MATFORS, Sweden, June 21, 2022 (GLOBE NEWSWIRE) -- Sweden-based NorthX Biologics (NorthX) is expanding into cell therapy manufacturing at its existing GMP-facility, as well as in premises at the Karolinska University Hospital campus in Stockholm. This initiative is part of NorthXs Innovation Hub, an Innovation Track designed to provide development and GMP-manufacturing services to the next generation of drug development companies and innovative research groups in need of NorthXs Good Manufacturing Practice (GMP) expertise.

NorthX has one of Northern Europes largest clinical-grade manufacturing capacities for plasmid DNA, recombinant proteins, cell banking and associated gene therapy services and this expansion into cell therapy is a major step to complete our offering for innovative clients. We are especially excited to work with Dr. Kristian Tryggvason, a leader in cell therapy technologies, and his team, said Dr. Ted Fjllman, CEO of NorthX.

After having built up BioLamina and its cell culture reagents that are used worldwide both in academia and industry, Dr. Tryggvason recently launched his latest venture: Alder Therapeutics. In addition to its own product development, the company now entered into an agreement to help NorthX expand its cell culture services to many new different cell types, including pluripotent stem cells. The Alder team will also help to design and validate NorthXs new process development and GMP-manufacturing labs in Matfors, alongside those being established at the Karolinska University Hospital campus in Stockholm.

Our goal is to be able to offer synergies to both cell and gene therapy clients and to collaborate with them through our Innovation Track, in which we work hand in hand with our clients regarding process development, manufacturing, and analytics to progress clinical programs and bring life-saving treatments to patients, added Aaron Small, NorthX VP of Global Sales and Corporate Development

Universities and cell therapy companies worldwide need GMP-grade development and manufacturing capacity, as it is complex and outside the scope of most biotech companies to build themselves. NorthX Biologics is already helping to translate cutting-edge gene therapy research into clinical development and now we will together build upon the existing broad cell therapy know-how in Sweden to do the same for cell therapies, said Dr. Kristian Tryggvason, CEO Alder Therapeutics.

About NorthX Biologics:

NorthX Biologics provides process development and manufacturing services with expertise in plasmids, proteins and other advanced biologics. NorthX Biologics sits in the heart of Sweden, and the team has been manufacturing biologics to GMP since 1988. In 2021 NorthX was recognized as a national innovation hub for advanced therapeutics and vaccines. NorthX has the ambition to become a leading cell and gene therapy manufacturer and partner of choice for innovative drug development companies. For more information see http://www.nxbio.com

About Alder Therapeutics:

Headquartered inStockholm, Alder Therapeutics AB is a novel development stage, cell therapy platform company aiming to develop and manufacture the best functional cell therapy products based on the most simple and robust processes. Alder Therapeutics unique cell therapy platform will allow manufacturing of better cells at a lower cost, which will make pluripotent cell therapy treatment available for ever more patients. Alder Therapeutics will play an important role in opening the next era in medicinal treatment. For more information and important updates, please visit. http://www.aldertx.com

For further information please email NorthX at:contact@nxbio.com

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NorthX Biologics expands to Cell Therapy: Partnership with Alder Therapeutics and new manufacturing site on Karolinska campus - GlobeNewswire

While historically lacking in foreign investments, Japans biotech scene is thriving with global investors showing increasing interest. Here are five of the hottest Japanese private companies innovating in the healthcare space.

Japan boasts one of the highest life expectancies in the world, and, faced with a rapidly aging population, is witnessing a growing burden of chronic conditions including cardiovascular disease and type 2 diabetes. For this reason, the Japanese healthcare authorities are encouraging research into the treatment and prevention of these diseases, in addition to promoting the potential of regenerative medicine.

In addition to having a roster of healthcare giants including Takeda, Astellas Pharma and Eisai, Japan is also an Asian hotspot for biotech companies. Upcoming startups have historically been limited in foreign funding and reliant on local venture capital players such as Nippon Venture Capital, Shinsei Capital Partners, and the University of Tokyo Edge Capital Partners.

