AVROBIO Receives Rare Pediatric Disease Designation from U.S. Food and Drug Administration (FDA) for First Gene Therapy in Development for Cystinosis…

CAMBRIDGE, Mass.--(BUSINESS WIRE)--AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company working to free people from a lifetime of genetic disease, today announced that the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to AVR-RD-04, an investigational gene therapy for the treatment of cystinosis, a life-threatening disease that causes progressive multi-organ damage, including early, acute kidney disease progressing to end-stage kidney disease.

FDAs Rare Pediatric Disease Designation and Voucher Program is intended to facilitate the development of new drugs and biologics for the prevention and treatment of rare pediatric diseases. Companies that receive approval for a New Drug Application (NDA) or Biologics License Application (BLA) for a rare pediatric disease may be eligible to receive a voucher for a priority review of a subsequent marketing application for a different product. The priority review voucher may be used by the company or sold to a third party.

AVR-RD-04 is designed to genetically modify patients own hematopoietic stem cells (HSCs) to express the gene encoding cystinosin, the protein that is critically deficient in people living with cystinosis.

Preliminary data from the ongoing University of California San Diego Phase 1/2 clinical trial suggest that this approach is well tolerated, with no adverse events (AEs) related to the drug product reported to date. All AEs reported were related to myeloablative conditioning, stem cell mobilization, underlying disease or pre-existing conditions. The majority of AEs were mild or moderate and resolved without clinical sequelae. Clinical data to date indicate this investigational approach provides benefits in multiple tissues evaluated, including the eyes, skin, gastrointestinal mucosa and the neurocognitive system. The collaborator-sponsored Phase 1/2 clinical trial is funded in part by grants to University of California San Diego from the California Institute for Regenerative Medicine (CIRM), Cystinosis Research Foundation (CRF) and National Institutes of Health (NIH).

About Cystinosis Cystinosis is a rare, progressive disease that impacts approximately 1,600 patients in the U.S., Europe and Japan and is marked by the accumulation of cystine in cellular organelles known as lysosomes. Untreated cystinosis is fatal at an early age. The current SOC for cystinosis, a treatment regimen that can require dozens of pills per day, does not prevent overall disease progression and carries side effects, such as breath and body odor and gastrointestinal symptoms, which can impede compliance. More than 90% of treated cystinosis patients require a kidney transplant in the second or third decade of life.

About AVROBIO Our vision is to bring personalized gene therapy to the world. We target the root cause of genetic disease by introducing a functional copy of the affected gene into patients own hematopoietic stem cells (HSCs), with the goal to durably express the therapeutic protein throughout the body, including the central nervous system. Our first-in-class pipeline includes clinical programs for cystinosis and Gaucher disease type 1, as well as preclinical programs for Gaucher disease type 3, Hunter syndrome and Pompe disease. Our proprietary plato gene therapy platform is designed to be scaled to support late-stage clinical development and commercialization globally. We are headquartered in Cambridge, Mass. For additional information, visit avrobio.com, and follow us on Twitter and LinkedIn.

Forward-Looking Statements This press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words and phrases such as aims, anticipates, believes, could, designed to, estimates, expects, forecasts, goal, intends, may, plans, possible, potential, seeks, will, and variations of these words and phrases or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding our business strategy for and the potential therapeutic benefits of our preclinical and clinical product candidates, including AVR-RD-04 for the treatment of cystinosis, the potential benefits and incentives provided by FDAs rare pediatric disease designation for AVR-RD-04, the design, commencement, enrollment and timing of planned clinical trials, preclinical or clinical trial results, product approvals and regulatory pathways, our plans and expectations with respect to interactions with regulatory agencies, anticipated benefits of our gene therapy platform including potential impact on our commercialization activities, timing and likelihood of success, the expected benefits and results of our implementation of the plato platform in our clinical trials and gene therapy programs, and the expected safety profile of our preclinical and investigational gene therapies. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Results in preclinical or early-stage clinical trials may not be indicative of results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.

Any forward-looking statements in this press release are based on AVROBIOs current expectations, estimates and projections about our industry as well as managements current beliefs and expectations of future events only as of today and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that any one or more of AVROBIOs product candidates will not be successfully developed or commercialized, the risk of cessation or delay of any ongoing or planned clinical trials of AVROBIO or our collaborators, the risk that AVROBIO may not successfully recruit or enroll a sufficient number of patients for our clinical trials, the risk that AVROBIO may not realize the intended benefits of our gene therapy platform, including the features of our plato platform, the risk that our product candidates or procedures in connection with the administration thereof will not have the safety or efficacy profile that we anticipate, the risk that prior results, such as signals of safety, activity or durability of effect, including beneficial effects seen in multiple organs and tissues, observed from preclinical or clinical trials, will not be replicated or will not continue in ongoing or future studies or trials involving AVROBIOs product candidates, the risk that we will be unable to obtain and maintain regulatory approval for our product candidates, the risk that the size and growth potential of the market for our product candidates will not materialize as expected, risks associated with our dependence on third-party suppliers and manufacturers, risks regarding the accuracy of our estimates of expenses and future revenue, risks relating to our capital requirements and needs for additional financing, risks relating to clinical trial and business interruptions resulting from the COVID-19 outbreak or similar public health crises, including that such interruptions may materially delay our enrollment and development timelines and/or increase our development costs or that data collection efforts may be impaired or otherwise impacted by such crises, and risks relating to our ability to obtain and maintain intellectual property protection for our product candidates. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause AVROBIOs actual results to differ materially and adversely from those contained in the forward-looking statements, see the section entitled Risk Factors in AVROBIOs most recent Annual or Quarterly Report, as well as discussions of potential risks, uncertainties and other important factors in AVROBIOs subsequent filings with the Securities and Exchange Commission. AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.

Read the original:
AVROBIO Receives Rare Pediatric Disease Designation from U.S. Food and Drug Administration (FDA) for First Gene Therapy in Development for Cystinosis...

Stem Cell Transplantation: What it Is, Process & Procedure

Overview What is a stem cell transplant?

Healthcare providers use stem cell transplants to treat people who have life-threatening cancer or blood diseases caused by abnormal blood cells. A stem cell transplant helps your body replace those blood cells with healthy or normal blood cells. If you receive a stem cell transplant, your provider may use your own healthy stem cells or donor stem cells.

Your blood cells come from stem cells in your bone marrow. Your bone marrow constantly creates new stem cells that become blood cells. Stem cell transplants can involve stem cells taken from bone marrow or from blood. Providers sometimes refer to stem cell and bone marrow transplants as haematopoietic stem cell transplants (HSCT). This article focuses on stem cells taken from blood.

Healthcare providers use stem cells to replace unhealthy blood cells that cause conditions such as several types of leukemia, lymphoma and testicular cancer. They also use transplanted stem cells to treat several types of anemia. Some people who have multiple sclerosis may benefit by receiving healthy stem cells. Researchers are investigating ways to treat other autoimmune diseases with stem cell transplants.

Healthcare providers typically use stem cell transplants to treat life-threatening cancer or blood diseases. Unfortunately, not everyone who has those conditions can have the procedure. Here are factors providers take into consideration:

Recently data reported nearly 23,000 people had stem cell transplants in 2018.

