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Reversal of biological age detected in mouse and human embryos – BioNews

Posted: July 6, 2021 at 1:56 am

5 July 2021

Germline cells seem to reset their biological clocks around the time of embryoimplantation, not when generating gametes, as previously thought.

Scientists measured an increase in genetic damage in embryonic cells during the early stages of embryogenesis in micebefore undergoing a total reset within a 'rejuvenation period', reversing any cell damage.

'This study uncovers a natural rejuvenation event during embryogenesis and suggests that the minimal biological age (ground zero) marks the beginning of organismal ageing,' wrote the researchers from Harvard Medical School and Brigham and Women's Hospital in Boston, Massachusetts.

Previously, it was thought that, unlike thesomatic cells which form our bodies, germline cells which differentiateinto either sperm or eggs were ageless and did not inherit genetic damage from their parent organisms. However, recent research has shown that germline cells do age and display hallmarks of genetic damage. Yet, babies do not inherit their parents' age, and start again from zero.

The team employed machine-learning algorithms as 'ageing clocks' to calculate the ages of human and mouse embryonic tissue by measuring the prevalence ofmethylation an epigenetic marker. These markers accumulate with age on certain sections of DNA and are influenced by environmental factors. Although these markers do not affect the DNA sequence, they can alter the way a gene is expressed and modify proteins produced.

Genetic data sets collected from mouse embryosduring different stages of embryonic development were analysed by these epigenetic ageing clocks. Data sets recorded from mouse embryos following fertilisation showed increased epigenetic ageing with time during the first six days of cell division. But, during its implantation within the uterus wall, the embryonic cells displayed a decrease in epigenetic damage, characteristic of a reversal in ageing.

The team were unable to perform the same experiment inhuman embryos but were able to compare methylation in human induced pluripotent stem cells and embryonic stem cell lines anddatasets detailing methylation in human fetal tissue samples and see that a similar reset appeared to have occurred.

The findings, published in the journal ScienceAdvances, have wide-reaching implications for aiding the treatment of age-related illnesses such as Alzheimer's and Parkinson's disease. These diseases feature cells with accelerated epigenetic ageing and through a greater understanding of these biological reset mechanisms, it is thought that epigenetic damage to these cells could be reversed. However, achieving this in practice may be challenging since knowledge of other causes of cellular ageing is needed.

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Genexine, Toolgen to co-develop CAR-NK cell gene therapy – Korea Biomedical Review

Posted: July 6, 2021 at 1:56 am

Genexine said Monday that it signed an agreement with Toolgen to co-develop and commercialize CAR-NK cell gene therapy using the latters CRISPR/Cas9 technology.

The two companies will co-own patent rights, utility model rights, trademark rights, and R&D data resulting from their co-development at a 5:5 equity ratio. They will also equally shoulder the cost incurred for the patent application, revision, registrations, and maintenance.

Genexine will make the most of Toolgens genetic scissors technology to develop new cell gene therapy to cure incurable diseases and expand its strategic pipelines.

CAR-NK cell gene therapy is an anticancer drug -- homogeneous natural killer (NK) cells whose immunity efficacy is strengthened through genetic manipulation administered to patients.

Unlike the CAR-T cell treatment that has to utilize the patients own T cell, the production of CAR-NK therapy is much cheaper, and mass production is also possible because NK cells could be abstracted from ready-made cell strain or induced pluripotent stem cells (IPSC). NK cells also can attack a wide variety of cancer cells, aside from targets introduced by CAR technology, as they have perception ability and offensive power.

Toolgen has confirmed that it could improve anticancer and immunity capacity through gene-editing of not only CAR-T, TCR-T but CAR-NK and various other anticancer immunocytes.

Genexine will make the best use of Toolgens CRISPR/Cas9 technology to develop new drugs of gene therapy to cure incurable diseases. This co-development MOU will be its start, Genexine CEO Sung Young-chul said. NK cell gene therapy, which is rapidly emerging as the next-generation anticancer immunotherapy, has cost advantages. By developing new drugs through cooperation with Toolgen, we will bring about a paradigm change on the anticancer treatment market.

Toolgen Kim Young-ho also said, Toolgen had put in a lot of effort to apply gene-editing technology to cell therapies, such as CAR-T and CAR-NK, and has recently produced successful results.

Kim added that if the two companies develop global blockbuster cell and gene therapies using Genexines know-how and Toolglens genetic scissors technology, it will maximize their corporate values.

