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Category Archives: Gene Medicine

Rigenerand receives regulatory approval for gene therapy production – Cleanroom Technology

5-Jun-2020

Pharmaceuticals | Regulatory

Authorisation has enabled Rigenerand to enter into clinic, offer CDMO services and consultancy, and plan to expand cleanroom capacity

Rigenerand, a biotech company that both develops and manufactures medicinal products for cell therapy applications, primarily for regenerative medicine and oncology, has announces it has received authorisation from the Italian Medicine Authority (AIFA) to produce gene therapy medicinal products for clinical purposes.

This authorisation enables Rigenerand to manufacturer its own autologous gene therapy medicinal product (RR001) for the treatment of pancreatic cancer. It also authorises the company to start its phase I first-in-man clinical trial of RR001, now expected to start in Q1, 2021.

RR001 has been granted Orphan Drug Designation (ODD) by both the US FDA and European Commission COMP (Committee for Orphan Medicinal Products). This ODD offers the opportunity for Rigenerand to request an accelerated assessment procedure and quicker development pathways towards a marketing authorisation in the US and EU.

Rigenerand will also now offer direct GMP CDMO services to international and Italian partners in clinical development of cell and gene therapy products (ATMPs). The company will utilise its experience from its drug development and diagnostic arms to deliver a science-based approach to its GMP manufacturing services and cell-based medicinal products development.

Rigenerand plans to expand its manufacturing facility from five sterile cleanrooms suits. This will be by implementing further closed system and isolator technology in pre-designated areas in its facilities.

The five sterile cleanrooms are within the Rigenerand GMP facility, which contains a Biosafety Level 3 (BSL3) negative pressure area, suitable to handle genetically modified microorganisms (MOGM), viruses and Risk Group 3 microorganisms, as well as a Biosafety Level 2 (BSL2), positive pressure area: suitable to manipulate non-infectious cell based products and Risk Group 2 microorganisms. Rigenerand's cleanroom technology offers the flexibility to scale-up the processes from academic / hospital laboratories, and the feasibility of technology transfer of manufacturing processes from other cell factories in order to expand their process capability.

In addition, Rigenerand is now authorised to deliver consultancy to biotechnology and pharmaceutical companies on cell and gene therapy development and manufacturing. This consultancy includes expanding process capabilities and developing early-stage cell and gene therapy medicinal products for clinical purposes.

As the cell and gene therapy sector continues to grow, with increased numbers of therapies moving through clinical development and onto commercialisation, demand for CDMO services will continue to grow. There is an increased demand for global networks of CDMO GMP cell and gene therapy manufacturing. This calls for an improved capacity to treat patients whilst reducing logistical complexities, issues, risks, and costs.

"The regulatory approval for Rigenerand to produce gene therapies for clinical development now enables Rigenerand to enter the clinic with its own gene therapy product to target pancreatic cancer," said Massimo Dominici, scientific founder, Rigenerand. "Combined with the Orphan Drug Designation, the approval will enable Rigenerand to choose an accelerated pathway to bring a gene therapy approach to pancreatic cancer patients with little alternative therapeutic option."

"The authorisation is also essential in allowing Rigenerand to offer its much needed GMP CDMO services to the wider cell and gene therapy sector," said Giorgio Mari, Rigenerand CEO. "We will be expanding our CDMO facility to cater for increasing demand. Operating as both a developer with a clinical pipeline as well as a CDMO has resulted in an unrivalled blend of expertise for us to offer to partners and the wider cell and gene therapy industry."

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Scientists Find A Switch To Turn Off A Gene That Causes Aggressive Breast Cancer – Forbes

ROSTOV-ON-DON, RUSSIA - FEBRUARY 11, 2020: Breast oncologist Yelena Chernikova examines a patient's ... [+] scan at the Rostov-on-Don Research Institute of Oncology. Valery Matytsin/TASS (Photo by Valery MatytsinTASS via Getty Images)

Researchers at Tulane University School of Medicine are celebrating a breakthrough in the fight against an aggressive breast cancer.

Theyve identified a gene that causes an aggressive form of breast cancer to rapidly grow. And most importantly, theyve found a way to flip the switch and turn the gene off to prevent cancer from occurring.

They say their studies on mice are so compelling they are seeking FDA approval to begin clinical trials.

