"The laboratory house mouse has maintained a standard 40-chromosome karyotypeor the full picture of an organism's chromosomesafter more than 100 years of artificial breeding," said Li Zhikun, a researcher at CAS's Institute of Zoology.

"Over longer time scales, however, karyotype changes caused by chromosome rearrangements are common. Rodents have 3.2 to 3.5 rearrangements per million years, whereas primates have 1.6," added Li, co-first author of the study.

The mouse, known as Xiao Zhu, or "Little Bamboo," was the world's first mammal with fully reprogrammed genes, according to the South China Morning Post.

The study claims to have provided important insight into how chromosomal rearrangements may affect evolution by showing that chromosome-level engineering is possible in mammals and by effectively deriving a laboratory house mouse with a novel and sustainable karyotype.

According to Li, such small changes can have a big impact. Humans and gorillas are separated by 1.6 changes in primates. Gorillas have two distinct chromosomes, whereas humans have two fused chromosomes, and a translocation between ancestor human chromosomes resulted in two distinct chromosomes in gorillas.

Individually, fusions or translocations can result in missing or extra chromosomes, as well as diseases like childhood leukemia.

While the chromosomes' consistent reliability is useful for understanding how things work on a short time scale, Li believes that the ability to engineer changes could inform genetic understanding over millennia, including how to correct misaligned or malformed chromosomes.

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Chinese scientists claim to have engineered the world's first mouse with fully reprogrammed genes - Interesting Engineering

DetailsCategory: DNA RNA and CellsPublished on Saturday, 27 August 2022 11:16Hits: 666

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, PA, USA I August 25, 2022 I 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.

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/.

SOURCE: Century Therapeutics

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

Part 1 of the new Climate Tech Weekly series, "What Would Dolly Parton Do? 9 to 5 in the Climate Tech Sector"

Before we dive in, I need to say thank you so much to everyone who responded to my call for climate tech startups with influential women steering the ship. I received so many messages I literally had to create a new folder in my inbox to keep everything organized a.k.a. Please be patient with me about my response time!

Welcome to the first article in our brand new series, "What Would Dolly Parton Do? 9 To 5 In the Climate Tech Sector." First up is Living Carbon, a synthetic biotechnology company based in Hayward, California.

One sunny Friday, I walked into an unassuming building and was met by a woman with a glowing smile Living Carbon CEO and co-founder Maddie Hall. She instantly set the tone of the tour as informative yet conversational, cracking jokes and sipping seltzer throughout our afternoon together.

So what does a synthetic biotech company do? Hall breaks it down for me: It all comes down to trees. As we walked through the lab and growing center, Hall explained that Living Carbon starts with the seedlings of hybrid poplar trees and genetically alters the seeds.

"We start with these undifferentiated plant cells, or the plant version of stem cells, and we go through this process called particle bombardment where we use a combination of helium, gold particles and a vacuum. We also do gene editing with CRISPR [a technology often used to edit genes]."

Living Carbon grows the seedlings from a few cells on a petri dish to fledging trees, and are in the process of building a specific greenhouse for the plants once they grow to a certain height; for now, the trees are housed in a special room with specifically designed LED lights.

Living Carbon then sells the trees to landowners seeking to transform their own properties to more productive systems. Additionally, Living Carbon sells credits to carbon credit buyers Ill expand on that aspect of the company later on.

Once transferred from the lab to the earth, the real fun begins. As the trees grow and live, they accumulate highly concentrated amounts of nickel from the soil and capture higher than normal amounts of carbon from the atmosphere once planted in the ground.

Nickel is a natural component of soil, but when too much has built up due to industrial processes, it can actually inhibit natural growth.

What makes these trees stand apart from the multitudes of tree planting campaigns around the world is the genetic engineering that increases their effectiveness for eliminating hazardous materials from the natural world and higher growth rate.

Lounging in the conference room, appropriately decorated with wall art depicting branches in a forest, Hall and I discuss Living Carbons inception and creation.

Before Living Carbon, Hall worked at venture capital firm Y Combinator on special projects focusing on the future of artificial intelligence (AI). As she learned about the technology of the future via her clients, her own passion to make an impact began to bloom.

"Were already working on something that is decades in the future and what the world will sort of look like. You start to think a lot more about existential threats to humanity, and climate is one of them. So I did a deep dive looking for something similar to AI but for climate change."

And her path became clear: plant biotechnology. "You have all of these really brilliant tenured researchers who have spent most of their lives working on crops or sometimes even chemicals, but you go into plant biotech because you really like plants." So Hall, alongside her co-founder, Patrick Mellor, decided to provide a third option for these singular minds: A private company that supports both lab and field research and implementation for multiple species of trees.

