Monthly Archives: October 2021


Strongbridge Biopharma plc Announces Completion of Acquisition by Xeris Pharmaceuticals, Inc.

DUBLIN, Ireland and TREVOSE, Pa., Oct. 05, 2021 (GLOBE NEWSWIRE) -- Strongbridge Biopharma plc (NASDAQ: SBBP) (the “Company” or “Strongbridge”) today announced that the High Court of Ireland (the "Court") has approved the previously announced proposed acquisition of Strongbridge by Xeris Pharmaceuticals, Inc. (Nasdaq: XERS) (“Xeris”) by means of a scheme of arrangement (the "Scheme") under Chapter 1 of Part 9 of the Irish Companies Act 2014 (the "Act").

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Strongbridge Biopharma plc Announces Completion of Acquisition by Xeris Pharmaceuticals, Inc.

Shockwave Medical to Report Third Quarter 2021 Financial Results on November 8, 2021

SANTA CLARA, Calif., Oct. 05, 2021 (GLOBE NEWSWIRE) -- Shockwave Medical, Inc. (Nasdaq: SWAV), a pioneer in the development and commercialization of Intravascular Lithotripsy (IVL) to treat complex calcified cardiovascular disease, today announced it will report financial results for the third quarter 2021 after market close on Monday, November 8, 2021. Company management will host a corresponding conference call beginning at 2:00 p.m. Pacific Time / 5:00 p.m. Eastern Time.

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Shockwave Medical to Report Third Quarter 2021 Financial Results on November 8, 2021

Terns Pharmaceuticals to Present at H.C. Wainwright 5th Annual NASH Investor Conference

FOSTER CITY, Calif., Oct. 05, 2021 (GLOBE NEWSWIRE) -- Terns Pharmaceuticals, Inc. (“Terns” or the “Company”) (Nasdaq: TERN), a clinical-stage biopharmaceutical company developing a portfolio of small-molecule single-agent and combination therapy candidates for the treatment of non-alcoholic steatohepatitis (NASH) and other chronic liver diseases, today announced that management will be presenting at the H.C. Wainwright 5th Annual NASH Investor Conference on Tuesday, October 12 at 2:30 p.m. ET.

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Terns Pharmaceuticals to Present at H.C. Wainwright 5th Annual NASH Investor Conference

Oncternal Therapeutics Announced Pre-Clinical Data from ONCT-534, an Androgen Receptor N-Terminal-Domain-Binding Small Molecule Degrader, Was Accepted…

SAN DIEGO, Oct. 05, 2021 (GLOBE NEWSWIRE) -- Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, today announced pre-clinical data from ONCT-534, an androgen receptor N-terminal-domain-binding small molecule degrader, was accepted for virtual poster presentation at the AACR-NCI-EORTC Virtual International Conference on Molecular Targets and Cancer Therapeutics on October 7-10, 2021. Oncternal has conducted a series of preclinical studies in collaboration with the Center for Cancer Research at the University of Tennessee Health Science Center.

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Oncternal Therapeutics Announced Pre-Clinical Data from ONCT-534, an Androgen Receptor N-Terminal-Domain-Binding Small Molecule Degrader, Was Accepted...

Neuronetics Announces Commercial Agreement with Success TMS

MALVERN, Pa., Oct. 05, 2021 (GLOBE NEWSWIRE) -- Neuronetics, Inc. (NASDAQ: STIM), a commercial-stage medical technology company focused on designing, developing and marketing products that improve the quality of life for patients who suffer from neurohealth disorders, today announced a commercial agreement with Success TMS in which Success TMS will exclusively utilize Neuronetics’ platform, NeuroStar Advanced Therapy for Mental Health, for the treatment of patients suffering from treatment-resistant major depressive disorder (MDD). In connection with the commercial agreement, the Company has made a strategic investment in Success TMS in the form of a $10 million term loan to further accelerate the growth of Success TMS’ business.

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Neuronetics Announces Commercial Agreement with Success TMS

Eledon Pharmaceuticals Announces Upcoming Presentation on AT-1501 at the Virtual 2021 International Pancreas and Islet Transplantation Association…

IRVINE, Calif., Oct. 05, 2021 (GLOBE NEWSWIRE) -- Eledon Pharmaceuticals, Inc. (“Eledon”) (NASDAQ: ELDN), a clinical stage biopharmaceutical company focused on developing targeted medicines for persons living with autoimmune disease, requiring an organ or cell-based transplant, or living with amyotrophic lateral sclerosis (ALS), announced an upcoming presentation on AT-1501, its lead clinical candidate, at the International Pancreas and Islet Transplantation Association (IPITA) Congress, which is being held virtually from October 20-23, 2021.  An oral presentation on the use of AT -1501, and pre-clinical data showing the effectiveness of AT-1501 in preventing islet allograft rejection in nonhuman primate models, will be featured.