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The cell therapy manufacturing market is projected to reach USD 14.5 billion by 2030, growing at – GlobeNewswire

Posted: July 6, 2021 at 1:56 am

London, July 01, 2021 (GLOBE NEWSWIRE) -- Roots Analysis has announced the addition of Cell Therapy Manufacturing Market (4th Edition), 2021-2030 report to its list of offerings.

Owing to the complex manufacturing processes, requirement of advanced production facilities and the growing demand for cell therapy products, developers are actively outsourcing certain manufacturing operations, in addition to expanding their in-house capabilities.

To order this 620 page report, which features 210+ figures and 280+ tables, please visit https://www.rootsanalysis.com/reports/view_document/cell-therapy-manufacturing/285.html

Key Market Insights

Around 200 organizations claim to be engaged in cell therapy manufacturingThe market landscape is dominated by industry players, which constitute 65% of the total number of stakeholders. Amongst these, over 25% companies are large firms.

280+ production facilities dedicated to cell therapies have been established worldwideNorth America has emerged as the manufacturing hub for cell therapies, with the presence of nearly 45% of the manufacturing facilities; this is followed by Europe (31%). Other emerging regions include China, Japan, South Korea and Australia.

90 cell therapy manufacturers are focused on immune cell and stem cell therapiesMost of the players in this domain are focused on manufacturing of T cell therapies, primarily CAR-T therapies, while the stem cell therapy manufacturers are primarily engaged in the production of adult stem cells and mesenchymal stem cell therapies

Presently, more than 70 companies carry out manufacturing at all scales of operation. Nearly 45% players have the required capabilities for commercial scale manufacturing. It is worth noting that all the industry players manufacture cell therapies required for clinical purposes.

35+ companies offer automated and closed systems to cell therapy developers More than 60 automated and closed systems are being used for cell therapy manufacturing. Organizations that are presently offering customized automated solutions for cell therapy processes / manufacturing are Fraunhofer Institute for Manufacturing Engineering and Automation IPA (Germany), KMC Systems (US), RoosterBio (US) and Mayo Clinic Center for Regenerative Medicine (US).

Several partnerships were established in this domain, during the period 2016-2021More than 180 deals have been inked during the given time period. A large proportion (34%) of the partnerships were related to manufacturing of cell therapies, followed by acquisitions (17%) and licensing agreements (14%).

Expansion activity in this domain has grown at a CAGR of 59%, between 2016 and 2021More than 75 facility expansions were reported during the given time period. Over 80% instances were related to the establishment of new facilities, followed by those involving the expansion of existing facilities (17%).

Role of big pharma players in this industry has evolved over the last few years; their initiatives increased at a CAGR of 41% during the period 2016-2020Several big pharma players have undertaken various initiatives focused on cell therapy manufacturing. Gilead sciences, Takeda Pharmaceutical and Novartis are some of the prominent big pharma players in this domain.

The currently available global cell therapy manufacturing capacity is estimated to be over 1.88 billion sq. ft. of dedicated cleanroom areaThe maximum (48%) installed capacity (in terms of cleanroom area) belongs to companies based in North America (48%); the region has higher number of players having multiple production facilities. This is followed by Asia Pacific (29%) and Europe (23%).

The demand for cell therapies is anticipated to grow at a CAGR of 22%, during 2021-2030Presently, the clinical demand for stem cell and CAR-T cell-based products is the highest; this trend is unlikely to change in the foreseen future as well. On the other hand, the demand for tumor cell, NK cell and dendritic cell therapies is expected to grow at a relatively faster pace, over the next decade.

By 2030, the market for commercial scale cell therapy manufacturing is likely to grow at an annualized rate of 31.5%Currently, North America and Europe capture more than 70% share of the overall market. Specifically, the cell therapy manufacturing market in Asia Pacific is driven by countries, such as China, Japan, South Korea, India and Singapore. It is worth noting that the current market in Asia Pacific is primarily driven by the clinical demand for cell therapies.

To request a sample copy / brochure of this report, please visit https://www.rootsanalysis.com/reports/view_document/cell-therapy-manufacturing/285.html

Key Questions Answered

The USD 14.5 billion (by 2030) financial opportunity associated with cell therapy manufacturing market has been analyzed across the following segments:

The report also features inputs from eminent industry stakeholders, according to whom, the manufacturing of cell therapies is largely being outsourced due to exorbitant costs associated with the setting-up of in-house expertise. The report includes detailed transcripts of discussions held with the following experts:

The research includes profiles of key players (industry and non-industry; listed below), featuring a brief overview company / organization, information on its manufacturing facilities, service portfolio, recent partnerships and an informed future outlook.