Dr. Reza Izadpanah and his team examined the role two genes play (including one they discovered) in causing triple negative breast cancer (TNBC), which is considered the most aggressive type of breast cancer.

They specifically identified an inhibitor of the TRAF3IP2 gene, which they found suppressed the growth and spread of TNBC in mouse models that closely resemble humans.

In parallel studies, they examined how TRAF3IP2 and another gene called RAB27a play roles in the secretion of substances that can cause tumor formation.

They found that suppressing the expression of either gene led to a decline in tumor growth and spread of cancer to other organs.

When Rab27a was suppressed, the tumor didnt grow but it continued to spread a small number of cancer cells to other parts of the body. But when researchers turned off the TRAF3IP2 gene, they found no spread (metastasis) of the original tumor cells for a full year following treatment.

In addition, they say inhibiting the TRAF3IP2 gene not only stopped future tumor growth, but caused existing tumors to shrink to undetectable levels.

Dr. Izadpanah says Our findings show that both genes play a role in breast cancer growth and metastasis. This exciting discovery has revealed that TRAF3IP2 can play a role as a novel therapeutic target in breast cancer treatment.

Other researchers, such as Dr. Bysani Chandrasekar of the University of Missouri, have joined in the Tulane research efforts and found that targeting TRAF3IP2 can stop the spread of glioblastoma, a deadly brain cancer.

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Why Editas Medicine Is Now the CRISPR Stock to Really Watch – Motley Fool

Based on market cap,CRISPR Therapeutics (NASDAQ:CRSP)ranks as the top biotech focused on developing CRISPR gene-editing therapies. It's more than 2 1/2 times the size ofEditas Medicine (NASDAQ:EDIT) and nearly four times larger thanIntellia Therapeutics (NASDAQ:NTLA).

But based on stock performance so far in 2020, Intellia wins the prize as the hottest CRISPR biotech stock. Its shares have soared more than 40%, thanks in large part to the expansion of its partnership with Regeneron.

While CRISPR Therapeutics and Intellia have captured investors' attention lately, Editas Medicine could now be the CRISPR stock to really watch. There are both near-term and long-term reasons why investors should keep their eyes on this company.

Image source: Getty Images.

In March, Editas and its partner Allerganannounced the dosing of the first patient in a phase 1/2 clinical study evaluating EDIT-101 in treating Leber congenital amaurosis type 10 (LCA10), an inherited form of blindness. Editas CEO Cynthia Collins called it "a truly historic event," as it wasthe world's first human study of anin vivo (inside the body) CRISPR gene-editing therapy.

Editas' Chief Scientific Officer Charlie Albright stated in the company's Q1 conference calllast month that the study "has been cleared to continue based on a review of safety data on the first patient." That's great news, especially considering the pioneering nature of the LCA10 therapy.

I don't necessarily look for this clinical trial to provide a big catalyst for Editas over the next few months, at least not directly. But it could give the biotech an indirect catalyst.

Editas Medicine's experience with EDIT-101 in targeting LCA10 has enabled it to move forward with EDIT-102, a CRISPR therapy targeting another genetic eye disease, Usher syndrome 2A. Allergan is currently reviewing a preclinical data package for the potential licensing of EDIT-102. Editas expects a decision from Allergan on exercising its option for EDIT-102 by the third quarter of 2020.

My hunch is that Allergan will decide to license EDIT-102 unless some safety issue emerges in the phase 1/2 study for EDIT-101. A positive decision would likely cause Editas' shares to jump.

CRISPR Therapeutics is the leader in developing a CRISPR therapy for treating rare blood diseases sickle cell disease and beta-thalassemia. The company and its partner, Vertex Pharmaceuticals, expect to report additional data from two phase 1/2 studies in progress evaluating CRISPR/Cas9 gene-editing therapy CTX001 later this year.

Editas is behind CRISPR Therapeutics right now. But I won't be surprised if Editas emerges as a winner in sickle cell disease and beta-thalassemia over the long term.

The company plans to file for FDA approval by the end of 2020 to begin clinical testing of EDIT-301 in treating sickle cell disease. EDIT-301 uses its proprietary enzyme Cas12a (also known as Cpf1) instead of Cas9, the enzyme most commonly used in CRISPR gene-editing therapies.

