Category Archives: Transhuman News

Sven Kili is a busy and influential man. In addition to being CEO of Geneva startup Antion Biosciences, hes also an Honorary Professor at University College London, a board member of the Centre for Commercialization of Regenerative Medicine, Chair of the BioIndustry Associations Cell and Gene Therapy Advisory Committee, Co-chair of the Finance and Business Development Committee at the International Society for Cell & Gene Therapy (ISCT), and the list goes on

We sat down with Kili to discuss his perspective on the future of cell and gene therapy and the success of ISCT 2022.

My time serving as a meeting co-chair at ISCT 2022 was absolutely amazing a reawakening! It was the second in person, three dimensional meeting that I had attended since the onset of the pandemic, and it was a real treat to return to catching up with real people old friends, in fact over real cups of coffee. Something as simple as being present in a room while a speaker was presenting, and then being able to interact with them in person was really great.

There were several noticeable changes. The first was the way one asked questions. Very often in remote meetings, your questions are asked in a text box. Then, they could end up being interpreted literally or figuratively by a moderator, before they reach the speaker.

In addition, asking follow-up questions or requesting clarification becomes very onerous. Thus, all questions in virtual events tend to be short and open ended.

By contrast, offline events are far better suited for dialogue for a back and forth that allows for additional clarifying questions. These can then be taken even further after the event, by approaching the given speaker for a longer discussion, perhaps over one of those real cups of coffee.

There is the magic of chance encounters. For example, you might run into an old friend in a corridor, and find yourself lured into tagging along at a session that turns out to be much better than you would have expected, covering topics and taking a format you might never have anticipated. Such novelty only happens offline. A little bit of chaos goes a long way.

More powerful forms of learning happen offline, too. This year I attended a session outside my comfort zone, on manufacturing. At the session, the speaker presented a comparison of a number of cell therapy manufacturing machines and compared their output. It opened up lines of thought in me that would have remained closed had I attended via laptop, perched on a distant dining room chair.

First, there was a roundtable on off-target effects of gene editing. Its a hot topic in the field, so I and many others were very keen to join in and learn. Next, there was another roundtable on the developing needs of the cell and gene therapy workforce. That was also extremely interesting and gave me an opportunity to learn about what other people in other countries and institutions are doing to address the growing shortage. Some of the ideas were truly inspirational. Third, there was a talk on making cell and gene therapies available to patients in less-wealthy countries. This was interesting for me because in my previous life at GSK my colleagues and I did a lot of work trying to make sure that patients in low- and medium-income countries got access to therapies. Sadly it is still a challenge, albeit better understood, and thus the talk allowed us to have a really constructive, engaging discussion and share ideas and successes.

We are now considering not only how to mint more MScs and postdocs, but also how to engage schoolchildren in science and the cell and gene therapy field.

Beyond those three, I attended a roundtable and a talk on CAR T infrastructure. The roundtable focused on the potential for disseminating CAR T administration from a very limited number of key centers to many more smaller hospitals to treat a greater number of patients. The talk was on CAR T optimization, specifically improving the efficacy of allogeneic therapies.

Without a doubt, yes. Though I cant claim to own a crystal ball, there are some areas in which the field is already starting to see effects linking back to the conversations on headcount and workforce planning that took place at our conference. Before the conference, we had already seen a number of articles in magazines and journals hitting on this topic, and even some podcast episodes being put together. Awareness of the challenges is now higher, and efforts to tackle them are growing more coordinated. We are now considering not only how to mint more MScs and postdocs, but also how to engage schoolchildren in science and the cell and gene therapy field. In fact in the UK, this very topic has reached the level of government engagement.

Another really important area where our conversations should have a massive effect is gene editing. Many people are realizing that editing as powerful as the technology may be has its limits. Theyre realizing, too, that within developing technologies lie alternatives to gene editing, especially for more complex engineering tasks.