For additional details, please visithttps://www.rootsanalysis.com/reports/view_document/cell-therapy-manufacturing/285.html or email sales@rootsanalysis.com

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Multiple myeloma treatment: Newest options, drugs, and more – Medical News Today

Posted: July 6, 2021 at 1:56 am

Multiple myeloma is a type of cancer that affects certain white blood cells, called plasma cells, in the bone marrow. The condition can cause people to feel unwell and extremely tired. It can also affect their bones and other organs, such as the kidneys.

The American Cancer Society estimates that in the United States, doctors will diagnose multiple myeloma in 19,320 men and 15,600 women in 2021. Researchers are continually working on new treatments to extend life expectancy for people with this condition.

Multiple myeloma treatment has come a long way since the days when chemotherapy was the only option. Today, more therapies exist than ever before.

Doctors can now customize a treatment plan for each person based on their diagnosis and unique needs. Options may include chemotherapy with stem cell transplantation, immunotherapy drugs, targeted therapies, and novel drug combinations.

This article looks at multiple myeloma treatment options, including chemotherapy and other drugs, bone marrow transplants, and radiation therapy. It also looks at a persons possible outlook following a multiple myeloma diagnosis.

Multiple myeloma is a type of cancer that changes someones plasma cells. These specialized cells are typically located in the bone marrow, and they play an essential role in the immune system.

If a person has multiple myeloma, the altered, cancerous plasma cells build up in the bone marrow, which means that there is not enough space for healthy blood cells. Instead of making antibodies that protect an individual from invading germs, the cancerous cells make an abnormal protein called monoclonal immunoglobulin, or monoclonal protein (M-protein).

Doctors may find these elevated M-proteins while testing the blood for another condition. They may also make a diagnosis because they suspect that someone has multiple myeloma based on their symptoms.

Although there are no specific causes of multiple myeloma, it appears to be more common among males, Black people, and individuals aged 45 years and above.

When treating multiple myeloma, doctors typically use chemotherapy to prepare the body for a stem cell transplant.

Chemotherapy is a powerful form of treatment that kills cells that divide and multiply quickly, such as cancer cells. However, healthy cells that grow rapidly may also become chemotherapy targets. This includes cells in the bone marrow, digestive tract, reproductive system, and hair follicles.

When chemotherapy damages healthy cells, it can cause side effects, including:

People undergoing chemotherapy may also develop low blood counts as a side effect of treatment. Having a low blood count means that an individual has an increased risk of infection. They bruise and bleed more easily, feel fatigued, and could be short of breath.

Most chemotherapy side effects disappear once a person has completed treatment.

Monoclonal antibodies are a form of immunotherapy that uses someones immune system to fight the cancer cells by targeting specific proteins. Some monoclonal antibodies target the CD38 protein, while others target the SLAM57 protein. Myeloma cells have both proteins.

Monoclonal antibodies affect cancer cells directly and help the immune system attack them. Doctors often use this treatment in combination with other types of drugs.

These drugs can cause a reaction at the time of administration or a few hours after. Some symptoms of a reaction include:

Immunomodulating drugs regulate certain aspects of the immune system. They can prevent some types of growth signals for cancer cells, activate specific immune cells, and kill myeloma cells.

The first immunomodulating drug that researchers developed was called thalidomide, and it caused severe congenital disabilities. Researchers still question the safety of newer immunomodulating drugs related to thalidomide, and people can only receive them through special programs.

The side effects of immunomodulating drugs depend on the specific drug but can include:

Steroids are an essential treatment for all stages of multiple myeloma. At high doses, steroids can kill cancer cells. They can also stop white blood cells from going to the disease-affected areas, thereby decreasing inflammation and relieving pain and pressure.

Some side effects of steroids include:

Long-term steroid use may suppress the immune system and weaken the bones. However, these side effects often go away once the steroid treatment is over.

People with myeloma can develop weak bones that may break easily. Bone modifying drugs called bisphosphonates can help strengthen the bones and slow down the weakening process. They can also reduce pain levels.

One rare but severe side effect of bone modifying drugs is osteonecrosis of the jaw. The drugs can destroy the jawbone and create an open wound that cannot heal. The open sore can lead to infection, and some people also experience tooth loss in the area.

One way to prevent this is by maintaining good oral hygiene and treating tooth or gum infections right away.

The standard of care for those with multiple myeloma involves a combination of high-dose chemotherapy and a bone marrow transplant. Getting a bone marrow transplant often means that an individual remains disease-free for a long time.

