

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Engineered Cas clears barrier to antiviral CRISPR therapies - LabPulse

Recommendation and review posted by Bethany Smith

A formidable collaboration between three University of California (UC) schools and leading global life sciences and diagnostics innovator the Danaher Corporation heralds a new era in the fight against rare and deadly genetic diseases, such as sickle cell disease which predominantly impacts Black and Hispanic populations in the U.S. through the innovative use of CRISPR technology.

Spearheaded by the Innovative Genomics Institute (IGI), this joint effort brings together genetics researchers and clinician experts from UC San Francisco, UC Los Angeles, UC Berkeley, and other research institutions, to expedite the development of curative therapies for diseases that have previously lacked effective treatments.

The Danaher-IGI Beacon for CRISPR Cures center will leverage genome editing technology to research a wide range of genetic disorders. The center, which will be led out of the IGI headquarters at UC Berkeley, combines expertise in genetics research, clinical practice, and industry resources to accelerate the development and deployment of CRISPR-based treatments. The goal is to establish new standards for safety and efficacy while streamlining the path from preclinical research to clinical trials.

The unique nature of CRISPR makes it ideal for developing and deploying a platform capability for CRISPR cures on demand, said Fyodor Urnov, PhD, IGIs Director of Technology and Translation, in a press release. Danaher and the IGI are in a unique position to potentially create a first-of-its-kind CRISPR cures cookbook that could be used by any team wishing to take on other diseases.

The centers initial focus will be on hemophagocytic lymphohistiocytosis (HLH) and Artemis-deficient severe combined immunodeficiency (ART-SCID), two conditions characterized by defects in a patients immune system. Traditional treatments for these disorders, such as bone marrow transplants, often fall short due to complications.

By targeting specific gene mutations associated with these diseases, researchers hope to develop therapies that address their underlying causes, improve outcomes, and enhance quality of life for those affected.

Using CRISPR, the IGI has already made incredible advancements in treating sickle cell disease through clinical trials at the Comprehensive Sickle Cell Center at UC San Francisco Benioff Childrens Hospital in Oakland which was established to address racial biases in health care. In 2021, the center received $17 million in funding to advance the use of CRISPR in sickle cell research.

This therapy has the potential to transform sickle cell disease care, said Mark Walters, MD, a pediatric professor at UC San Francisco and principal investigator of the clinical trials. If this is successfully applied in young patients, it has the potential to prevent irreversible complications of the disease.

Since then, researchers have been testing the possibility of replacing the gene that causes sickle cell with a healthy one manufactured using a patients own stem cells. Early tests have been positive, indicating a potential cure for the disease.

With CRISPR, we can speed up the development of improved therapies that can reach all the patients who need them, said Jennifer Puck, MD, a faculty member at the Jeffrey Modell Diagnostic Center for Primary Immunodeficiencies and Institute for Human Genetics, both at UC San Francisco. All patients deserve a sense of urgency. including those with rare diseases, many of whom are children.

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CRISPR Center Advances Genetic Disease Research - INSIGHT Into Diversity

Recommendation and review posted by Bethany Smith