Were also seeing ongoing dialogue on making advanced therapies available in less wealthy countries. The solution here is not something as simple as shipping cells directly off to the worlds poorest regions. This is about starting at home, and thinking about how we manufacture these technologies at a lower cost, but with the same safety and the same efficacy. By improving the cost:benefit ratio, we can open the door to patients in lower- and medium-income countries. To pull that off, however, we need to keep these critical conversations going.

Without a doubt, we're seeing a shift in priorities towards employees quality of life.

The last hot topic Ill mention is engagement with regulators. In the last year or so it has been very difficult to engage face-to-face with regulators, such as the EMA and the FDA all thanks to a pandemic that not only closed off travel options but left many institutions, services, and corporations short-staffed. And so it was all the more special to have the opportunity to bring representatives from those regulatory bodies into ISCT 2022 for in-person discussions. Very recently, the FDA announced that from 2023 they will be moving back to business as usual, and hiring more staff for assessments and reviews. I like to think that our discussions (and the concerns we relayed) at ISCT 2022 helped sow seeds that will have influenced that decision.

Multiplex cell engineering presents serious challenges. No matter how efficient the modality, once you move into multiplexing, things rapidly grow more complex. Lets say you are editing out four different receptors to silence them. Each edit requires two double-stranded breaks. Thats four DNA breaks multiplied by four targets, giving a total of 16 breaks in the genome. Expecting them all to heal up spontaneously after disruption with congruent end-to-end joining and no translocations or other malformations would be unwise, because this is very seldom possible.

Furthermore, we should acknowledge that editing is not 100 percent effective. If you put 100 cells into the petri dish, youre not going to successfully edit all of them. Youll generally manage a rate of 9096 percent, but in some cases youll score as low as 3040 percent. As you introduce more edits, fewer cells survive the process. To mitigate that, you need to edit in steps. So you do one edit, then another, then another and at every step you have to select and expand the cells that have actually been edited. If youre doing that to T cells repeatedly pushing them into battle-readiness theyll be absolutely exhausted after just three or four edits. From there, theyll be of little to no use in the body.

Without a doubt, were seeing a shift in priorities towards employees quality of life. Instead of paying prospective staff over the odds, a number of companies are now advertising a market rate salary but then offering additional incentives tied to quality of life, such as uncapped holiday time, gym memberships, regular health checkups, coaching and development, and so on.

Much of the point here is to generate mutual feelings and engagement and purpose between the company and the employee. In some ways, it does feel like a return to older models that prioritized bonds and loyalty over employees ship-jumping self-interest and managements hire-and-fire flexibility. Were even seeing this play out in smaller companies that dont necessarily have the financial power to offer super high salaries.

These smaller companies can find themselves at a disadvantage in terms of staff loyalty, since they may lack the resources to train and develop their staff for a range of wider and more advanced roles. If you come in as a technician, youll likely stay a technician. In a larger company, that wont necessarily be the case. And thats why state-sponsored organizations, such as the UKs Cell and Gene Catapult and Canadas CCRM, are absolutely critical; they can provide essential on-the-job training to people working outside big pharma.

Apprenticeship schemes are a great option, too. Either way, on-the-job training is absolutely critical in our environment. We need it to let people grow, develop, and expand their knowledge and horizons. As companies grow in size, theyre able to offer more and more training, which then has a trickle down effect benefiting smaller companies that can continue to germinate and generate knowledge and skills. This tendency should encourage people to stay longer at their companies and develop more value. Ultimately, its about making the working environment as appealing to the worker as possible.

It depends on the company developing the therapy, and it depends on the indication and where the patients are located. For beta thalassemia or sickle cell disease, Sub-Saharan Africa is the most interesting spot. But then for certain other indications, Southeast Asia or South America will be far more interesting.