Whether or not someone is a good candidate for a bone marrow transplant depends on many factors, such as their age and the stage of their myeloma.

People frequently receive chemotherapy before a bone marrow transplant, which weakens their immune system and can lead to infections.

Some other side effects of bone marrow transplants can include:

Radiation therapy uses rays of particles to damage multiple myeloma cells and prevent them from growing. Not only can radiation therapy treat multiple myeloma, but it can also reduce complications from bone disease.

Like chemotherapy, radiation therapy can damage some normal cells, especially those that divide quickly. However, most healthy cells recover fully from the effects of radiation therapy.

Some side effects of radiation therapy include fatigue and a loss of appetite. A person may also have some sensitivity and irritation in the skin around the treatment area.

Doctors cannot cure multiple myeloma. There are times when it may lie dormant, but the cancer usually returns. For some people, multiple myeloma may present with no symptoms and progress slowly. A good response to initial treatment can lead to a promising outlook.

Even when treatment is over, regular follow-up testing is necessary to monitor for relapse. The 5-year survival rate for multiple myeloma in 20102016 was 53.9%. This rate has been steadily increasing.

Multiple myeloma is a cancer that affects the plasma cells in the bone marrow. There are many treatment options for multiple myeloma.

The standard of care is a combination of chemotherapy and radiation therapy. In most cases, treatment includes a combination of multiple therapies and drugs.

Multiple myeloma has no cure, but people can go into remission. People in remission must have regular follow-up testing to monitor for disease recurrence.

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Human Embryonic Stem Cells Market Share, Growth by Business Developments 2021 to 2025 CellTherapies P/L, BioRestorative Therapies Inc., BrainStorm…

Posted: July 6, 2021 at 1:56 am

The Human Embryonic Stem Cells Market research report provides detailed observation of several aspects, including the rate of growth, regional scope and recent developments by the primary market players. The report offers Porters Five Forces, PESTEL, and market analysis to provide a 360-degree research study on the global Human Embryonic Stem Cells market. The research study discusses about important market strategies, future plans, market share growth, and product portfolios of leading companies. The final report copy provides the impact analysis of novel COVID-19 pandemicon the Human Embryonic Stem Cells market as well as fluctuations during the forecast period.

Top Companies in the global Human Embryonic Stem Cells market areCellTherapies P/L, BioRestorative Therapies Inc., BrainStorm Cell Therapeutics Inc., NuVasive Inc., Beike Biotechnology, Cynata Therapeutics Ltd., Lonza Group Ltd., Kite Pharma Inc., Vericel Corporation, Sumanas, Aastrom Biosciences, Ocata Therapeutics Inc., Genlantis, Cesca Therapeutics Inc., Life Technologies Corporation, TiGenix N.V, Anterogen Co., Ltd, Orthofix International N.V., PromoCell and Other.

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By types market is divided into

Adult Sources

Fetal Sources

Others

By applications market is divided into

Hematopoietic stem cell transplantation

Tissue repair damage

Autoimmune diseases

As gene therapy vectors.

Regional Analysis:Asia-Pacific (China, India, Japan, South Korea, Australia, Indonesia, Malaysia, and Others), North America (United States, Canada, and Mexico), Central & South America (Brazil, and Rest of South America), Europe (Germany, France, UK, Italy, Russia, and Rest of Europe), Middle East & Africa (GCC Countries, Turkey, Egypt, South Africa and Other)

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The Human Embryonic Stem Cells market report highlights are A comprehensive evaluation of all opportunities and risks in the market. Human Embryonic Stem Cells market current developments and significant occasions. A deep study of business techniques for the development of the market-driving players. Conclusive study about the improvement plot of the market for approaching years. Top to bottom approach of market-express drivers, targets, and major littler scale markets.

Important Features that are under Offering and Key Highlights of the Reports: Potential and niche segments/regions exhibiting promising growth. Detailed overview of Market Changing market dynamics of the industry In-depth market segmentation by Type, Application, etc. Historical, current, and projected market size in terms of volume and value Recent industry trends and developments Competitive landscape of Market Strategies of key players and product offerings

Free customization of the report: This report can be further customized according to the clients specific requirements. No additional charges will be added for limited additional research.