To understand how stem cell transplants work, it may help to know more about stem cells and their role in your body:

Healthcare providers obtain stem cells from several sources:

If youre a candidate for a stem cell transplant, your healthcare provider will perform the following tests to confirm youre physically able to manage transplantation processes, including pre-treatment chemotherapy called conditioning and transplantation side effects:

Before your blood tests, your provider may place a central venous catheter (CVC) in one of the large veins in your upper chest. CVCs are tubes that serve as central lines that providers use to take blood and provide medication and fluids. CVCs eliminate repeated needle sticks to draw blood or insert intravenous tubes throughout the transplantation process.

Transplant conditioning is intensive chemotherapy and/or radiation therapy that kills cancer cells in your bone marrow. Conditioning also kills existing blood cells.

If youre receiving your own stem cells, your provider may give you medication to boost your stem cell production. Theyll do follow-up blood tests to check on stem cell production.

If youre receiving your own stem cells, your providers will take blood so they can remove healthy stem cells for transplant. . To do that, they connect veins in both of your arms to a cell separator machine. The machine pulls your blood from one arm, filters the blood and then returns it to through your other arm. This process doesnt hurt. Providers may need to take blood more than once to ensure they have enough stem cells to transplant. The actual transplantation involves receiving your stem cells via your CVC.

Just like someone receiving their own cells, youll receive healthy stem cells via your CVC.

Your new stem cells will need time to produce new blood cells. If you received donor stem cells, your transplanted stem cells will replace unhealthy stem cells and begin to build a new immune system. This process is engraftment.

Either way, you may need to stay in or close to the hospital for several months so your healthcare providers can support your recovery and monitor your progress. Heres what you can expect after your stem cell transplant:

Successful stem cell transplants may help people when previous treatments dont slow or eliminate certain cancers.

The greatest risk is that youll go through the procedure and your transplanted stem cells cant slow or eliminate your illness.

Allogenic and autologous stem cell transplants have different complications. Allogenic stem cell transplants can result in graft versus host disease. This happens when your immune system attacks new stem cells. Potential complications will vary based on your overall health, age and previous treatment. If youre considering a stem cell transplant, your healthcare provider will outline potential complications so you can weigh those risks against potential benefits.

It can take several weeks to several months to recover from a stem cell transplant. Your healthcare provider may recommend you stay in or near the hospital or transplant center for the first 100 days after your procedure.

Its difficult to calculate an overall success rate. That said, the most recent data show the highest number of stem cell transplants involved people with multiple myeloma or Hodgkin and non-Hodgkin lymphoma who received autologous stem cell transplants. Here is information on three-year survival rates:

A successful stem cell transplant can change your life, curing your condition or slowing its growth. But its not an overnight transformation. It can take a year or more for you to recover. Here are some challenges and ways to overcome them:

You may have days when you feel exhausted and days when you feel fine. A hard day doesnt mean youre not doing well. It means you need to give yourself a break and take it easy.

Youll have regular follow-up appointments with your provider. But its important to remember your immune system likely will be weak for a year or so after your transplantation. Contact your provider right away if you develop any of the following symptoms:

A note from Cleveland Clinic

If youve been coping with cancer or a blood disease, a stem cell transplant can be a new lease on life. It can mean hope for a cure or remission when other treatments havent worked. But stem cell transplants come with demanding physical challenges and significant risks. Not everyone who has cancer or blood conditions is a candidate for a stem cell transplant. Unfortunately, not everyone who is a candidate but needs donor stem cells finds a donor. If youre considering a stem cell transplant, talk to your healthcare provider about potential risks and benefits. Theyll evaluate your situation, your options and potential outcomes.

Last reviewed by a Cleveland Clinic medical professional on 03/14/2022.

Visit link:
Stem Cell Transplantation: What it Is, Process & Procedure

Century Therapeutics Receives Study May Proceed Notification from FDA for CNTY-101, the First Allogeneic Cell Therapy Product Candidate Engineered to…

Century Therapeutics, Inc.

Investigational New Drug Application for CNTY-101, a CAR-iNK product candidate targeting CD19 for B-cell malignancies, cleared by FDA

First cell product candidate engineered with six precision gene edits including a CD19-CAR, Allo-Evasiontechnology, IL-15 cytokine support and a safety switch

Phase 1 ELiPSE-1 trial evaluating CNTY-101 in relapsed or refractory CD19 positive B-cell malignancies anticipated to begin in 2H22

PHILADELPHIA, Aug. 25, 2022 (GLOBE NEWSWIRE) -- Century Therapeutics, Inc., (NASDAQ: IPSC), an innovative biotechnology company developing induced pluripotent stem cell (iPSC)-derived cell therapies in immuno-oncology, announced today that the company has been notified by the U.S. Food and Drug Administration (FDA) that the Companys ELiPSE-1 clinical study may proceed to assess CNTY-101 in patients with relapsed or refractory CD19 positive B-cell malignancies. CNTY-101 is the first allogeneic cell therapy product candidate engineered with four powerful and complementary functionalities, including a CD19 CAR for tumor targeting, IL-15 support for enhanced persistence, Allo-Evasiontechnology to prevent host rejection and enhance persistence and a safety switch to provide the option to eliminate the drug product if ever necessary. CNTY-101 is manufactured from a clonal iPSC master cell bank that yields homogeneous product, in which all infused cells have the intended modifications.

This IND clearance is a significant milestone for Century as we execute on our vision to merge two disruptive platforms, precision gene editing and the powerful potential of iPSCs, to potentially move the allogeneic cell therapy field forward, and continue on our path to becoming a leader in the space, said Lalo Flores, Chief Executive Officer, Century Therapeutics. We believe that CNTY-101, our first and wholly owned product candidate, will be the most technically advanced and differentiated CD19-targeted cell product when it enters the clinic, which is anticipated to occur later this year. We look forward to assessing the potential of Allo-Evasionto prevent immunological rejection and enhance persistence of multiple dosing of CNTY-101 regimens with the aim to increase the proportion of patients that achieve durable responses.

Story continues

CNTY-101 is the first allogeneic cell product candidate with six genetic modifications incorporated using sequential rounds of CRISPR-mediated homologous recombination and repair that has received IND clearance by the FDA, said Luis Borges, Chief Scientific Officer, Century Therapeutics. We believe CNTY-101 will demonstrate the power of Centurys iPSC technology and cell engineering technology platforms. This accomplishment is a testament to the expertise and dedication of our team as we continue to make progress developing our pipeline of iPSC-derived NK and T cell product candidates.

The Phase 1 trial, ELiPSE-1 (NCT05336409), is intended to assess the safety, tolerability, pharmacokinetics and preliminary efficacy of CNTY-101 in patients with relapsed or refractory CD19-positive B-cell malignancies. All patients will receive an initial standard dose of conditioning chemotherapy consisting of cyclophosphamide (300 mg/m2) and fludarabine (30mg/m2) for 3 days. Schedule A of the trial includes a single-dose escalation of CNTY-101 and subcutaneous IL-2. Schedule B will evaluate a three-dose schedule per cycle of CNTY-101. Patients who demonstrate a clinical benefit are eligible for additional cycles of treatment with or without additional lymphodepletion pending FDA consent. We anticipate initiation of the Phase 1 trial later this year.