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Eledon Pharmaceuticals Announces Upcoming Presentation on AT-1501 at the Virtual 2021 International Pancreas and Islet Transplantation Association...

The Role of Quality and Speed in Custom Model Generation – FierceBiotech

The pressure to produce results quickly during the drug discovery and development process continues to increase as does the role of genetically engineered custom mouse models. However, even the fastest custom mouse model generation projects take about 6 months to reach the stage when a few F1 heterozygous mice are available for experiments or breeding. This timeline increases if more than a few heterozygotes or homozygotes are needed. Taconics ExpressMODEL portfolio of products shifts the deliverable of a model generation project from a few heterozygous F1 mice to a much larger cohort of 10-100 mice while reducing the timeline to obtaining data, adding predictability, all without compromising essential quality control steps. By applying innovative thinking and leveraging our ability to seamlessly integrate custom model generation, embryology and colony management services, the ExpressMODEL portfolio achieves the industry's fastest timelines to study cohort with no compromise in quality for models generated using embryonic stem cell (ESC), CRISPR, or random integration transgenic (RITg) methodology.

Regardless of the methodology used to generate the founder animals, ExpressMODEL is built around the concept that using in vitro fertilization (IVF) rather than conventional breeding to generate the F1 mice from founders has a number of distinct advantages including:

However, ExpressMODEL is more than simply using IVF to produce F1 mice because the quality of the male founders needs to be high in order to fully realize the advantages IVF provides. High quality means that founder males need to have both a high percentage of the desired genetically-modified gene and a high fertility rate. Thus, the candidate founder males need to be well-characterized. Because the different methodologies used for model generation produce founders with different characteristics, we have developed unique founder analysis protocols to fit the three different methodologies. The founders produced from injection of ESCs into blastocyst-stage embryos are called chimeras because they are derived from two different populations of genetically distinct cells that originated from different embryos. The founders produced by introducing CRISPR reagents or transgenic DNA into one-cell embryos (zygotes) are mosaics meaning they are composed of two or more different populations of genetically distinct cells that originated from the same embryo.

ExpressMODEL: Embryonic stem cell (ESC)

ESC-mediated mouse model generation remains the gold standard and best choice for complex projects such as genomic replacement humanizations. Using ExpressMODEL: ESC, the timeline for a typical project that would take 66 weeks to deliver a homozygous study-size cohort is reduced to 54 weeks, saving at least 3 months. The key components of ExpressMODEL: ESC are:

The data from these analyses facilitate the choice of founder male(s) to be utilized for the IVF to produce an F1 heterozygous cohort that is sized to meet the customers downstream goals and timeline requirements. It is important to note that all quality control steps in vector construction and ES cell targeting are preserved.

ExpressMODEL: CRISPR

Two great advantages of CRISPR methodology are the speed at which a genetically engineered model can be generated and the ability to modify a wide range of genetic backgrounds, including existing genetically-engineered models. Using ExpressMODEL: CRISPR, the timeline for a typical project that would take 48 weeks to deliver a homozygous study-size cohort is reduced to 36 weeks, saving at least 3 months. ExpressMODEL: CRISPR combines our ability to produce founders with a low degree of mosaicism and to accurately estimate the degree of mosaicism of each founder male. The key components of ExpressMODEL: CRISPR are:

ExpressMODEL: Random Integration Transgenic (RITg)

More than 30 years since the first RITg model was generated, the method continues to be a favored path to quickly generate gain of function models that express an ectopic gene. However, because genomic integration of the transgene is random in each injected embryo, the resulting founder line are unique and may or may not perform to the desired specifications. Additionally, each founder often has transgene insertions at multiple sites and the configuration and copy number of those insertions differs. Thus, RITg founders can be more genetically complex than CRISPR founders. As a result, common practice is to separately propagate multiple lines to generate offspring for extensive transgene expression studies. These data are then used to determine which founder line(s) to propagate. The cost and time of this downstream breeding and characterization of multiple founder lines greatly exceeds the original cost to generate the lines and takes significant additional time. Because transgene expression is assessed in founder animals, ExpressMODEL: RITg takes the guesswork out of the process and allows one to avoid the cost of breeding and characterizing multiple founder lines. Moreover, it reduces project timeline by at least 12 weeks and potentially up to 24 weeks or more. The key components of ExpressMODEL: RITg are:

Additional customizable options are available including the provision of tissue lysates and fixed tissue for protein expression analyses, and transgene mapping analysis to accurately determine the transgene integration site and configuration.

Taconics ExpressMODEL suite of technologies is designed to reduce the custom model generation timeline from project conception to study cohort without taking any shortcuts that compromise quality. Taconic provides a seamless end-to-end solution incorporating industry leading model generation, embryology, and colony management capabilities that allows your project to travel in the express lane.