Editas thinks that EDIT-301 could be the best-in-class CRISPR therapy for treating both sickle cell disease and beta-thalassemia. One reason behind the biotech's confidence is that the therapy edits the HBG1 and HBG2 genes rather than theBCL11Ae gene targeted by CRISPR Therapeutics' CTX001. Editas believes that this difference will give EDIT-301 a better safety profile than CTX001 will have. The company also thinks that using Cas12a will lead to sustained higher fetal hemoglobin levels than using the Cas9 enzyme will.

There's another intriguing possibility for Editas Medicine. Its partner on EDIT-101, Allergan, was recently acquired by AbbVie (NYSE:ABBV). The primary reason for this deal was for AbbVie to reduce its dependence on Humira, which faces biosimilar competition in the U.S. beginning in 2023.

AbbVie has other arrows in its quiver for offsetting the inevitable loss of revenue from Humira -- notably including its new immunology drugs Rinvoq and Skyrizi. However, the closer the date approaches for Humira's U.S. sales decline, the more I suspect that AbbVie will be interested in making additional smaller deals to boost its top line.

If EDIT-101 is successful in phase 1 testing and advances to phase 2, Editas Medicine could very well be on AbbVie's acquisition radar. The biotech wouldn't be so expensive that it would require AbbVie to take on a lot of additional debt. Buying Editas could also boost AbbVie's oncology program since Editas has several preclinical programs that use CRISPR gene editing in cancer cell therapies.

To be sure, Editas Medicine is a speculative play. For that matter, so are CRISPR Therapeutics and Intellia Therapeutics. All of these biotech stocks face significant risks that their gene-editing therapies won't work or won't be safe. But the possibility of near-term catalysts and the tremendous long-term potential for Editas make this CRISPR biotech one for investors to closely watch.

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Research Roundup: How Tau Proteins Spread in Alzheimer’s and More – BioSpace

Every week there are numerous scientific studies published. Heres a look at some of the more interesting ones.

Toxic Tau Proteins Spread in Alzheimers Patients Via Connected Neurons

Two abnormal proteins are associated with Alzheimers disease, beta-amyloid and tau. A study out of Lund University in Sweden and McGill University in Canada showed how toxic tau in the human brain in elderly individuals spreads by way of connected neurons. They also found that beta-amyloid facilitates the spread of toxic tau. The research was published in the journal Nature Communications.

Our research suggests that toxic tau may spread across different brain regions through direct neuronal connections, much like infectious diseases may spread to different cities through different transportation pathways, said lead author Jacob Vogel from McGill. The spread is restricted during normal aging, but in Alzheimers disease the spread may be facilitated by beta-amyloid, and likely leads to widespread neuronal death and eventually dementia.

Beta-amyloid forms plaques in the brain and tau forms tangles within brain cells. Toxic tau, in particular, has been linked to brain degeneration and cognitive symptoms. In general, beta-amyloid appears earlier in the disease with tau appearing later.

Our findings have implications for understanding the disease, but more importantly for the development of therapies against Alzheimers, which are directed against either beta-amyloid or tau, said Oskar Hansson, co-lead investigator of the study and professor of neurology at Lund. Specifically, the results suggest that therapies that limit uptake of tau into the neurons or transportation or excretion of tau, could limit disease progression.

Improving on Gene Therapy by Decreasing Immune Response to AAV

Biotech company Spark Therapeutics published research in the journal Nature Medicine showing that treatment with immunoglobulin G-degrading enzyme of Streptococcus pyogenes (IdeS0 caused fast and transient decrease of neutralizing anti-adeno-associated virus (AAV) antibodies and restored gene therapy efficacy in laboratory animals. The study was conducted by Spark, Genethon, the Centre de Recherche des Cordeliers (Inserm, Sorbonne Universite, Universite de Paris) and the National Centre for Scientific Research (CNRS) in France.

Biggest Risk Factors for Severe COVID-19 in UK

In a large cohort study published in The BMJ of COVID-19 patients in the UK, the biggest risk factors for severe disease or death were found to be age over 50, being male, obese, or having underlying heart, lung, liver and kidney disease. The study, which is still ongoing, recruited over 43,000 patients. The study essentially looked at data from a third of all COVID-19 patients admitted to hospitals in the UK between February 6 and April 19, 2020. Overall, the data confirms studies conducted in China, although obesity was not highlighted in the China data. The researchers believe that reduced lung function or obesity-related inflammation are the factors involved in increased disease severity or mortality in obese patients.