About Allo-Evasion

Centurys proprietary Allo-Evasiontechnology is used to engineer cell therapy product candidates with the potential to evade identification by the host immune system so they can be dosed multiple times without rejection, enabling increased persistence of the cells during the treatment period and potentially leading to deeper and more durable responses. More specifically, Allo-Evasion1.0 technology incorporates three gene edits designed to avoid recognition by patient/host CD8+ T cells, CD4+ T cells and NK cells. Knockout of beta-2-microglobulin or 2m, designed to prevent CD8+ T cell recognition, knock-out of the Class II Major Histocompatibility Complex Transactivator, or CIITA, designed to prevent CD4+ T cell recognition, and knock-in of the HLA-E gene, designed to enable higher expression of the HLA-E protein to prevent killing of CNTY-101 cells by host NK cells. Allo-Evasiontechnology may allow the implementation of more flexible and effective repeat dosing protocols for off-the-shelf product candidates.

About CNTY-101

CNTY-101 is an investigational off-the-shelf cancer immunotherapy product candidate that utilizes iPSC-derived natural killer (NK) cells with a CD19-directed chimeric antigen receptor (CAR) and includes Centurys core Allo-Evasionedits designed to overcome the three major pathways of host versus graft rejection - CD8+ T cells, CD4+ T cells and NK cells. In addition, the product candidate is engineered to express IL-15 to provide homeostatic cytokine support, which has been shown pre-clinically to improve functionality and persistence. Further, to potentially improve safety, the iNK cells were engineered with an EGFR safety switch, and proof-of-concept studies have demonstrated that the cells can be quickly eliminated by the administration of cetuximab, an antibody against EGFR approved by the U.S. Food and Drug Administration (FDA) for certain cancers. Initiation of the Phase 1, ELiPSE-1 trial in relapsed or refractory CD19-positive B-cell malignancies in multiple centers in the United States is anticipated to begin in the second half of 2022.

About Century Therapeutics

Century Therapeutics, Inc. (NASDAQ: IPSC) is harnessing the power of adult stem cells to develop curative cell therapy products for cancer that we believe will allow us to overcome the limitations of first-generation cell therapies. Our genetically engineered, iPSC-derived iNK and iT cell product candidates are designed to specifically target hematologic and solid tumor cancers. We are leveraging our expertise in cellular reprogramming, genetic engineering, and manufacturing to develop therapies with the potential to overcome many of the challenges inherent to cell therapy and provide a significant advantage over existing cell therapy technologies. We believe our commitment to developing off-the-shelf cell therapies will expand patient access and provide an unparalleled opportunity to advance the course of cancer care. For more information on Century Therapeutics please visithttps://www.centurytx.com/.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of, and made pursuant to the safe harbor provisions of, The Private Securities Litigation Reform Act of 1995. All statements contained in this press release, other than statements of historical facts or statements that relate to present facts or current conditions, including but not limited to, statements regarding our clinical development plans and timelines are forward-looking statements. These statements involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance, or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. In some cases, you can identify forward-looking statements by terms such as may, might, will, should, expect, plan, aim, seek, anticipate, could, intend, target, project, contemplate, believe, estimate, predict, forecast, potential or continue or the negative of these terms or other similar expressions. The forward-looking statements in this presentation are only predictions. We have based these forward-looking statements largely on our current expectations and projections about future events and financial trends that we believe may affect our business, financial condition, and results of operations. These forward-looking statements speak only as of the date of this press release and are subject to a number of risks, uncertainties and assumptions, some of which cannot be predicted or quantified and some of which are beyond our control, including, among others: our ability to successfully advance our current and future product candidates through development activities, preclinical studies, and clinical trials; our ability to obtain FDA acceptance for our future IND submissions and commence clinical trials on expected timelines, or at all; our reliance on the maintenance of certain key collaborative relationships for the manufacturing and development of our product candidates; the timing, scope and likelihood of regulatory filings and approvals, including final regulatory approval of our product candidates; the impact of the COVID-19 pandemic, geopolitical issues and inflation on our business and operations, supply chain and labor force; the performance of third parties in connection with the development of our product candidates, including third parties conducting our future clinical trials as well as third-party suppliers and manufacturers; our ability to successfully commercialize our product candidates and develop sales and marketing capabilities, if our product candidates are approved; and our ability to maintain and successfully enforce adequate intellectual property protection. These and other risks and uncertainties are described more fully in the Risk Factors section of our most recent filings with the Securities and Exchange Commission and available at http://www.sec.gov. You should not rely on these forward-looking statements as predictions of future events. The events and circumstances reflected in our forward-looking statements may not be achieved or occur, and actual results could differ materially from those projected in the forward-looking statements. Moreover, we operate in a dynamic industry and economy. New risk factors and uncertainties may emerge from time to time, and it is not possible for management to predict all risk factors and uncertainties that we may face. Except as required by applicable law, we do not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.

For More Information:

Company: Elizabeth Krutoholow investor.relations@centurytx.com

Investors: Melissa Forst/Maghan Meyers century@argotpartners.com

Media: Joshua R. Mansbach century@argotpartners.com

Continue reading here:
Century Therapeutics Receives Study May Proceed Notification from FDA for CNTY-101, the First Allogeneic Cell Therapy Product Candidate Engineered to...

This company is about to grow new organs in a person for the first time – MIT Technology Review

And the lymph nodes near the liver are close enough to receive chemical distress signals sent out by the dying tissue of a diseased liver, says Lagasse. These signals are meant to encourage any remaining healthy liver tissue to regenerate, but this doesnt work in cases of severe disease. However, the signals do appear to help along the growth of liver tissue in neighboring lymph nodes.

Its incredible, says Gouon-Evans. Having this little incubator in the body [that can grow organs] is just amazing.

LYGENESIS

Around five years ago, Lagasse, along with entrepreneur and drug developer Michael Hufford and transplant surgeon Paulo Fontes, founded LyGenesis to take the technology further. The team are exploring the use of lymph nodes to grow new thymuses, kidneys, and pancreases.

But the companys priority is livers. Over the last 10 years, members of the team have collected promising evidence that suggests they can use their approach to grow new mini livers in mice, pigs, and dogs. The mini livers dont grow indefinitelythe body has an internal regulator that stops liver growth at a certain point, which is why healthy livers dont overshoot when they regenerate.

The teams research in mice with a genetic liver disorder has shown that most of the cells injected into a lymph node will stay there but some will migrate to the liver, providing there is enough healthy liver tissue remaining. These migrating cells can help the remaining liver tissue regenerate and heal. When this happens, the new mini liver in the lymph node will shrink, keeping the total amount of liver tissue in balance, says Lagasse.

Other studies have focused on pigs and dogs that have the blood supply to the liver diverted, causing the organ to die. Injecting liver cells into the animals lymph nodes will eventually rescue their liver function.

In the pig study, for example, the team first surgically diverted the blood supply away from the liver in six animals. Once the pigs had recovered from surgery, the team injected healthy liver cells into their lymph nodes. The doses ranged from 360 million cells injected across three lymph nodes to 1.8 billion cells across 18 lymph nodes.

Within a couple of months, all the animals appeared to show recovery from their liver damage. Tests suggested their liver function had improved. And when the team later performed autopsies on the animals, the new organs in the lymph nodes looked very much like miniature healthy livers, each up to around 2% of the size of a typical adult liver. Other studies suggest it takes around three months for the treatment to have significant benefits.

Excerpt from:
This company is about to grow new organs in a person for the first time - MIT Technology Review

Inside the race to make human sex cells in the lab – MIT Technology Review

But there are other hurdlessome so challenging that many scientists have given up. For one thing, nudging the stem cells in the right direction requires, it seems, a unique touch and expertise. Not just anyone will be able to make egg and sperm cells in the lab, says Saitou.