Contact UsIrfan Tamboli (Sales Manager) Market Insights ReportsPhone: + 1704 266 3234 | +91-750-707-8687sales@marketinsightsreports.com | irfan@marketinsightsreports.com

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Global Synthetic Stem Cells Market Advancements and Outlook 2020 to 2027 North Carolina State University and Zhengzhou University and others KSU |…

Posted: July 6, 2021 at 1:56 am

The report released by Zion Market Research on the global Synthetic Stem Cells Market is a comprehensive and in-depth study on the market landscape and prevailing dynamics during the forecast period. The report is a complete evaluation of market dynamics regarding the competitive scenario and major ongoing trends in the global Synthetic Stem Cells Market. The report is aimed to offer useful insights on value chain analysis along with a vivid picture of other trends prevailing in the market. The report provides key insights on the profile leaders along with the new market entrants in the global Synthetic Stem Cells Market.

Global Synthetic Stem Cells Market Advancements and Outlook 2020 to 2027 North Carolina State University and Zhengzhou University and others

The prominent players in the market are North Carolina State University and Zhengzhou University

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The report dedicates a whole unit to the trends and opportunities during Covid-19 and post-Covid-19 in the global Synthetic Stem Cells Market. Along with this, it also mentions the opportunities and the restraints followed by Covid-19 in the market. Moreover, the report is structured into different segments namely overview, growth factors, segmentation analysis, and competitive analysis. Each section is further detailed to facilitate better understanding of the global market.

The overview section helps users to understand the complete landscape of the global Synthetic Stem Cells Market. It mentions the key patterns and strategies in the development of the global market. The section consists of market size and volume along with the prevailing business policies in the global market. The growth and restraining factors segment elaborate on the various factors that will promulgate or hinder the market growth in the forthcoming years. It also consists of an organized evaluation of the market opportunities that will help the users to make their growth strategy and expand their reach in the global market.

Analysts have adopted various methodologies and approaches like SWOT analysis to state the strengths and weaknesses of the global Synthetic Stem Cells Market during the forecast period. The report also compasses the potency of buyers and suppliers to help stakeholders develop a profit-oriented business plan and take effective decisions to expand their portfolio. The data interpretation regarding the global Synthetic Stem Cells Market in the report is the emulsification of primary and secondary research. Therefore, the quantitative analysis of the global market concluded by our analysts is completely reliable and trustworthy.

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The regional analysis section reveals the potential areas all around the globe in the global Synthetic Stem Cells Market. Each potential region is mapped with the market size and volume to help users discover the untapped potential in the global Synthetic Stem Cells Market. Moreover, the competitive analysis segment mentions the key players along with their strategies to understand their working in the global market.

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2 Reasons to Buy Intellia — and 1 Big Reason Why I Won’t – The Motley Fool

Posted: July 6, 2021 at 1:56 am

Clinical trial data supporting the safety of the CRISPR-Cas9 genomic editing tool was presented on Monday by Intellia Therapeutics (NASDAQ:NTLA) for its lead product, NTLA-2001. The data was highly encouraging. However, despite NTLA-2001's positive early results as a potential treatment for the rare disease transthyretin (TTR) amyloidosis, there's still a long way to go before Intellia could bring it to market.

In transthyretin amyloidosis, cells in the liver produce misfolded TTR proteins, which accumulate throughout the body, causing debilitating complications that can involve the digestive system, nervous system, and heart. Once symptoms appear, they grow progressively worse, and the disease leads to death within a median of 4 to 17 years among patients with nervous system involvement, and 2 to 6 years among patients with cardiac involvement.

NTLA-2001 edits the genes in those liver cells, removing the segment that produces those lethal misfolded proteins.

Worldwide, an estimated 250,000-550,000 people suffer from some form of amyloidosis.

IMAGE SOURCE: GETTY IMAGES.

An interim readout from Intellia's ongoing phase 1 trial found that a single high-dose infusion of NTLA-2001 led to an 87% mean reduction in the amount of misfolded TTR in patients' bloodstreams, with a maximum reduction of 96% by day 28 in one patient. Encouragingly, no serious adverse events were observed in the six study participants. While this is a small pilot study, in previous studies of NTLA-2001 in mice, the maximum reductions in TTR persisted for 12 months after a single treatment.

All of this data provides an early indication that CRISPR gene therapies are safe and efficacious as treatments for at least some genetic diseases.

There are other treatments on the market for TTR amyloidosis, but one thing that would set CRISPR apart is the relative simplicity of administering it. And that factor could lead insurers to favor CRISPR treatments for certain rare and debilitating diseases such as TTR amyloidosis and hemophilia.

For example, Alnylam's (NASDAQ:ALNY) RNA-silencing therapy Onpattro requires an infusion every three weeks at a clinician's office. Ionis Pharmaceuticals' (NASDAQ:IONS) Tegsedi requires regular injections, though they can be self-administered. Both are priced in the neighborhood of $345,000 per year, and Onpattro comes with the additional costs associated with going to a medical office and having an infusion set up. Then there is Pfizer's (NYSE:PFE) once-daily oral medication Vyndamax, which costs $225,000 annually.