In 2021, however, the amount of foreign investment flowing into the Japanese biotech space rose to $98 million, almost triple the haul of previous years. The most prominent global backers included Newton Biocapital, F-Prime Capital, and SoftBank Group. This trend arose as the COVID-19 pandemic triggered a wave of investor enthusiasm in biotechnology around the world.

With the help of local experts, weve listed five of the hottest private biotech companies in Japan. These firms, shown in alphabetical order, have raised large funding rounds in the last two years and are developing innovative treatments for a range of conditions including cancer, cardiovascular disease and inflammatory disorders.

Source: Shutterstock

Founded: 2017

Headquarters: Fujisawa

Chordia Therapeutics derives its name from the English term chord referring to a collection of musical notes normally played in harmony. In a similar way, the company aims to work in harmony with stakeholders and collaborators to develop first-in-class small molecule treatments for cancer.

Chordias lead program is a drug that disrupts the processing of RNA in tumor cells. In a healthy cell, RNA molecules are typically transcribed from a DNA template and spliced together to guide the production of new proteins. Some cancer cells accumulate mutations in the RNA splicing machinery and become vulnerable to Chordias drugs that interfere with this process.

Chordia raised $31 million (4 billion yen) in a Series C round in May 2022. The aim of the round was to push the companys lead drug through phase I testing and fund the preclinical development of the rest of its pipeline.

This month, the company announced interim results from the phase I trial of its lead candidate, with four of the recruited patients so far showing signs of responding to the treatment.

Founded: 2015

Headquarters: Tokyo

Heart failure occurs when the heart muscle is irreparably damaged and is unable to pump blood. While this deadly condition can be treated with a heart transplant, there is a general shortage of donors available, making a pressing need for alternatives.

In June 2021, the stem cell therapy developer Heartseed raised $36.5 million (4 billion yen) in a Series C round. The mission is to provide a regenerative route to saving the heart via stem cell therapy.

In the lab, Heartseed reprograms skin cells from the patient into a type of stem cell called induced pluripotent stem cells and grows these stem cells into heart muscle cells. The company then injects the muscle cells as a small cluster, or seed, into heart tissue to repair the muscle.

The proceedings from its Series C round will allow Heartseed to take its lead candidate into clinical development, including a phase I/II trial scheduled for later this year. Last year, Heartseed also licensed its treatment to Novo Nordisk in Denmark to co-develop the treatment outside of Japan.

Founded: 2018

Headquarters: Tokyo

LUCA Science hit the headlines in the last week for raising an impressive $30.3 million (3.86 billion yen) in a Series B round. The company is developing an unusual approach for treating a wide range of diseases: delivering a therapy based on mitochondria, the energy production plants in human cells.

One example where the technology could work well is in strokes and heart attacks, where blood flow is blocked to critical tissue in the brain and heart respectively. The reperfusion of blood to these tissues after the blockage can kill the tissue by damaging its mitochondria. Delivering healthy mitochondria could keep the tissue working properly and protect it from harm.

LUCA Science plans to use its recent Series B winnings to accelerate the preclinical development of its mitochondrial therapies and establish its manufacturing process. In May 2022, the firm also inked a collaboration deal with compatriot pharmaceutical company Kyowa Kirin Co., Ltd. to co-develop a mitochondrial therapy for rare genetic diseases.

Founded: 2016

Headquarters: Boston, U.S., and Tokyo

Modulus Discovery is a preclinical-stage drug discovery specialist. The company focuses on developing small molecule treatments for conditions such as cancer, inflammatory disorders and rare genetic conditions.

The firm uses a mixture of strategies to speed up the drug discovery process. These include simulating target proteins using a supercomputer; structural protein biology; forming collaborations such as with the peptide drug expert PeptiDream; and tapping into global networks for biological expertise. Modulus most advanced drug program is in late-stage preclinical testing for the treatment of chronic inflammatory diseases.

In March 2022, Modulus bagged $20.4 million (2.34 billion yen) in a Series C round. The cash is earmarked to advance the companys R&D programs by expanding its infrastructure, collaborations and headcount.