Saitou and Hayashi, now at Kyushu University, lead world-renowned teams of extraordinary skill. Their achievements might not have been possible without the contributions of Hiroshi Ohta, for example. Ohta is an expert in anesthetizing newborn mice using ice, performing intricate surgery on them, and injecting cells into the animals miniature gonads. The entire procedure must be completed within five minutes or the animals die. Only a few people have such skills, which take months to develop. I think our group was kind of lucky, says Saitou. It was a get-together of many talented scientists.

The work is hampered by the lack of in-depth knowledge about how the primitive forms of egg and sperm cells develop naturally in the embryoa process that is far from fully worked out in humans. Some of the embryos cells begin to differentiate into these primitive sex cells at around 14 days. But in some countries, it is illegal for researchers to even grow human embryos beyond 14 days. They would send me to jail if I went beyond day 14, says Azim Surani, who is working with precursors to artificial sex cells at the University of Cambridge in the UK.

The problem, from a research point of view, is that the 14-day rule comes in just as the embryos start to get interesting, says Surani. Without being able to easily study the critical process of how primitive cells begin forming egg and sperm cells, scientists are limited in their ability to mimic it in the lab.

Even if scientists were able to study embryos more freely, some mysteries would remain. Once the cells that make eggs and sperm are created, they are held in a kind of suspended animation until puberty or ovulation. What happens to them in the years in between? And how important is this phase for the health of mature eggs and sperm? The honest answer is we dont know, says Surani.

The stem cells in the lab must also be generated and cared for under precise conditions. To survive, they must be bathed in a cocktail of nutrients that must be replaced every day. Its very time consuming and labor intensive and it takes a lot of money, says Bjorn Heindryckx at Ghent University in Belgium, one of the scientists who have given up on creating human eggs this way in the lab. The outcome was too limited for the effort and the money that we spent on it, he says.

Part of the challenge is that for the precursor stem cells to develop into fully matured egg or sperm cells, they must be placed in an environment mimicking that of newly developing ovaries or testes. Researchers studying mice use tissue taken from mouse embryos to induce the stem cells to differentiate into sex cells. But similarly using human tissue from discarded embryos is ethically and legally problematic. So scientists are working on ways to create the right environment without using tissue from embryos.

The upshot is that it will likely take a highly skilled team years of dedicated research. Its not impossible, but it would not be easy to do, says Surani.

Read more here:
Inside the race to make human sex cells in the lab - MIT Technology Review

Global Cell Banking Outsourcing Market to Grow at a CAGR of ~18% during 2022-2031; Market to Expand Owing to the Development of Advanced Cell…

New York, Aug. 23, 2022 (GLOBE NEWSWIRE) -- Kenneth Research has published a detailed market report on Global Cell Banking Outsourcing Market for the forecast period, i.e., 2022 2031, which includes the following factors:

Global Cell Banking Outsourcing Market Size:

The global cell banking outsourcing market generated the revenue of approximately USD 7200.1 million in the year 2021 and is expected to garner a significant revenue by the end of 2031, growing at a CAGR of ~18% over the forecast period, i.e., 2022 2031. The growth of the market can primarily be attributed to the development of advanced preservation techniques for cells, and increasing adoption of regenerative cell therapies for the treatment of chronic diseases such as cancer. Additionally, factors such as growing demand for gene therapy, and increasing worldwide prevalence of cancer are expected to drive the market growth. According to the World Health Organization, nearly 10 million people died of cancer across the globe in 2020. The most recurrent cases of deaths because of cancer were lung cancer which caused 1.80 million deaths, colon, and rectum cancer which caused 916 000 deaths, liver cancer which caused 830 000 deaths, stomach cancer which caused 769 000 deaths, and breast cancer which caused 685 000 deaths. Furthermore, it was noticed that about 30% of cancer cases in low and lower-middle income nations are caused by cancer-causing diseases such the human papillomavirus (HPV) and hepatitis.

Get a Sample PDF of This Report @ https://www.kennethresearch.com/sample-request-10070777

Global Cell Banking Outsourcing Market: Key Takeaways

Increasing Geriatric Population across the Globe to Boost Market Growth

Increasing demand for stem cell therapy, and increasing biopharmaceutical production are estimated to fuel the growth of the global cell banking outsourcing market. Among the geriatric population around the world, the demand of stem cell therapy is at quite a high rate. Hence, growing geriatric population across the globe is also expected be an important factor to influence the market growth. According to the data by World Health Organisation (WHO), the number and proportion of geriatric population, meaning the people aged 60 years and older in the population is rising. The number of people aged 60 years and older was 1 billion in 2019. This number is estimated to increase to 1.4 billion by 2030 and 2.1 billion by 2050.

In addition to this, increasing prevalence of chronic diseases, supportive initiatives by governments around the world, and growing awareness about stem cell banking are predicted to be major factors to propel the growth of the market. The growth of the global cell banking outsourcing market, over the forecast period, can be further ascribed to the rising investments in the R&D activities to continuously bring up more feasible solutions for medical procedures. According to research reports, since 2000, global research and development expenditure has more than tripled in real terms, rising from approximately USD 680 billion to over USD 2.5 trillion in 2019.

Browse to access In-depth research report on Global Cell Banking Outsourcing Market with detailed charts and figures: https://www.kennethresearch.com/report-details/cell-banking-outsourcing-market/10070777

Global Cell Banking Outsourcing Market: Regional Overview

The global cell banking outsourcing market is segmented into five major regions including North America, Europe, Asia Pacific, Latin America, and the Middle East and Africa region.

Advanced Healthcare Facilities Drove Market in the North America Region

The market in the North America region held the largest market share in terms of revenue in the year 2021. The growth of the market in this region is majorly associated with the increasing number of pharmaceutical companies & manufacturers in the region, and increasing awareness for the use of stem cells as therapeutics. Increasing number of bone marrow and cord blood transplants throughout the region is also estimated to positively influence the market growth. It was noted that, 4,864 unrelated and 4,160 related bone marrow and cord blood transplants were performed in the United States in 2020.

Increasing Prevalence of Chronic Diseases to Influence Market Growth in the Asia Pacific Region

On the other hand, market in the Asia Pacific region is estimated to grow with the highest CAGR during the forecast period. The market in this region is driven by the increasing investment in biotechnology sector by government and private companies specifically in countries such as China, India, and Japan. Moreover, the increasing pool of patient with chronic diseases, such as cancer, and the ongoing research & development activities for cancer treatment is expected to propel the growth of the market. Further, increasing percentage of regional health expenditure contributing to the GDP is also estimated to be a significant factor to influence the growth of the cell banking outsourcing market in the Asia Pacific region. As per The World Bank, in the year 2019, share of global health expenditure in East Asia & Pacific region accounted to 6.67% of GDP.

Get a Sample PDF of the Global Cell Banking Outsourcing Market @ https://www.kennethresearch.com/sample-request-10070777

The study further incorporates Y-O-Y growth, demand & supply and forecast future opportunity in:

Middle East and Africa (Israel, GCC [Saudi Arabia, UAE, Bahrain, Kuwait, Qatar, Oman], North Africa, South Africa, Rest of Middle East and Africa).