As a one-time infusion, gene therapy may become a compelling option for both patients and insurers, particularly given the high prices of currently available treatments. Though TTR amyloidosis treatments are a niche market, in 2020, Onpattro generated sales of $306 million, Tegsedi just under $70 million, and Vyndamax $429 million. Assuming that Intellia charges more for NTLA-2001 -- a one-time treatment with bluebird bio's (NASDAQ:BLUE) gene therapy for beta-thalassemia, Zynteglo, costs about $1.8 million -- TTR amyloidosis treatment could easily become a multibillion-dollar addressable market for the biotech.

Notably, CRISPR therapy for TTR amyloidosis may also put less stress on the healthcare system than the lentivirus and adenovirus gene therapies that are further along in clinical trials. Consider, for instance, Zynteglo, which requires a significant amount of effort and processing prior to treatment. First, physicians must extract stem cells from the patient, which must then be transported to and treated by bluebird bio. In the meantime, the patient undergoes "myeloablative conditioning" -- essentially knocking down the patient's bone marrow in preparation for a transplant of the edited stem cells, which will contain a repaired version of the gene that (when mutated) causes beta-thalassemia. This complicated process requires treatment at a qualified transplant center.

By comparison, for TTR amyloidosis, NTLA-2001 requires pre-medication with steroids and antihistamines. That's it. No prolonged patient preparation at the hospital. No bone marrow suppression. No shipping the patient's stem cells to a lab. The relative simplicity of administering CRISPR therapies is just one reason for the degree of excitement they are generating.

It may also give them a lower total cost of treatment than current gene therapies, which could make these therapies more palatable to insurers. If NTLA-2001 pans out, we may see a new biotech boom, with Intellia leading the charge.

Before investors get their hopes up too much, remember that these results were from a six-person, phase 1 trial, and that Intellia now holds a market cap of roughly $11 billion. In fact, its valuation rose by about $2.8 billion in a single trading session after the interim trial data was made public. That gain was more than the current $2.1 billion market cap of bluebird bio, which already has an approved gene therapy on the market as well as a CAR-T therapy, and has two more candidates in phase 3 trials.

For further context, bluebird bio announced phase 1 results for Zynteglo in December 2014. While Zynteglo was approved for use in the EU in late 2019, bluebird bio faced some backlash on pricing, and the company isn't selling it in Germany because the two sides could not agree on pricing.

Moreover, the NTLA-2001 study excluded patients who had previously received RNA-silencing therapy, and none of these patients had previously taken Vyndamax either. How previous treatments will affect the way patients respond to NTLA-2001 is not yet known. And with hundreds of millions of dollars in revenue annually on the line, it is doubtful that Alynam, Ionis, or Pfizer will surrender this market without a fight.

In sum, Intellia will still need to conduct several years of trials, leap many regulatory hurdles, and outmaneuver an array of rivals stand before it can declare the CRISPR-Cas9 platform a winner. Not only that, but -- recognizing that future studies won't be cheap -- Intellia has already proposed another public offering of $400 million worth of common stock this week, diluting its current shareholders.

So while long-term Intellia shareholders have reason to celebrate, let bluebird bio serve as a cautionary tale. That biotech was once flying high on positive trial data, hitting a market cap of around $15.5 billion in March 2018. Since then, its shares have nose-dived by more than 80%. This despite the fact that it now has two approved therapies and two more candidates in phase 3 trials.

As such, I would be concerned about investing new money in Intellia now. I suspect it will soon reach its peak for the foreseeable future. Biotech investing can be gut-wrenchingly fickle, and investors may want to consider taking a basket approach to high-risk clinical-stage biotechs, rather than investing too heavily in a single player.

This article represents the opinion of the writer, who may disagree with the official recommendation position of a Motley Fool premium advisory service. Were motley! Questioning an investing thesis -- even one of our own -- helps us all think critically about investing and make decisions that help us become smarter, happier, and richer.

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2 Reasons to Buy Intellia -- and 1 Big Reason Why I Won't - The Motley Fool

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‘Time is of the essence’: Alberta family seeking treatment for 3 kids with rare, degenerative illness – CBC.ca

Posted: July 6, 2021 at 1:56 am

Katie and Jacob Lamprecht are doing what they can to buy more time for their three children, all of whom have a rare degenerative illness that has no cure.