Hall counts herself among those who really like plants. Since childhood, the CEO explained her whole world has been influenced by greenery via her family, saying, "My grandmother was on the board of the arboretum in Seattle for 50 years as a volunteering memberand my other grandma was a librarian who studied plants and birds and my mom did flowers."

When I asked Hall to describe her experience as a female founder in Silicon Valley a space famously oversaturated with male CEOs she dropped the jovial tone.

"Neither [my grandmother or mother] had the chance to start a company. So I feel like [Living Carbon] is the company that I would have wanted all of the women in my family who came before me to also have the opportunity to start."

And it's not just a family legacy Hall considers when evaluating her role as CEO. She describes the implications of starting a business in her late 20s, acknowledging that if she wanted to become a mother, the time for Living Carbon was now or never. And this dilemma, she elaborates, of family versus work is a decision frequently weighed by women that men in her position dont tend to consider.

Furthermore, Hall describes her hesitancy to engage in PR for Living Carbon until multiple clients were booked, saying, "I wanted us to have scientific credibility because I saw so many hit pieces on female founders coming out and Im like, I know at least 20 male founders who had done the same things but didnt get an entire article."

I recently wrote about Frontier, a new philanthropic advance market commitment (AMC) that finances early-stage climate tech companies with the potential to invigorate a carbon capture and credit market. In June, Living Carbon was announced as one of six companies chosen to receive money from the AMC, allowing the company to sell carbon credits created with the carbon sequestered from the atmosphere via the trees planted.

With this new funding, Living Carbon will continue to scale up. When I asked if the public would be able to pick up one of these super trees from their local Lowes sometime in the near future, the answer was a firm no. Hall made it clear that Living Carbon wants to make a massive impact, saying, "If were at Home Depot or Lowes, it's one person planting a tree, but what we want is to be working on a massive scale." That massive scale is more to the tune of planting thousands of trees where an old mine used to be, rehabilitating the soil and habitat. And isnt that, after all, the reason why trees exist in the first place?

So there you have it, folks. The first official entry in the series "What Would Dolly Parton Do? 9 To 5 in the Climate Tech Sector." And dont worry. Ill let Dolly play me out(skip to one minute in to get to the actual song).

"You better get to livin"

Some quotes in this article were altered for clarity or length.

[Want more great insight on technologies and trends accelerating the clean economy? Subscribe to our free Climate Tech Weekly newsletter. ]

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Living Carbon: The startup setting down roots from 9 to 5 | Greenbiz - GreenBiz

GAITHERSBURG, Md., Aug. 26, 2022 /PRNewswire/ -- Novavax, Inc. (Nasdaq: NVAX), a biotechnology company dedicated to developing and commercializing next-generation vaccines for serious infectious diseases, today announced that the Medicines and Healthcare products Regulatory Agency (MHRA) in the United Kingdom (UK) has granted expanded conditional marketing authorization (CMA) for Nuvaxovid (NVX-CoV2373) COVID-19 vaccine for active immunization to prevent coronavirus disease 2019 (COVID-19) caused by the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) in adolescents aged 12 through 17.

"As we start to prepare for a potential fall surge, we are pleased to offer the first protein-based COVID-19 vaccine to adolescents aged 12 through 17 in the U.K.," said Stanley C. Erck, President and Chief Executive Officer, Novavax. "We believe that our vaccine, developed using an innovative approach to traditional technology, may help increase adolescent vaccination rates."

The expanded CMA was based on data from the ongoing pediatric expansionof the Phase 3 PREVENT-19 trial of 2,247 adolescents aged 12 through 17 years across 73 sites in the U.S., to evaluate the safety, effectiveness (immunogenicity), and efficacy of Nuvaxovid. In the pediatric expansion, Nuvaxovid achieved its primary effectiveness endpoint and demonstrated 80% clinical efficacy overall at a time when the Delta variant was the predominant circulating SARS-CoV-2 strain in the U.S.

Preliminary safety data from the pediatric expansion showed the vaccine to be generally well-tolerated. Serious and severe adverse events were low in number and balanced between vaccine and placebo groups, and not considered related to the vaccine. Local and systemic reactogenicity was generally lower than or similar to adults, after the first and second dose. The most common adverse reactions observed were injection site tenderness/pain, headache, myalgia, fatigue, and malaise. There was no increase in reactogenicity in younger (12 to <15 years old) adolescents compared to older (15 to <18 years old) adolescents. No new safety signal was observed through the placebo-controlled portion of the pediatric expansion.

The next step for the vaccine is a policy recommendation for use from the UK Joint Committee on Vaccination and Immunisation (JCVI). Doses of Nuvaxovid will be made available for use in adolescents based on the JCVI's recommendation.