Global Cell Banking Outsourcing Market, Segmentation by Bank Phase

The bank storage segment held the largest market share in the year 2021 and is expected to maintain its share by growing with a notable CAGR during the forecast period. The market growth is anticipated to be driven by the development of effective preservation technologies such as cryopreservation technique. Cryopreservation is a technique in which low temperature is used to preserve the living cells and tissue for a longer time. With the growing healthcare expenditure per capita across the world, demand for bank storage increasing notably. As sourced from The World Bank, in 2019, worldwide health expenditure per capita was USD 1121.97.

Access full Report Description, TOC, Table of Figure, Chart, etc. @ https://www.kennethresearch.com/sample-request-10070777

Global Cell Banking Outsourcing Market, Segmentation by Product

The adult cell banking segment is estimated to hold a substantial market share in the global cell banking outsourcing market during the forecast period. The growth of this segment can be attributed to the significant prevalence of chronic diseases among the adults around the globe. For instance, according to the National Library of Medicine 71.8% of adult population suffered from cardiovascular diseases, 56% had diabetes, and 14.7% adults had arthritis as of 2020.

Global Cell Banking Outsourcing Market, Segmentation by Cell Type

Global Cell Banking Outsourcing Market, Segmentation by Bank Type

Few of the well-known market leaders in the global cell banking outsourcing market that are profiled by Kenneth Research are SGS SA, WuXi AppTec, LifeCell International Pvt. Ltd., BSL Bioservice, LUMITOS AG, Cryo-Cell International, Inc., REPROCELL Inc, CORDLIFE GROUP LIMITED, Reliance Life Sciences, and Clean Biologics and others. Enquiry before Buying This Report @ https://www.kennethresearch.com/sample-request-10070777

Recent Developments in the Global Cell Banking Outsourcing Market

Browse More Related Reports:

Immunoassays in R&D Market Segmentation by Application (Cancer Research, Infectious Diseases, Autoimmune Diseases, Endocrinology, and Others); by End-Use (Academic Laboratories & Institutions, Biotechnology & Pharmaceutical Companies, and Others); and by Product & Services (Analyzers, Software & Services, and Kits & Reagents)-Global Demand Analysis & Opportunity Outlook 2031

Global Vaginal Slings Market Segmentation by End-Use (Clinics, Hospitals, and Ambulatory Surgical Centers); and by Slings Type (Conventional, and Advanced Vaginal Slings)-Demand Analysis & Opportunity Outlook 2031

Chemotherapy Market Segmentation by Cancer Type (Lung, Breast, Stomach, and Ovarian Cancer, and Others); by Gender Type (Male, and Female); and by Product Type (Pump, Cannula, Catheter, and Others)-Global Demand Analysis & Opportunity Outlook 2022-2031

Lancet Market Segmentation by Type (Push Button Safety, Pressure Activated Safety, Personal, and Side Button Safety); by Application (Glucose Testing, Hemoglobin Testing, and Coagulation Testing); and by End Users (Homecare, Hospitals & Clinics, Blood Banks, and Others)-Global Demand Analysis & Opportunity Outlook 2031

eClinical Solutions Market Segmentation by Product Type {Clinical Data Management Systems (CDMS), Electronic Data Capture (EDC), Clinical Trial Management Systems (CTMS), Electronic Clinic Outcome Assessment0 (eCOA), Randomization & Trial Supply Management (RTSM), Electronic Patient-Reported Outcome (ePRO), Electronic Trial Master File (eTMF), and Clinical Analytics Platform}; by Delivery Mode (Web-based, On-Premise, and Cloud); and by End users {Pharma & Biotech, Hospitals, and Contract Research Organizations (CROs)}-Global Demand Analysis & Opportunity Outlook 2031

About Kenneth Research

Kenneth Research is a leading service provider for strategic market research and consulting. We aim to provide unbiased, unparalleled market insights and industry analysis to help industries, conglomerates and executives to take wise decisions for their future marketing strategy, expansion and investment, etc. We believe every business can expand to its new horizon, provided a right guidance at a right time is available through strategic minds. Our out of box thinking helps our clients to take wise decision so as to avoid future uncertainties.

Contact for more Info:

AJ Daniel

Email: info@kennethresearch.com

U.S. Phone: +1 313 462 0609

Web: https://www.kennethresearch.com/

View post:
Global Cell Banking Outsourcing Market to Grow at a CAGR of ~18% during 2022-2031; Market to Expand Owing to the Development of Advanced Cell...

Myeloid Therapeutics Strengthens Senior Team to Support the Expansion of its Manufacturing Capabilities – PR Newswire

CAMBRIDGE, Mass., Aug. 23, 2022 /PRNewswire/ -- Myeloid Therapeutics, Inc. ("Myeloid"), a clinical stage mRNA-immunotherapy company harnessing the power of myeloid and innate biology to engineer novel therapies that elicit a broad immune response for cancer and autoimmune diseases, today announced that it has expanded its manufacturing capabilities with the appointments of Jerome Chal, Ph.D., as Vice President, Process Development, and Eric Chapdelaine as Vice President, CMC Operations.

"We are thrilled to continue to attract the highest levels of talent to Myeloid, as we expand our manufacturing capabilities to support our clinical-stage cell therapy programs and the advancement of our in vivo cell programming programs into the clinic next year," said Daniel Getts, Ph.D., CEO of Myeloid. "Jerome and Eric bring a diverse set of skills with significant quality and CMC experience at leading companies like Vertex, Alnylam, and Genzyme. As senior leaders, they will provide invaluable stewardship as we advance and expand our pipeline and read out more clinical data. We are thrilled to welcome them to the Myeloid team, and we look forward to their contributions to our technology and the cell therapy field generally."

Jerome Chal, Ph.D., Vice President, Process Development

Dr. Chal brings to Myeloid over twenty years of experience in the biotechnology industry, with a focus on CMC leadership across multiple therapeutic modalities, including cell and gene therapies, with landmark achievements in the field. Prior to Myeloid, Jerome was Executive Director, Analytical Sciences at Vertex Pharmaceuticals, where he provided strategic leadership to a multi-site and multi-department CMC organization across the T1D, blood disorders and DMD programs, along with end-to-end implementation of all analytical CMC activities from preclinical to pivotal stage. Prior to Vertex, Dr. Chal was Associate Director, Cell and Analytical Development at Semma Therapeutics, where he directed cell, process and assay development programs for cell therapy products based on a proprietary preclinical-stage PSC-based pancreatic islets (SC-islets) platform. Earlier in his career, he held positions of increasing responsibility at Coyne Scientific, IGBMC in Strasbourg, France, and faculty scientist at Brigham and Women's Hospital, Harvard Medical School. Dr. Chal received his MS in Biology-Biochemistey from ENS Paris, Paris 7 University, an MS in Molecular and Cellular Developmental Biology from Sorbonne University, and a Ph.D. in Stem Cell and Developmental Biology from Sorbonne University/Stowers Institute, Kansas City, MO. Dr. Chal is a decorated scientist with multiple patents and over twenty peer-reviewed publications in the fields of cell and gene therapy.