At ISCT 2022, we were fortunate enough to have speakers from across the global south. We had someone from India, someone from South Africa, and someone from South America, all there to talk about their experiences. Sitting in our comparatively very rich countries, we have a tendency to group all the low- and middle-income countries together. But, of course, what works in Chile will not necessarily work in Nigeria or Thailand. Different countries require different solutions. Understanding those specific contexts and challenges in detail is key to helping cell and gene therapy go global. The rest of the world just needs some love and cell attention.

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Sven Kili on reconvening with the ISCT - The Medicine Maker

UAB researchers show there is no longer a connection between the previously linked gene and elevated blood pressure in those of African ancestry.

UAB researchers, Vibhu Parcha, M.D. (left), and Pankaj Arora, M.D. (right), were involved in this study. Photography: Andrea MabryFor many years, the Corin gene was believed to be associated with high blood pressure in Black individuals. Now, a recent study published in the Journal of the American Heart Association involving researchers from the University of Alabama at Birmingham disputes that belief. The research team says it is a misconception that Corin gene variants are responsible for the high prevalence of hypertension in the African American community.

Although we know there is a high prevalence of high blood pressure in the African American community, our research shows that Corin gene variants are not the cause, said Pankaj Arora, M.D., a physician-scientist in UABs Division of Cardiovascular Disease. There is still much work that needs to be done to fully understand why high blood pressure and poor cardiometabolic health is disproportionately higher among Black individuals.

The Corin gene regulates the processing of heart hormones called natriuretic peptides that help regulate blood pressure. It was previously presumed that the Corin gene variants seen almost exclusively in those of African ancestry contributed to the higher prevalence of elevated blood pressure in this community. In a study involving more than 11,000 Black individuals, researchers found that being a carrier of the Corin gene variants was not associated with higher blood pressure levels and did not impact the circulating levels of the natriuretic peptides. They also found that there were no expression differences in natriuretic peptides in the heart tissue of individuals with and without the Corin gene variant.

We know that hypertension results from numerous environmental factors, pervasive systemic health inequities and multiple social determinants of health that disproportionately impact the African American community, said Vibhu Parcha, M.D., a clinical research fellow in the Division of Cardiovascular Diseases and the UAB Cardiogenomics Clinic. It is unfair to simply blame this on the Corin gene. Our findings suggest that we must look elsewhere for the root causes of the high prevalence of hypertension in this community and find ways to mitigate those causes.

Parcha says the scientific community has largely been unsuccessful in including Black individuals into genomic research, which has limited the understanding of the genetic determinants of cardiovascular health in Black individuals.

Research studies in the UAB Department of Medicine involving underrepresented communities help to advance the implementation of precision medicine an approach of using ones genetic information to guide their medical therapy. Using the latest advances in medicine, physician-scientists at the UAB Marnix E. Heersink School of Medicine are continuing to make UAB a center of excellence for precision cardiovascular medicine. At the UAB Cardiogenomics Clinic, a patients care team uses their genetic history to help develop a personalized cardiovascular treatment plan. UAB Cardiovascular Institute has been advancing the initiative for the inclusion of Black individuals in clinical research and helping improve its understanding of what drives the development offatal cardiac illness in the Black community.

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Study refutes long-held belief that the Corin gene causes hypertension in African Americans - University of Alabama at Birmingham

Akouos, Inc.

-The IND for AK-OTOF is the first to receive FDA clearance for a genetic form of hearing loss and the first for an AAV vector therapy with the potential to treat an inner ear condition

-Akouos plans to initiate a pediatric Phase 1/2 clinical trial, including children as young as two years of age in the dose-escalation phase (Part A), to evaluate AK-OTOF for the treatment of OTOF-mediated hearing loss

-Based on auditory brainstem response data from nonclinical studies, a one-time administration of AK-OTOF has the potential to deliver durable restoration of auditory function

BOSTON, Sept. 13, 2022 (GLOBE NEWSWIRE) -- Akouos, Inc. (NASDAQ: AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today announced that it has received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to initiate a Phase 1/2, first in human, pediatric clinical trial of AK-OTOF, a gene therapy intended for the treatment of patients with otoferlin gene (OTOF)-mediated hearing loss. Currently, there are no approved pharmacologic treatment options for individuals with OTOF-mediated hearing loss, a form of sensorineural hearing loss caused by mutations in the OTOF gene.