Warmer Temperatures Slow COVID-19A Little Bit

Researchers at Mount Auburn Hospital evaluated the impact of temperature, precipitation and UV index on COVID-19 cases in the U.S. during the spring of 2020. They found that while the rate of COVID-19 incidence decreases with warmer temperatures up to 52 degrees F, anything warmer than that does not decrease disease transmission all that much. Precipitation doesnt seem to have any effect and UV index helps a little bit. The bottom line, they say, is that their research supports what the Centers for Disease Control and Prevention (CDC) is saying, which is that although the pandemic might abate a little bit in the summer, it is expected to be worse in the fall and winter.

Antibodies Against Alzheimers Toxic Particles

Investigators at the University of Cambridge have identified a method to design an antibody that can seek out and attack the toxic particles that destroy healthy brain cells, such as in Alzheimers disease. These antibodies recognize amyloid-beta oligomers. They believe this could lead to new diagnostics or possible treatments for Alzheimers and other types of dementia.

Oligomers are difficult to detect, isolate, and study, said Francesco Aprile, the studys lead author. Our method allows the generation of antibody molecules able to target oligomers despite their heterogeneity, and we hope it could be a significant step towards new diagnostic approaches.

Physical Distancing, Masks and Eye Protection Help Prevent COVID-19

As has been suggested all along, the use of physical distancing, face masks and eye protection does appear, in a systematic review of the literature by researchers at McMaster University, to help prevent the transmission of COVID-19. The two meters (about six feet) physical distancing seems to prevent person-to-person transmission and face masks and eye protect decrease the risk of infection.

Although the direct evidence is limited, the use of masks in the community provides protection, and possibly N95 or similar respirators worn by health care workers suggest greater protection than other face masks, said Holger Schunemann, professor of the departments of health research methods, evidence, and impact, and medicine at McMaster. Availability and feasibility and other contextual factors will probably influence recommendations that organizations develop about their use. Eye protection may provide additional benefits.

The review was led by McMaster researchers, but also included a large, international collaboration of researchers, front-line and specialist clinicians, epidemiologists, patients, public health and health policy experts of published and unpublished studies in any language. They also evaluated direct evidence on COVID-19 and indirect evidence on other coronaviruses, such as the ones that cause SARS and MERS. Although there were no randomized control trials addressing the three coronaviruses (SARS, MERS and COVID-19), they found 44 relevant comparative studies in health care and community settings across 16 countries and six continents from inception to early May 2020. The study was published in The Lancet.

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Research Roundup: How Tau Proteins Spread in Alzheimer's and More - BioSpace

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The Boy Slumped to the Floor. Could These Be Seizures? – The New York Times

Earlier that day, her teenage daughters watched a Netflix documentary series called Diagnosis, which is produced by The New York Times and is based on this column. Before filming began, I wrote about patients with undiagnosed cases in special online versions of this column. The idea was to use the broad reach of the internet to try to find help for them.

The daughters watched an episode featuring a 6-year-old girl named Kamiyah who had spells that looked exactly like Dannys. They called their mother, told her what theyd seen and stayed on the phone as she watched the show. Twenty minutes in, they heard her gasp. She saw it, too. Thats when she picked up her phone and called her sister-in-law.

Dannys mother fast-forwarded through the first few minutes of the episode until she saw the girl collapse and then recover, just as her son did. She rewound it and watched from the beginning.

The mother on the screen, Breteni, described how Kamiyah first started to have these spells when she was 8 months old and learning to crawl. That child had gone to the National Institutes of Health Undiagnosed Diseases Network (U.D.N.) a program dedicated to finding answers for patients who did not have diagnoses after a full investigation. Doctors at the U.D.N. discovered that the girl had a rare genetic abnormality shared by only a handful of children in the world. The affected gene, known by the name KCNMA1, made an aberrant version of a piece of cellular machinery in the brain. That abnormality causes episodes of collapse in which the body simply seems to grind to a halt, then start again.

The woman called her husband, and they watched the show together. He, too, was convinced that theyd found the cause of their sons episodes. Theyd taken Danny to a geneticist early in their search for a diagnosis, but the genetic analysis didnt reveal anything. They went back and asked the same doctor to test their son again, this time specifically for the KCNMA1 gene an abnormality unknown at the time he had his first test. It took two months for the results to come back: The boy had a mutation in his KCNMA1 gene, just the way Kamiyah did.