Eric Chapdelaine , Vice President, CMC Operations

Mr. Chapdelaine has over twenty years of experience leading Manufacturing, CMC, Quality (QC+QA), Analytical Development, and Regulatory Affairs organizations in cell and gene therapy. Eric has overseen and led IND and NDA filings for multiple products that received approval in the US and EU. Prior to joining Myeloid, Eric was Senior Vice President of Operations at iVexSol, Inc., where he designed and optimized the manufacturing facility layout, flow and associated office space, as well as overseeing the buildout of the cGMP manufacturing facility. Earlier, he was Vice President of Manufacturing at Genprex, Inc., where he was responsible for translating the corporate strategy into supportive strategies for global pharmaceutical manufacturing, packaging, supply chain, logistics and quality control from early product development through global manufacturing for commercial products. He has also held senior positions at Cognate Bioservices, Inc., and Alnylam Pharmaceuticals, where he was responsible for authoring CMC sections for regulatory submissions, IMPD/IND/NDA, for Patisiran and Givosiran, which are the first two RNA interference therapeutics approved for commercialization in the US and EU. Eric started his career in positions within quality control, manufacturing and CMC at Genzyme Corporation, VaxInnate Corporation, and Pfizer, Inc, respectively. Mr. Chapdelaine holds a Bachelor's of Science from the University of Massachusetts at Amherst and a Masters of Science, Chemistry, from Vanderbilt University.

About Myeloid Therapeutics

Myeloid Therapeutics is a clinical stage mRNA-immunotherapy company harnessing the power of myeloid cell biology to engineer new therapeutic alternatives for patients with cancer and autoimmune diseases.Integrating the fields of RNA biology, immunology, and medicine, the Company's proprietary platform provides clinical solutions that match therapeutic modalities to disease conditions, including use of autologous cell therapies, in vivo cell programming using mRNA, RNA-based gene-editing using RetroT and multi-targeted biologics. Myeloid is advancing a broad portfolio of clinical and preclinical candidates designed to enable full immune system responses. Myeloid has entered into strategic partnerships with Prime Medicine and Acuitas and is supported by well-known biotechnology investors. Myeloid is headquartered in Boston, MA. For more information, visit https://www.myeloidtx.com/.

Investor and Media ContactAmy Conrad Juniper Point [emailprotected] 858-914-1962

SOURCE Myeloid Therapeutics

Here is the original post:
Myeloid Therapeutics Strengthens Senior Team to Support the Expansion of its Manufacturing Capabilities - PR Newswire

Is Regen BioPharma a Good Investment? – Value the Markets

Regen BioPharma recently filed a patent related to a mRNA cancer immunotherapeutic vaccine. Does the market growth potential make RGBP a good investment opportunity?

Oncology stock Regen BioPharma (OTCMKTS: RGBP) is a penny stock listed on the OTC pink market.

Regen BioPharma, Inc focuses on the development of translational medicine platforms for the commercialization of stem cell therapies. The company aims to obtain regulatory approval and execute clinical trials in cell therapy. Regen BioPharma develops medicines for diabetes, heart-related illness, circulatory issues, and Chronic Obstructive Pulmonary Disease (COPD).

The company actively identifies small molecules that inhibit or express NR2F6 leading to immune cell activation for oncology applications and immune cell suppression for autoimmune diseases.

Regen BioPharma is in the early stages of developing its products and therapies. The business was incorporated in 2012 and is based in La Mesa, California.

Regen has identified and filed patents on small molecules that activate and inhibit a novel gene (NR2F6) which controls how the immune system reacts to cancer cells and inflammatory responses.

Its progress is currently pre-clinical, so commercialization is a long way off.

The conditions Regen hopes its investigations will eventually be able to address include:

Initial indications (inhibitor): bladder cancer, myelodysplastic syndrome, lung cancer

Initial indications (activator): rheumatoid arthritis, inflammatory bowel disease, psoriasis

Additional indications include solid tumors, acute leukemia and GVHD

Regen currently has the following products in its pipeline:

HemaXellerate: Cleared to proceed to Phase I/II clinical trials (stimulates blood production in patients whose bone marrow is not correctly functioning)

dCellVax: a breast cancer therapy (a dendritic cell-based immunotherapy that stimulates the patient's immune system)

DiffronC: a novel form of therapy called differentiation therapy that is expected to have much milder toxicity than chemotherapy.

For the quarterly period ended June 30, 2022

Revenues: $31.2k (up 6.8% Y/Y)

Total Revenues: $58.7k (up 3.7% Y/Y)

Operating Loss: -$58.9k (from -$42.4k Y/Y)

Between September 2021 and June 2022, Regen saw its cash decline by 85%. This is primarily due to paying off debts and using cash to fund the business.

Regen BioPharma is expected to report its Fiscal Year 2022 results on December 22, 2022.

RGBP stock has five billion shares outstanding.

On July 19, 2022, Regen BioPharma issued 54.5 million shares to Coventry Enterprises LLC to settle $180.5k in convertible debt.

On August 9, 2022, KCL Therapeutics, Inc. (a wholly owned subsidiary of Regen BioPharma) filed a provisional patent application with the US Patent and Trademark Office covering Regen's novel approach for enabling chimeric antigen receptor (CAR)-T cell-based therapies to kill solid tumors through prevention of a process called "T cell exhaustion." T cells are part of the immune system and develop from stem cells in the bone marrow.

Regen BioPharma's patent application protects the use of its patented survivin mRNA both as a stand-alone vaccine and as an immunotherapy.

David Koos, Chairman and CEO of Regen BioPharma, commented:

The currently filed application discloses means of significantly increasing efficacy by combining modified-mRNA with unique cellular immunotherapy as well as adjuvant approaches,

CAR-T cells have been in the clinic for 6 years, but they have hardly made a dent in treating solid tumors,

We hope that through the diligent work of our scientists and collaborators that advancements such as what we announced today will pave the way to bring this incredible cellular immunotherapy to patients suffering from solid tumors such as lung, colon, prostate and others.

Regen BioPharma is not profitable but makes a small income from royalties and anniversary fees under license agreements.

The company holds shares in Oncology Pharma (OTCMKTS: ONPH) and Zander Therapeutics.

Zander Therapeutic has been granted an exclusive license to develop and commercialize IP controlled by Regen BioPharma for non-human veterinary therapeutic use.

Meanwhile, Regen has granted an exclusive license to Oncology Pharma, Inc. to develop and commercialize "Antigen specific mRNA cellular cancer vaccines" for treating pancreatic cancer.

And KCL Therapeutics, Inc. has granted an exclusive license to Oncology Pharma, Inc. to develop and commercialize certain intellectual property for the treatment of colon cancer.

David Koos, Chairman and CEO of Regen BioPharma, said:

We are very proud of our broad, deep and cutting-edge patent portfolio,

We are continuing to develop additional IP focused on immunotherapy of cancer and look forward to developing some of these technologies in-house and some of them via outlicensing.

RGBP stock has a price-to-earnings ratio (P/E) of 48.7. Its price-to-book-value (P/BV) is negative, which means it owes more than it owns. RGBP stock does not come with a shareholder dividend.

Over the past year, Regen BioPharma Inc (RGBP) has traded between $0 and $0.08. Today the stock trades at $0.0096 and has a market cap of $48m.

Year-to-date, the RGBP stock price is down by -50%, while the S&P 500 is down -13.73% over the same period.

Furthermore, on August 22, 2022, Regen BioPharma (RGBP-US) closed down -86.3% off its 52-week high vs. the subsector average change of -39.4%.

Regen BioPharma plans to rapidly advance novel technologies through pre-clinical and Phase I/ II clinical trials. If it can successfully advance its small molecule therapies for treating cancer and autoimmune disorders, the addressable market is significant, and it could potentially make money.