Founded: 2015

Headquarters: Tokyo

The name Noile-Immune is derived from blending together the phrases no illness and no immunity, no life. This company is developing CAR-T cell therapies for the treatment of cancer, which traditionally consist of extracting the patients immune T cells, engineering them in the lab to hunt down cancer cells, and reinfusing them into the patient.

Unlike approved CAR-T cell therapies, which are limited to treating forms of blood cancer, Noile-Immune aims its therapies at treating solid tumors. The company does this by engineering immune T cells to produce proteins that cause immune cells to migrate into the tumor site.

Noile-Immune is testing its lead candidate in a phase I in patients with solid tumors. The firm is also co-developing therapies with partners including Takeda and the European cell therapy specialists Adaptimmune and Autolus. Additionally, Noile-Immune has an allogeneic version of its cell therapy in the pipeline where immune T cells are sourced from healthy donors rather than the patient.

To finance the clinical development of its lead candidate, Noile-Immune raised $21.8 million (2.38 billion yen) in a Series C round in early 2021. The company hit a setback in January 2022 when a collaboration deal fell through with the U.S. player Legend Biotech. Nonetheless, other external companies remain interested in Noile-Immunes offering, including Japan-based Daiichi Sankyo Company Ltd., which opted to assess Noile-Immunes technology in late 2021.

Cover image via Elena Resko. Inline images via Shutterstock.

Thanks to feedback from Shiohara Azusa, VC investor at the University of Tokyo Edge Capital Partners, and Hironoshin Nomura, Chief Financial Officer at Sosei Group Corporation.

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Japan's five hottest biotech companies in healthcare - Labiotech.eu

Sickle cell beta-thalassemia refers to an inherited condition that impacts hemoglobin. People with the condition have different changes in each copy of their hemoglobin gene. One causes red blood cells (RBCs) to form a sickle shape and another reduces the amount of hemoglobin.

Sickle cell beta-thalassemia is a type of RBC disorder known as a hemoglobinopathy. These are conditions that cause abnormal hemoglobin production or a change in its structure. Hemoglobin is the protein in RBCs responsible for carrying oxygen around the body.

Both sickle cell disease and beta-thalassemia are genetic conditions that affect hemoglobin. A person with sickle cell beta-thalassemia inherits a trait for both conditions, impacting the shape and number of hemoglobin.

In this article, we will discuss the causes and risk factors for sickle cell beta-thalassemia, as well as symptoms and treatment options for the condition.

Sickle cell beta-thalassemia is a genetic condition and a type of sickle cell disease that features symptoms of both sickle cell disease and beta-thalassemia. It causes RBCs to take on a sickle shape, making them unable to flow through the blood vessels as smoothly. It also affects the amount of normal hemoglobin a person has in their blood.

It is an inherited condition, meaning that parents pass it on to their children. Researchers estimate that 57% of the global population are carriers of a significant hemoglobin mutation. People with sickle cell beta-thalassemia inherit a sickle trait from one parent and a beta-thalassemia trait from the other.

There are two types of sickle cell beta-thalassemia: plus (HbS beta+) and zero (HbS beta0). The former is the milder variant. The plus indicates that the blood contains a lower-than-average amount of normal hemoglobin. This differs from the latter, in which a person has no normal hemoglobin.

Sickle cell beta-thalassemia results from a change in the beta-hemoglobin (HBB) gene. The beta-hemoglobin gene is responsible for forming the hemoglobin subunit beta component of the hemoglobin protein.

A person develops sickle cell beta-thalassemia when they inherit one sickle cell trait from one parent and one beta-thalassemia trait from the other. The beta-thalassemia gene can be either beta+, which results in a lower production of normal hemoglobin, or beta0, which leads to a complete absence of normal hemoglobin.

Risk factors for a person developing sickle cell beta-thalassemia include having parents that may be carriers of the sickle cell, HbS beta+, or HbS beta0 gene. The condition follows an autosomal recessive inheritance pattern. This means that if a person has a beta-thalassemia trait and their partner has a sickle cell trait, there is a 25% chance with each pregnancy that their child will have sickle cell beta-thalassemia.