Interested in learning more about custom animal model generation? Visit Taconic's website at http://www.taconic.com.

This article was created in collaboration with the sponsoring company and our sales and marketing team. The editorial team does not contribute.

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The Role of Quality and Speed in Custom Model Generation - FierceBiotech

Stem cells may be the key to saving white rhinos from extinction – Sciworthy

It is too late for conservation efforts to save the northern white rhinoceros, but with recent scientific advancements there may still be hope to bring back this beloved species. In a recently published paper, scientist Marisa Korody and her colleagues at San Diego Zoo Global (USA) and at the Department of Molecular Medicine at Scripps Research (USA) describe their exciting progress on using stem cells to revive the northern white rhino.

The northern white rhino is functionally extinct, meaning there are not enough of these rhinos left to save the species. In fact there are only two northern white rhinos left: a mother and a daughter. But for decades, scientists have preserved cell samples from 15 northern white rhinos containing enough genetic material to potentially bring this species back from the brink. These preserved samples hold fibroblast cells the type of skin cells that secrete collagen from white rhinos. With these scientists newly developed methods, fibroblast cells can be converted into something much more valuable: induced pluripotent stem cells. These stem cells can differentiate into any cell type in the body including heart cells, muscle cells, and reproductive cells.

In theory, by converting fibroblast cells into reproductive cells, scientists could create genetically unique rhino embryos. Alongside other assisted reproduction technologies, scientists could implant a new embryo into a closely-related southern white rhino, where the baby northern white rhino could develop as an otherwise normal pregnancy. By completing this process multiple times, scientists may be able to establish a stable population of northern white rhinos.

In 2011, this research team generated induced pluripotent stem cells from the samples of another endangered species, but unfortunately since this process was found to harm the recipient genomes, this method was largely unsuccessful. Despite this setback, in 2015 the authors met with colleagues worldwide to consider ways to save the northern white rhino, and they concluded that methods involving induced pluripotent stem cells may still be the most promising solution. Over the following years, the scientists worked to improve their methods, and these improvements are documented in their recent paper. These experiments represent the first step in a long-term plan to bring the northern white rhino back through assisted reproduction techniques.

Right from the start, the scientists faced a whole host of challenges. Through trial and error they modified the growth medium for the cells, optimizing it for rhinoceros cells. With their improved growth medium, scientists successfully generated induced pluripotent stem cell lines from 11 rhinoceros individuals. This has never been done before and represents a huge stride forward in the path to recovering this species.

Before trying to make their first rhino, the scientists needed to stress these induced pluripotent stem cells and sequence their genomes to determine if the cell quality is good enough to potentially produce new, viable rhinos. They maintained colonies of these cells in long-term cultures and exposed these colonies to different conditions to give insight into how resilient these cells could be. These tests demonstrated that long-term culture did not affect the potential for these cells to differentiate into cardiac lineage cells, confirming that these cells are stable long-term. The researchers also confirmed that these pluripotent cells could potentially produce gametes, the egg and sperm cells that are used for sexual reproduction. These advancements indicate that with these newly developed protocols, induced pluripotent stem cells are a promising tool that could someday help recover the northern white rhino.

Although this study includes some exciting results, there is still much work to do. For example, scientists must now sequence the genomes of the northern and southern white rhino so other researchers can analyze the stem cells ability to stay the same over time. Despite the work that still needs to be done, these promising advancements could someday help the northern white rhino population recover. This method may also work for saving other endangered or extinct species, as long as the genetic material needed is available. Long-term, these scientists plan to continue a series of experiments that could ultimately bring this beloved rhino, and potentially other endangered species, back from the brink of extinction.

Original study: Rewinding Extinction in the Northern White Rhinoceros: Genetically Diverse Induced Pluripotent Stem Cell Bank for Genetic Rescue

Study published on: February 15, 2021

Study author(s): Marisa L. Korody, Sarah M. Ford, Thomas D. Nguyen, Cullen G. Pivaroff, Iigo Valiente-Alandi, Suzanne E. Peterson, Oliver A. Ryder, and Jeanne F. Loring

The study was done at: San Diego Zoo Global (USA), Scripps Research (USA)

The study was funded by: San Diego Zoo Global and San Diego Zoo Wildlife Conservancy donors, including Anne and Christopher Lewis, and the Robert Kleberg and John and Beverly Stauffer Foundations; and Uma Lakshmipathy from Thermo Fisher Scientific for providing supplies

Raw data availability: Contact author for data (all other data are accessible from the article or supplementary materials)

Featured image credit: Hein waschefort, CC BY-SA 3.0, via Wikimedia Commons

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Stem cells may be the key to saving white rhinos from extinction - Sciworthy