Last fall, 10-year-old Kiara Lamprecht and her eight-year-old sister Hannah were diagnosed with juvenile neuronal ceroid lipofuscinoses also known as CLN3 or juvenile Batten disease.

Then five-year-old AJ, theirhalf-brother,was also diagnosed with the illness last month.

As the parents scour research seeking potential treatments, fundraisers are underwayto allow the family to afford them.

"Time is of the essence. We don't have years and years and years to figure this out," said Katie Lamprecht from the family's home in Evansburg, Alta.,about 90 kilometres west of Edmonton.

Batten disease, an inherited and fatal genetic illness, has various forms. In this case, it's caused by a mutation that results in cells not producing enough of the CLN3 protein that helps clear waste products. Over time, toxins build up and brain cells start to die.

Symptoms usually start showing in children aged four to seven. The first is often vision loss, followed by changes in cognition and behaviour, seizures, then declining motor skills.

Those with the disease will die in their teens or 20s.

CLN3 is a rare condition in Canada, according toStphane Lefranois, researcher professor at the Institut National de la Recherche Scientifique in Quebec.

Juvenile Batten disease develops when a child inherits two copies of the genetic mutation from their parents, each of whom would be carrying one defective gene. Because two defective copies are needed for Batten disease to develop, the parents would not be affected, said Lefranois.

The mutation's prevalence varies throughout the world. In some regions,it shows up in one of 12,000 live births; in others, it's found in one of 100,000 births, he said.

It's"quite rare" that sisters Kiara and Hannah were diagnosed with the disease,said Lefranois, but theodds of AJ's diagnosisgiven he has a different motherare "astronomically low."

Jacob Lamprecht moved to another part of the world andmarried Katie, an unknowing carrier,and AJ inherited both mutations. The Lamprechtshave been told the chance of their situation occurring is about one in eight billion.

According to the parents, both girls are legally blind. AJ's eyesight is worsening but he hasn't had a vision test yet.Hannah has developed behavioural issues, with episodes of kicking, screaming and biting.Abrain scan conducted last year shows she has sufferedsome brain damage and is experiencing seizures, said her father.

So far, over $113,500 has been raised for potential treatments. But the parents are unsure about their next move.

"There's nothing concrete that we can follow," said Katie Lamprecht.

There is currently no cure for CLN3, so most treatment just manages the symptomslike medications for seizures or Braille instruction for children losing their vision, said Dr. Jonathan Mink, CLN3 researcher and a professor of pediatric neurology at New York's University of Rochester.

But clinical trials in the United States some completed, some ongoing are makingheadway, he said.

Among them are clinical trials for gene therapy, a technique that modifies a person's faulty genes to help cells function normally.

"The hope is, particularly with gene therapy, that we could give something to a very young child and that would correct the problem and they never have any symptoms," said Mink.

The Lamprechts are looking at gene therapy, which is deemed an experimental treatment option by Health Canada, as well as a stem cell operation. The latter is a last resort, they said, requiringtravelto the U.S. and a cost ofabout $1.8 million per child.

Some people have undergone such operations, but Mink would advise against it because there aren't many published results and, through his clinical practice, he hasn't seen any significant improvement in patients.

Undergoing a stem cell operation might also disqualify a child from future clinical trials or treatments, he added.

After the initial diagnosis, families often go through the stages of grief before focusing on giving their child the best possible quality of life for their remaining years, said Mink.

Families who have more than one child with the disease feel a greater impact, but Mink and his team are "so impressed by how resilient people are."

Jacob and Katie Lamprecht are among the resilient, savouring moments with their children while focusing on how to save them.

COVID-19, for example, became an opportunity for Katie Lamprechtto embracefull-time homeschooling, lettingher be with them every day.

Kiara has been approved for Make-A-Wish Canada, so the family is debating whether to visit SeaWorld or Disney World.

"We're not just sitting around crying all the time," said Katie.

"Most of the time I feel like we're going to beat this."

Excerpt from:
'Time is of the essence': Alberta family seeking treatment for 3 kids with rare, degenerative illness - CBC.ca

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Hear top VCs Albert Wegner, Jenny Rooke, and Shilpi Kumar talk green bets at the Extreme Tech Challenge finals – TechCrunch

Posted: July 6, 2021 at 1:53 am

This year, TechCrunch is proudly hosting the Extreme Tech Challenge Global Finals on July 22. The event is among the worlds largest purpose-driven startup competitions that are aiming to solve global challenges based on the United Nations 17 sustainability goals.

If you want to catch an array of innovative startups across a range of categories, all of them showcasing what theyre building, you wont want to miss our must-see pitch-off competition.