Dannys parents found Breteni and her daughter and told them about Dannys successful treatment. Kamiyah, too, was first thought to have epilepsy, but the medicines she was given seemed to make her episodes even worse. Since then, Breteni had been reluctant to treat Kamiyah with any medication. But after hearing what this medication did for Danny allowing him to go to school, to learn, to make friends Breteni reached out to her daughters neurologist, and with his approval started Kamiyah on Vyvanse. The results were immediate. Within days, Kamiyah went from having hundreds of spells a day to having none at all at least not while the medication was in her system.

After Kamiyahs story was told, researchers began looking for ways to help patients with this unusual genetic mutation. Andrea Meredith, a neuroscientist at the University of Maryland School of Medicine, had spent her career studying this gene in mice. She contacted Breteni, after hearing about Kamiyah, to share what she had learned about the gene and its diseases and to work with them to find a treatment. Matthew Might, a researcher at the University of Alabama at Birmingham School of Medicine, also decided to look for medications to help those with this mutation after reading Kamiyahs story. Might hadnt even considered Vyvanse until Breteni told him about how well it worked for her daughter and for Danny. And hes now looking for other drugs that will have the same positive effects as Vyvanse, but with fewer of the side effects from taking amphetamines.

Until then, Kamiyah and the handful of others like her can still enjoy something they never had before a nearly normal life. In April, after three weeks on this medication, Kamiyah learned to ride a bike, something her mother never dreamed would be possible. She still has the training wheels on, but she and her mother are looking forward to a day when even those might come off.

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The Boy Slumped to the Floor. Could These Be Seizures? - The New York Times

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Some types of prostate cancer may not be as aggressive as originally thought – Newswise

FINDINGS

Newswise Researchers at the UCLA Jonsson Comprehensive Cancer Center analyzed gene-expression patterns in the most aggressive prostate cancer grade group known as Gleason grade group 5 and found that this grade of cancer can actually be subdivided into four subtypes with distinct differences. The findings may affect how people are treated for the disease.

One subtype, which accounts for about 15% of the grade group 5 cancers, has highly aggressive features and is associated with much worse outcomes than the other subtypes. Another, which makes up about 20% of the tumors, appears to be much less aggressive and may not require intensified and aggressive treatments. Traditionally, all tumors in Gleason grade group 5 have been treated in the same way.

BACKGROUND

Prostate cancer is the leading solid-tumor cancer among men in the United States and a major cause of morbidity globally. While early-stage, localized prostate cancer is curable, current treatments dont always work for everyone. To find out why standard treatment may work for some and not others, the UCLA researchers looked at tumors in the Gleason grade group 5 subset of prostate cancer. These tumors are at the highest risk to fail standard treatment, leading to metastasis and death. The researchers thought that studying the gene expression the unique signature of each cancer cell in these tumors might provide insight into how to make treatments more personalized for each patient.

METHOD

The researchers first analyzed data from more than 2,100 Gleason grade group 5 tumors, looking at how the genetic blueprints differed among the tumors. They identified distinct clusters of subgroups and validated their findings by analyzing an additional cohort of more than 1,900 Gleason grade group 5 prostate cancers.

IMPACT

By using the genetic information from tumors in men with prostate cancer, physicians hope to one day create more personalized treatments based on the actual characteristics of the cancer. This information will help optimize quality of life and avoid overtreating subgroups of men who may not need aggressive treatments.

AUTHORS

The studys lead author is Dr. Amar Kishan, an assistant professor of radiation oncology at the David Geffen School of Medicine at UCLA and a researcher at the UCLA Jonsson Comprehensive Cancer Center. The co-senior authors are Dr. Joanne Weidhaas, a professor of radiation oncology and director of translational research at the Geffen School of Medicine, and Paul Boutros, a professor of urology and human genetics and director of cancer data science for the Jonsson Cancer Center. Boutrosis also a member of the UCLA Institute of Urologic Oncology and the Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Research at UCLA. Other UCLA authors include David Elashoff, Dr. Rob Reiter and Dr. Matthew Rettig.

JOURNAL

Thestudy was publishedin the journal European Urology.

FUNDING

The research was funded in part by an award from the American Society for Radiation Oncology and the Prostate Cancer Foundation, the Radiological Society of North America, and the National Institutes of Health.

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