The symptoms a person may experience will depend on whether they have HbS beta+ or HbS beta0. The amount of normal hemoglobin a person can produce largely determines the severity of the condition.

A person with HbS beta+ will likely have milder symptoms, as while they produce less functioning hemoglobin than is typical, they can still produce normal hemoglobin. Individuals with either type will produce sickle-shaped RBCs that can block circulation and cause cell damage and pain.

A person with HbS beta+ may experience:

The symptoms of HbS beta0 are typically similar but with a more severe case of anemia.

Some newborn screenings include testing for sickle cell disorders such as sickle cell beta-thalassemia. Screening involves taking a blood sample and looking at the types of hemoglobin present in the newborns blood. However, newborn screenings only suggest the possibility of sickle cell beta-thalassemia and require further confirmatory tests. This may include the parents and any siblings having tests.

These tests may include:

Treatment aims to prevent complications from occurring and treat the symptoms a person may experience. This generally involves continuous care to prevent and manage potential problems.

In addition to the treatments below, a person may receive pain relief medications and antibiotics to help reduce pain and infections. The spleen usually helps prevent infections, but many people with sickle cell beta-thalassemia may lose spleen function. As such, children may receive penicillin prophylaxis to prevent pneumococcal sepsis.

Treatment options for sickle cell beta-thalassemia may include:

People may require hydroxyurea if they experience frequent periods of pain. Hydroxyurea is a drug that makes RBCs bigger and changes their shape to the typical round and flexible composition. This can help slow or prevent complications.

Hydroxyurea increases the level of fetal hemoglobin (HbF) in the body. HbF is present in higher quantities in newborns and can help protect against sickle cell complications. With higher levels of HbF, RBCs are less likely to become sickle-shaped.

According to the American Society of Hematology, people with sickle cell beta-thalassemia who take hydroxyurea also have fewer:

Some people with sickle cell beta-thalassemia may require blood transfusions. This is when a healthcare professional infuses healthy donor blood into the body of a person with sickle cell beta-thalassemia via a tube. The donor blood will need to have matching antigens to the blood of the person receiving the transfusion. These antigens, known as human leukocyte antigens, are proteins present on the surface of RBCs.

If the antigens do not match, the immune system of the person receiving the blood donation is more likely to reject the transfusion, and it may lead to a reaction that can cause health problems.

The bone marrow in the body produces blood cells. A person with dysfunctional bone marrow, such as in sickle cell beta-thalassemia, may receive hematopoietic stem cells from a healthy donor. This may help improve bone marrow function and reduce the symptoms of sickle cell beta-thalassemia. A hematopoietic stem cell transplant is currently the only cure for this condition.

Sickle cell beta-thalassemia is a type of sickle cell disease. Some evidence suggests the life expectancy of a person living with sickle cell disease is reduced by 2030 years compared with a healthy individual. Similarly, a 2019 study suggests a person with sickle cell disease may live roughly 22 fewer years than a person without the condition.

Advances in therapy and treatment options are helping to improve the outlook of people with sickle cell disease. However, statistics like these highlight the necessity to further develop approaches to improve the underlying morbidity and mortality of individuals with the condition.

Most often, a person will receive a diagnosis of sickle cell beta-thalassemia shortly after birth. Later in life, if they believe their current treatment regime is not suitable or notice worsening symptoms, they should contact their doctor.

If a person is aware of a family history of sickle cell disease, they may wish to consider undergoing genetic screening before attempting to have children.

Sickle cell beta-thalassemia is a type of sickle cell disease. It occurs when a person inherits a sickle cell trait and a beta-thalassemia trait from their parents. It results in a person having sickle-shaped RBCs and either producing a low amount of hemoglobin or none at all.

Symptoms of the condition can include mild to severe anemia, tiredness, weakness, pain, and possible organ damage. Treatment may involve pain relief medications, antibiotics, hydroxyurea, and blood transfusions. Some people may also receive a bone marrow transplant to help them produce healthy hemoglobin.