In the 12 through 17 year-old population, the Novavax COVID-19 vaccine has been granted authorization in the U.S., India, the European Union, Australia, Japan, Thailand, and New Zealand, and is actively under review in other markets.

The MHRA previously granted CMA for Nuvaxovid in adults aged 18 and older in February 2022. The vaccine is given as a primary vaccination in two doses administered 21 days apart. Novavax filed for expanded CMA for use as a booster in adults aged 18 and over in June 2022.

This medicine is subject to additional monitoring. This will allow quick identification of new safety information. If you are concerned about an adverse event, it should be reported on a Yellow Card. Reporting forms and information can be found at https://coronavirus-yellowcard.mhra.gov.uk/or search for MHRA Yellow Card in the Google Play or Apple App Store. When reporting please include the vaccine brand and batch/Lot number if available.

Trade Name in the U.S.The trade name Nuvaxovid has not yet been approved by the U.S. Food and Drug Administration.

Important Safety Information: UK

For more information on Nuvaxovid, including the Summary of Product Characteristics with Package Leaflet, adverse event reporting instructions, or to request additional information, please visit the following websites:

About Nuvaxovid (NVX-CoV2373)Nuvaxovid (NVX-CoV2373) is a protein-based vaccine engineered from the genetic sequence of the first strain of SARS-CoV-2, the virus that causes COVID-19 disease. The vaccine was created using Novavax' recombinant nanoparticle technology to generate antigen derived from the coronavirus spike (S) protein and is formulated with Novavax' patented saponin-based Matrix-M adjuvant to enhance the immune response and stimulate high levels of neutralizing antibodies. Nuvaxovid contains purified protein antigen and can neither replicate, nor can it cause COVID-19.

Nuvaxovid is packaged as a ready-to-use liquid formulation in a vial containing ten doses. The vaccination regimen calls for two 0.5 ml doses (5 mcg antigen and 50 mcg Matrix-M adjuvant) given intramuscularly 21 days apart. The vaccine is stored at 2- 8 Celsius, enabling the use of existing vaccine supply and cold chain channels. Use of the vaccine should be in accordance with official recommendations.

Novavax has established partnerships for the manufacture, commercialization, and distribution of Nuvaxovid worldwide. Existing authorizations leverage Novavax' manufacturing partnership with Serum Institute of India, the world's largest vaccine manufacturer by volume. They will later be supplemented with data from additional manufacturing sites throughout Novavax' global supply chain.

About the Novavax COVID-19 vaccine (NVX-CoV2373) Phase 3 TrialsThe Novavax COVID-19 vaccine (NVX-CoV2373) continues being evaluated in two pivotal Phase 3 trials.

PREVENT-19 (thePRE-fusion protein subunitVaccineEfficacyNovavaxTrial | COVID-19) is a 2:1 randomized, placebo-controlled, observer-blinded trial to evaluate the efficacy, safety and immunogenicity of the Novavax COVID-19 vaccine with Matrix-M adjuvant in 29,960 participants 18 years of age and over in 119 locations inthe U.S.andMexico. The primary endpoint for PREVENT-19 was the first occurrence of PCR-confirmed symptomatic (mild, moderate or severe) COVID-19 with onset at least seven days after the second dose in serologically negative (to SARS-CoV-2) adult participants at baseline. The statistical success criterion included a lower bound of 95% CI >30%. A secondary endpoint was the prevention of PCR-confirmed, symptomatic moderate or severe COVID-19. Both endpoints were assessed at least seven days after the second study vaccination in volunteers who had not been previously infected with SARS-CoV-2. In the trial, the Novavax COVID-19 vaccine achieved 90.4% efficacy overall. It was generally well-tolerated and elicited a robust antibody response after the second dose in both studies. Full results of the trial were published in theNew England Journal of Medicine(NEJM).

The pediatric expansion of PREVENT-19 is a 2:1 randomized, placebo-controlled, observer-blinded trial to evaluate the safety, effectiveness, and efficacy of the Novavax COVID-19 vaccine with Matrix-M adjuvant in 2,247 adolescent participants 12 to 17 years of age in 73 locations in the United States, compared with placebo. In the pediatric trial, the vaccine achieved its primary effectiveness endpoint (non-inferiority of the neutralizing antibody response compared to young adult participants 18 through 25 years of age from PREVENT-19) and demonstrated 80% efficacy overall at a time when the Delta variant of concern was the predominant circulating strain in the U.S.Additionally, immune responses were about two-to-three-fold higher in adolescents than in adults against all variants studied.