Immunotherapy of cancer represents a vast market currently being led by the class of drugs called "checkpoint inhibitors" and "CAR-T" cells. To date, there is no mRNA immunotherapy available for treating cancer.

A stock with a beta higher than 1.0 is expected to be more volatile than the S&P 500. RGBP stock has a beta of 3.75, which accurately reflects its volatile nature.

Its price-to-book-value (P/BV) is negative, meaning it owes more than it owns.

RGBP common stock is a "penny stock," and therefore comes with the associated risk in dealing in penny stocks, such as lack of liquidity.

The economy is experiencing a period of high inflation, which could lead to interest rate rises and/or a recession. This is not conducive to raising funds at favorable rates and could adversely affect the company's ability to raise the money needed to conduct its research and trials.

There is a lack of liquidity in trading a stock such as Regen BioPharma. Therefore, there will probably be a large spread between the bid and ask price of RGBP shares. This means if you want to sell your shares in a hurry, you may not get a reasonable price for them.

Furthermore, the company has a history of issuing additional stock, which dilutes existing shareholder value.

Regen BioPharma stock seems like a hugely speculative investment with a lot of shareholder risk in the current investing environment.

Author: Kirsteen Mackay

This article does not provide any financial advice and is not a recommendation to deal in any securities or product. Investments may fall in value and an investor may lose some or all of their investment. Past performance is not an indicator of future performance.

Kirsteen Mackay does not hold any position in the stock(s) and/or financial instrument(s) mentioned in the above article.

Kirsteen Mackay has not been paid to produce this piece by the company or companies mentioned above.

Digitonic Ltd, the owner of ValueTheMarkets.com, does not hold a position or positions in the stock(s) and/or financial instrument(s) mentioned in the above article.

Digitonic Ltd, the owner of ValueTheMarkets.com, has not been paid for the production of this piece by the company or companies mentioned above.

More:
Is Regen BioPharma a Good Investment? - Value the Markets

Personalized Regenerative Medicine Celebrates the Grand Reopening of their San Clemente Location – Benzinga

Personalized Regenerative Medicine, the San Clemente office of Dr. Steenblock, announces its grand reopening celebration. This event will be held at 187 Av. La Pata, San Clemente, CA 92673 on Thursday, September 1st from 5:30 to 7:30 PM. The celebration produced by Rachel Dares PR includes a red carpet, a ribbon-cutting ceremony, a DJ, delectable food, refreshments, press coverage, VIP swag bags, and raffle prizes. Come enjoy this wonderful occasion with the staff of Dr. Steenblock, his patients, generous sponsors, the local community, San Clemente Chamber of Commerce members, and local officials.

Dr. David Steenblock is widely regarded as one of the country's foremost stem cell experts, specializing in the treatment of ALS, TBI, Cerebral Palsy, Osteoarthritis, and stroke. Dr. David Steenblock, D.O., is an interdisciplinary medical genius who is exploring uncharted territory to find the best treatment for his patients.

The San Clemente Chamber of Commerce will host a Ribbon Cutting Ceremony as part of the festivities. Author, humanitarian, superfood creator, TV show, and radio personality Dr. Tony O'Donnell will serve as the event's emcee. Delicious food and beverages will be provided by local businesses, including mouthwatering appetizers sponsored by BASANTI Indian Cuisine, signature cheese boards by Riviera, delicious chips & salsa from Chelas Mexican Restaurant, scrumptious fresh baked goods from Elisa Marie Baking, thirst-quenching bottled water from MOTUS Physical Therapy, appetizing deli platters from Primerica, and delightful salad trays from The Kitchen Lady. The party will also feature music by DJ George.

The first 25 early attendees will receive VIP Swag Bags from Orange Sculpting inclusive of Dr.Steenblocks break-through supplement, Stemgevity which utilizes encapsulated stem cell growth activators to help patients relieve pain, promote tissue regeneration, stimulate collagen, and see fast results in overall youth factors.

With the generous sponsorship fromHome Saver Realty, the event will also feature great photo opportunities with a red carpet and backdrop banner. Amazing raffle prizes will be given out to lucky winners throughout the evening provided by Laguna Aesthetics, The Kitchen Lady, Orange Sculpting, Radiant Greens, The Dorothea Clinic and Rachel Dares PR!

The office of Personalized Regenerative Medicine invites everyone to witness and experience the future of regenerative medicine and to see for yourselves what all the excitement is about! RSVP now on Event Brite!

Media Contact Company Name: Rachel Dares PR Contact Person: RACHEL B DARES Email: Send Email Phone: 7147189043 Address:16480 Bake Parkway #106 City: Irvine State: CA Country: United States Website: http://www.RachelDaresPR.com

Press Release Distributed by ABNewswire.com To view the original version on ABNewswire visit: Personalized Regenerative Medicine Celebrates the Grand Reopening of their San Clemente Location

Read more:
Personalized Regenerative Medicine Celebrates the Grand Reopening of their San Clemente Location - Benzinga

A 5th person is likely cured of HIV, and another is in long-term remission – NBC News

Two new cases presented Wednesday at the International AIDS Conference in Montreal have advanced the field of HIV cure science, demonstrating yet again that ridding the body of all copies of viable virus is indeed possible, and that prompting lasting viral remission also might be attainable.

In one case, scientists reported that a 66-year-old American man with HIV has possibly been cured of the virus through a stem cell transplant to treat blood cancer. The approach which has demonstrated success or apparent success in four other cases uses stem cells from a donor with a specific rare genetic abnormality that gives rise to immune cells naturally resistant to the virus.

In another case, Spanish researchers determined that a woman who received an immune-boosting regimen in 2006 is in a state of what they characterize as viral remission, meaning she still harbors viable HIV but her immune system has controlled the viruss replication for over 15 years.

Experts stress, however, that it is not ethical to attempt to cure HIV through a stem cell transplant a highly toxic and potentially fatal treatment in anyone who is not already facing a potentially fatal blood cancer or other health condition that would make them a candidate for such a treatment.

While a transplant is not an option for most people with HIV, these cases are still interesting, still inspiring and illuminate the search for a cure, Dr. Sharon Lewin, an infectious disease specialist at the Peter Doherty Institute for Infection and Immunity at the University of Melbourne, told reporters on a call last week ahead of the conference.

There are also no guarantees of success through the stem cell transplant method. Researchers have failed to cure HIV using this approach in a slew of other people with the virus.

Nor is it clear that the immune-enhancing approach used in the Spanish patient will work in additional people with HIV. The scientists involved in that case told NBC News that much more research is needed to understand why the therapy appears to have worked so well in the woman it failed in all participants in the clinical trial but her and how to identify others in whom it might have a similar impact. They are trying to determine, for example, if specific facets of her genetics might favor a viral remission from the treatment and whether they could identify such a genetic profile in other people.

The ultimate goal of the HIV cure research field is to develop safe, effective, tolerable and, importantly, scalable therapies that could be made available to wide swaths of the global HIV population of some 38 million people. Experts in the field tend to think in terms of decades rather than years when hoping to achieve such a goal against a foe as complex as this virus.

Diagnosed with HIV in 1988, the man who received the stem cell transplant is both the oldest person to date 63 years old at the time of the treatment and the one living with HIV for the longest to achieve an apparent success from a stem cell transplant cure treatment.