You can also catch feature panels hosted by TechCrunch editors, including one of the most highly anticipated discussions of the event, a talk on going green with guest speakers Shilpi Kumar, Jenny Rooke, and Albert Wenger, all of whom are actively investing in climate startups that are targeting big opportunities

Shilpi Kumar is a partner with Urban Us, an investment platform focused on urban tech and climate solutions. She previously led go-to-market and early sales efforts at Filament, a startup focused on deploying secure wireless networks for connected physical assets. As an investor, Shilpi has also focused on hardware, mobility, energy, IoT, and robotics, having worked previously for VTF Capital, First Round Capital, and Village Global.

Jenny Rooke is the founder and managing director of Genoa Ventures, but Rooke has been deploying capital into innovative life sciences opportunities for years, including at Fidelity Biosciences and later the Gates Foundation, where she helped managed more than $250 million in funding, funneling some of that capital into genetic engineering, diagnostics, and synthetic biology startups. Rooke began independently investing under the brand 5 Prime Ventures, ultimately establishing among the largest life sciences syndicates on AngelList before launching Genoa.

Last but not least, Albert Wenger, has been a managing partner at Union Square Ventures for more than 13 years. Before joining USV, Albert was the president of del.icio.us through the companys sale to Yahoo and an angel investor, including writing early checks to Etsy and Tumblr. He previously founded or co-founded several companies, including a management consulting firm and an early hosted data analytics company. Among his investments today is goTenna, a company trying to advance universal access to connectivity by building a scalable mobile mesh network.

Sustainability is the key to our planets future and our survival, but its also going to be incredibly lucrative and a major piece of our world economy. Hear from these seasoned investors about how VCs and startups alike are thinking about Greentech and how that will evolve in the coming years.

Join us on July 22 to find out how the most innovative startups are working to solve some of the worlds biggest problems. And best of all, tickets are free book yours today!

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Hear top VCs Albert Wegner, Jenny Rooke, and Shilpi Kumar talk green bets at the Extreme Tech Challenge finals - TechCrunch

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Science, industry team up in Italy to zap COVID with laser – New York Post

Posted: July 6, 2021 at 1:53 am

ROME, July 2 A United Nations-backed scientific research centre hasteamedupwith an Italian tech firm to explore whetherlaserlight can be used to kill coronavirusparticles suspended in the air and help keep indoor spaces safe.

The joint effort between the International Centre for Genetic Engineering and Biotechnology (ICGEB) of Trieste, a city in the north ofItaly, and the nearby Eltech K-Lasercompany, was launched last year as COVID-19 was battering the country.

They created a device that forces air through a sterilization chamber which contains alaserbeam filter that pulverizesviruses and bacteria.

I thoughtlasers were more for a shaman rather than a doctor but I have had to change my mind. The device proved able to kill theviruses in less than 50 milliseconds, said Serena Zacchigna, groupleader for Cardiovascular Biology at the ICGEB.

Healthy indoor environments with a substantially reduced pathogen count are deemed essential for public health in the post COVID-19 crisis, a respiratory infection which has caused more than four million deaths worldwide in barely 18 months.

Zacchigna hookedupwith Italian engineer Francesco Zanata, the founder of Eltech K-Laser, a firm specialised in medicallasers whose products are used by sports stars to treat muscle inflammation and fractures.

Some experts have warned against the possible pitfalls of using light-based technologies to attack thevirusthat causes COVID-19.

A study published by the Journal of Photochemistry & Photobiology in November 2020 highlighted concerns ranging from potential cancer risks to the cost of expensive light sources.

But Zacchigna and Zanata dismissed any health issues, saying thelasernever comes into contact with human skin.

Our device uses nature against nature. It is 100% safe for people and almost fully recyclable, Zanata told Reuters.

The technology, however, does not eliminateviruses and bacteria when they drop from the air onto surfaces or the floor. Nor can it prevent direct contagion when someone who is infected sneezes or talks loudly in the proximity of someone else.

Eltech K-Laserhas received a patent from Italian authorities and is seeking to extend this globally.

The portable version of the invention is some 1.8 metres (5.9 ft) high and weighs about 55 lb. The company said the technology can also be placed within air-conditioning units.

In the meantime, the first potential customers are liningup, including Germanys EcoCare, a service provider of testing and vaccination solutions.

The company aims to license the technology for German and UAE markets, an EcoCare spokesperson said in an email to Reuters.

Excerpt from:
Science, industry team up in Italy to zap COVID with laser - New York Post

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