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Sickle cell beta thalassemia: Causes, symptoms, and treatments - Medical News Today

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SEATTLE, June 16, 2022 (GLOBE NEWSWIRE) -- Umoja Biopharma, Inc., an immuno-oncology company pioneering off-the-shelf, integrated therapeutics that reprogram immune cells in vivo to treat patients with solid and hematologic malignancies, announced today that it will have a poster presentation at the 2022 International Society for Stem Cell Research (ISSCR) Annual Meeting, to be held June 15-18, 2022 in San Francisco, California.

On Wednesday, June 15th, Principal Scientist & iPSC Team Lead, Teisha Rowland, Ph.D., will give a poster presentation titled, A Synthetic Cytokine Receptor Platform for Producing Cytotoxic Innate Lymphocytes as Off-the-Shelf Cancer Therapeutics. The presentation will discuss Umojas engineered induced pluripotent stem cell (iPSC) platform, that incorporates the synthetic cytokine receptor system rapamycin-activated cytokine receptor (RACR) platform. Umojas engineered iPSCs that are modified to express RACR, called RACR-induced cytotoxic innate lymphoid (iCIL) cells, drive differentiation and expansion of the cells while eliminating the need for expensive cytokines and other raw materials. The RACR platform has the potential to enable cytokine-free manufacturing and engraftment of the engineered cells in the patient without the need for toxic lymphodepletion.

Despite the advances chimeric antigen receptor T cell therapies have provided to the oncology space, we continue to battle significant challenges that these therapies cannot address, like limited expansion capacity and scalability, manufacturing complexity, variability among patients, and the need for toxic chemotherapy administration to combat patients anti-allograft response, said Andy Scharenberg, M.D., co-founder and Chief Executive Officer of Umoja. We are developing an engineered iPSC platform, including the RACR platform, to address these challenges by enabling a scalable, virtually unlimited, and simplified manufacturing of engineered, cancer-fighting cytotoxic innate lymphocytes.

About Umoja Biopharma

Umoja Biopharma, Inc. is an early clinical-stage company advancing an entirely new approach to immunotherapy. Umoja Biopharma, Inc. is a transformative multi-platform immuno-oncology company founded with the goal of creating curative treatments for solid and hematological malignancies by reprogramming immune cells in vivo to target and fight cancer. Founded based on pioneering work performed at Seattle Childrens Research Institute and Purdue University, Umojas novel approach is powered by integrated cellular immunotherapy technologies including the VivoVec in vivo delivery platform, the RACR/CAR in vivo cell expansion/control platform, and the TumorTag targeting platform. Designed from the ground up to work together, these platforms are being developed to create and harness a powerful immune response in the body to directly, safely, and controllably attack cancer. Umoja believes that its approach can provide broader access to the most advanced immunotherapies and enable more patients to live better, fuller lives. To learn more, visit http://umoja-biopharma.com/.

About RACR

CAR T cells generated by the body with VivoVec can be expanded and sustained with the rapamycin activated cytokine receptor (RACR) system, an engineered signaling system designed to improve chimeric antigen receptor (CAR) T cell persistence and produce durable anti-tumor responses. The RACR/CAR payload is integrated into the genomic DNA of a patients T cells. Rapamycin activates the RACR system resulting in preferential expansion and survival of cancer-fighting T cells. The RACR technology enables a patients cells to expand in a manner that resembles a natural immune response that does not require lymphodepletion, promoting durable T cell engraftment. RACR/CAR technology can also be used to enhanceex vivomanufacturing in support of more traditional autologous or allogeneic cell therapy manufacturing processes. To learn more about Umojas RACR platform please visit https://www.umoja-biopharma.com/platforms/

Media Contact:Darren Opland, Ph.D.LifeSci Communications[emailprotected]

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Umoja Biopharma Presents Data on its Engineered Induced Pluripotent Stem Cell Platform at the 2022 International Society for Stem Cell Research Annual...