The white male dubbed the City of Hope patient after the Los Angeles cancer center where he received his transplant 3years ago has been off of antiretroviral treatment for HIV for 17 months.

We monitored him very closely, and to date we cannot find any evidence of HIV replicating in his system, said Dr. Jana Dickter, an associate clinical professor in the Division of Infectious Diseases at City of Hope. Dickter is on the patients treatment team and presented his case at this weeks conference.

This means the man has experienced no viral rebound. And even through ultra-sensitive tests, including biopsies of the mans intestines, researchers couldnt find any signs of viable virus.

The man was at one time diagnosed with AIDS, meaning his immune system was critically suppressed. After taking some of the early antiretroviral therapies, such as AZT, that were once prescribed as individual agents and failed to treat HIV effectively, the man started a highly effective combination antiretroviral treatment in the 1990s.

In 2018, the man was diagnosed with acute myeloid leukemia, or AML. Even when HIV is well treated, people with the virus are still at greater risk of a host of cancers that are associated with aging, including AML and other blood cancers. Thanks to effective HIV treatment, the population of people living with the virus in the U.S. is steadily aging; the majority of people diagnosed with HIV is now older than 50.

He was treated with chemotherapy to send his leukemia into remission prior to his transplant. Because of his older age, he received a reduced intensity chemotherapy to prepare him for his stem cell transplant a modified therapy that older people with blood cancers are better able to tolerate and that reduces the potential for transplant-related complications.

Next, the man received the stem cell transplant from the donor with an HIV-resistant genetic abnormality. This abnormality is seen largely among people with northern European ancestry, occurring at a rate of about 1% among those native to the region.

According to Dr. Joseph Alvarnas, a City of Hope hematologist and a co-author of the report, the new immune system from the donor gradually overtook the old one a typical phenomenon.

Some two years after the stem cell transplant, the man and his physicians decided to interrupt his antiretroviral treatment. He has remained apparently viable-virus free ever since. Nevertheless, the study authors intend to monitor him for longer and to conduct further tests before they are ready to declare that he is definitely cured.

A second report presented at the Montreal conference detailed the case of a 59-year-old woman in Spain who is considered to be in a state of viral remission.

The woman was enrolled in a clinical trial in Barcelona in 2006 of people receiving standard antiretroviral treatment. She was randomized to also receive 11 months of four therapies meant to prime the immune system to better fight the virus, according to Nria Climent, a biologist at the University of Barcelona Hospital Clinic, who presented the findings.

Then Climent and the research team decided to take the woman off her antiretrovirals, per the studys planned protocol. She has now maintained a fully suppressed viral load for over 15 years. Unlike the handful of people either cured or possibly cured by stem cell transplants, however, she still harbors virus that is capable of producing viable new copies of itself.

Her body has actually controlled the virus more efficiently with the passing years, according to Dr. Juan Ambrosioni, an HIV physician in the Barcelona clinic.

Ambrosioni, Climent and their collaborators said they waited so long to present this womans case because it wasnt until more recently that technological advances have allowed them to peer deeply into her immune system and determine how it is controlling HIV on its own.

Its great to have such a gaze, Ambrosioni said, noting that the point is to understand what is going on and to see if this can be replicated in other people.

In particular, it appears that what are known as her memory-like NK cells and CD8 gamma-delta T cells are leading this effective immunological army.

The research team noted that they do not believe that the woman would have controlled HIV on her own without the immune-boosting treatment, because the mechanisms by which her immune cells appear to control HIV are different from those seen in elite controllers, the approximately 1 in 200 people with HIV whose immune systems can greatly suppress the virus without treatment.

Lewin, of Australias Peter Doherty Institute, told reporters last week that it is still difficult to judge whether the immune-boosting treatment the woman received actually caused her state of remission. Much more research is needed to answer that question and to determine if others might also benefit from the therapy she received, she said.

Over four decades, just five people have been cured or possibly cured of HIV.

The virus remains so vexingly difficult to cure because shortly after entering the body it infects types of long-lived immune cells that enter a resting, or latent, state. Because antiretroviral treatment only attacks HIV when infected cells are actively churning out new viral copies, these resting cells, which are known collectively as the viral reservoir and can stay latent for years, remain under the radar of standard treatment. These cells can return to an active state at any time. So if antiretrovirals are interrupted, they can quickly repopulate the body with virus.

The first person cured of HIV was the American Timothy Ray Brown, who, like the City of Hope patient, was diagnosed with AML. His case was announced in 2008 and then published in 2009. Two subsequent cases were announced at a conference in 2019, known as the Dsseldorf and London patients, who had AML and Hodgkin lymphoma, respectively. The London patient, Adam Castillejo, went public in 2020.

Compared with the City of Hope patient, Brown nearly died after the two rounds of full-dose chemotherapy and the full-body radiation he received. Both he and Castillejo had a devastating inflammatory reaction to their treatment called graft-versus-host disease.

Dr. Bjrn Jensen, of Dsseldorf University Hospital, the author of the German case study one typically overlooked by HIV cure researchers and in media reports about cure science said that with 44 months passed since his patient has been viral rebound-free and off of antiretrovirals, the man is almost definitely cured.

We are very confident there will be no rebound of HIV in the future, said Jensen, who noted that he is in the process of getting the case study published in a peer-reviewed journal.

For the first time, University of Cambridges Ravindra Gupta, the author of the London case studystated, in an email to NBC News, that with nearly five years passed since Castillejo has been off of HIV treatment with no viral rebound, he is definitely cured.

In February, a research team announced the first case of a woman and the first in a person of mixed race possibly being cured of the virus through a stem cell transplant. The case of this woman, who had leukemia and is known as the New York patient, represented a substantial advance in the HIV cure field because she was treated with a cutting-edge technique that uses an additional transplant of umbilical cord blood prior to providing the transplant of adult stem cells.

The combination of the two transplants, the study authors told NBC News in February, helps compensate for both the adult and infant donors being less of a close genetic match with the recipient. Whats more, the infant donor pool is much easier than the adult pool to scan for the key HIV-resistance genetic abnormality. These factors, the authors of the womans case study said, likely expand the potential number of people with HIV who would qualify for this treatment to about 50 per year

Asked about the New York patients health status, Dr. Koen van Besien, of the stem cell transplant program at Weill Cornell Medicine and New York-Presbyterian in New York City, said, She continues to do well without detectable HIV.

Over the past two years, investigators have announced the cases of two women who are elite controllers of HIV and who have vanquished the virus entirely through natural immunity. They are considered likely cured.

Scientists have also reported several cases over the past decade of people who began antiretroviral treatment very soon after contracting HIV and after later discontinuing the medications have remained in a state of viral remission for years without experiencing viral rebound.

Speaking of the reaction of the City of Hope patient, who prefers to remain anonymous, to his new HIV status, Dickter said: Hes thrilled. Hes really excited to be in that situation where he doesnt have to take these medications. This has just been life-changing.

The man has lived through several dramatically different eras of the HIV epidemic, she noted.

In the early days of HIV, he saw many of his friends and loved ones get sick and ultimately die from the disease, Dickter said. He also experienced so much stigma at that time.

As for her own feelings about the case, Dickter said, As an infectious disease doctor, Id always hoped to be able to tell my HIV patients that theres no evidence of virus remaining in their system.

FollowNBC HEALTHonTwitter&Facebook.

See more here:
A 5th person is likely cured of HIV, and another is in long-term remission - NBC News