Additionally, a trial conducted in the U.K. with 14,039 participants aged 18 years and over was designed as a randomized, placebo-controlled, observer-blinded study and achieved overall efficacy of 89.7%. The primary endpoint was based on the first occurrence of PCR-confirmed symptomatic (mild, moderate or severe) COVID-19 with onset at least seven days after the second study vaccination in serologically negative (to SARS-CoV-2) adult participants at baseline. Full results of the trial were published inNEJM.

About Matrix-M AdjuvantNovavax' patented saponin-based Matrix-M adjuvant has demonstrated a potent and well-tolerated effect by stimulating the entry of antigen-presenting cells into the injection site and enhancing antigen presentation in local lymph nodes, boosting immune response.

About NovavaxNovavax, Inc. (Nasdaq: NVAX) is a biotechnology company that promotes improved health globally through the discovery, development, and commercialization of innovative vaccines to prevent serious infectious diseases. The company's proprietary recombinant technology platform harnesses the power and speed of genetic engineering to efficiently produce highly immunogenic nanoparticles designed to address urgent global health needs. The Novavax COVID-19 vaccine, has received authorization from multiple regulatory authorities globally, including the U.S., EC and the WHO. The vaccine is currently under review by multiple regulatory agencies worldwide, including for additional indications and populations such as adolescents and as a booster. In addition to its COVID-19 vaccine, Novavax is also currently evaluating a COVID-seasonal influenza combination vaccine candidate in a Phase 1/2 clinical trial, which combines NVX-CoV2373 and NanoFlu*, its quadrivalent influenza investigational vaccine candidate, and is also evaluating an Omicron strain-based vaccine (NVX-CoV2515) as well as a bivalent format Omicron-based / original strain-based vaccine. These vaccine candidates incorporate Novavax' proprietary saponin-based Matrix-M adjuvant to enhance the immune response and stimulate high levels of neutralizing antibodies.

For more information, visitwww.novavax.comand connect with us on LinkedIn.

*NanoFlu identifies a recombinant hemagglutinin (HA) protein nanoparticle influenza vaccine candidate produced by Novavax. This investigational candidate was evaluated during a controlled phase 3 trial conducted during the 2019-2020 influenza season.

Forward-Looking StatementsStatements herein relating to the future of Novavax, its operating plans and prospects, its partnerships, the potential for subsequent orders from the U.S. government for additional doses of NVX-CoV2373 and other potential formulations, the timing of clinical trial results, the ongoing development of NVX-CoV2373, including an Omicron strain based vaccine and bivalent Omicron-based / original strain based vaccine, a COVID-seasonal influenza investigational vaccine candidate, the scope, timing and outcome of future regulatory filings and actions, including Novavax' plans to supplement existing authorizations with data from the additional manufacturing sites in Novavax' global supply chain, additional worldwide authorizations of NVX-CoV2373 for use in adults and adolescents, and as a booster, the potential impact and reach of Novavax and NVX-CoV2373 in addressing vaccine access, controlling the pandemic and protecting populations, the efficacy, safety and intended utilization of NVX-CoV2373, and the expected administration of NVX-CoV2373 are forward-looking statements. Novavax cautions that these forward-looking statements are subject to numerous risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. These risks and uncertainties include, without limitation, challenges satisfying, alone or together with partners, various safety, efficacy, and product characterization requirements, including those related to process qualification and assay validation, necessary to satisfy applicable regulatory authorities; difficulty obtaining scarce raw materials and supplies; resource constraints, including human capital and manufacturing capacity, on the ability of Novavax to pursue planned regulatory pathways; unanticipated challenges or delays in conducting clinical trials; challenges meeting contractual requirements under agreements with multiple commercial, governmental, and other entities; and those other risk factors identified in the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of Novavax' Annual Report on Form 10-K for the year ended December 31, 2021 and subsequent Quarterly Reports on Form 10-Q, as filed with the Securities and Exchange Commission (SEC). We caution investors not to place considerable reliance on forward-looking statements contained in this press release. You are encouraged to read our filings with the SEC, available at http://www.sec.gov and http://www.novavax.com, for a discussion of these and other risks and uncertainties. The forward-looking statements in this press release speak only as of the date of this document, and we undertake no obligation to update or revise any of the statements. Our business is subject to substantial risks and uncertainties, including those referenced above. Investors, potential investors, and others should give careful consideration to these risks and uncertainties.

Contacts:

InvestorsErika Schultz | 240-268-2022[emailprotected]

MediaAli Chartan or Giovanna Chandler | 202-709-5563[emailprotected]

SOURCE Novavax, Inc.

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Novavax Nuvaxovid COVID-19 Vaccine Granted Expanded Conditional Marketing Authorization in the United Kingdom for Use in Adolescents Aged 12 Through...