The AK-OTOF IND clearance from FDA is an important step toward achieving our mission of making healthy hearing available to all, said Manny Simons, Ph.D., M.B.A., co-founder, president, and chief executive officer of Akouos. This first in human clinical trial for AK-OTOF is groundbreaking and highlights Akouoss leadership in the field -- we expect this to be the first clinical trial for a genetic inner ear condition, the first in which an AAV gene therapy is administered to the inner ear, and the first for any inner ear condition to begin in a pediatric population.

We are excited to advance AK-OTOF into clinical development. There is a significant unmet need in OTOF-mediated hearing loss, as individuals typically have Severe to Profound sensorineural hearing loss from birth, and there are currently no approved pharmacologic options, said Jen Wellman, chief operating officer of Akouos. This clinical trial is designed not only to evaluate the safety and potential benefit of AK-OTOF for individuals with OTOF-mediated hearing loss, but also to help us demonstrate the applicability of our novel delivery approach to a broad range of inner ear conditions. We look forward to sharing what we learn from this pioneering work.

Story continues

OTOF-mediated hearing loss is a form of sensorineural hearing loss caused by mutations in the otoferlin gene, which encodes otoferlin, a protein that enables the inner hair cells of the cochlea to release neurotransmitter vesicles in response to stimulation by sound to activate auditory neurons. Individuals with OTOF-mediated hearing loss have bilateral hearing loss that is typically Severe to Profound and congenital, exhibiting absent or highly abnormal auditory brainstem response (ABR) from birth. Approximately 20,000 individuals are affected in the United States and Europe. In April 2021, FDA granted both Orphan Drug Designation and Rare Pediatric Disease Designation for AK-OTOF.

AK-OTOF is a dual adeno-associated viral (AAV) vector-based gene therapy intended to treat patients with OTOF-mediated hearing loss by delivering transgenes encodingOTOFto the inner hair cells (IHCs) of the cochlea. A one-time, unilateral intracochlear administration of AK-OTOF is intended to result in the expression of normal full-length functional otoferlin protein in the IHCs, which has the potential to lead to recovery of auditory function.

The advancement of AK-OTOF into clinical development is supported by nonclinical data demonstrating administration of AK-OTOF in Otof knockout mice results in durable expression of human otoferlin protein sufficient for sustained restoration of auditory function, as assessed by translationally relevant ABR assessments. In both mice and non-human primates, AK-OTOF was systemically and locally well tolerated, and no adverse effects were observed in clinical pathology, otic pathology, systemic histopathology, or auditory or cochlear function.

The Phase 1/2 clinical trial is designed to evaluate the safety and tolerability of escalating doses of AK-OTOF administered unilaterally to trial participants with OTOF-mediated hearing loss; it is also designed to assess efficacy through clinical measures such as ABR, which is an objective, clinically accepted endpoint. Given both the early onset of serious manifestations, as well as the need for timely intervention due to anatomical considerations and developmental considerations, eligible participants for the clinical trial will be pediatric. Based on interactions with FDA during the 30-day IND review period, the Company expects the first two participants will be as young as seven years of age, and that subsequent participants will be as young as two years of age at the time of administration.

The Company plans to provide an update on clinical trial initiation activities for AK-OTOF later this year.

About AkouosAkouos is a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for individuals living with disabling hearing loss worldwide. Leveraging its precision genetic medicine platform that incorporates a proprietary adeno-associated viral (AAV) vector library and a novel delivery approach, Akouos is focused on developing precision therapies for forms of sensorineural hearing loss. Headquartered in Boston, Akouos was founded in 2016 by leaders in the fields of neurotology, genetics, inner ear drug delivery, and AAV gene therapy.

Forward-Looking StatementsStatements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the initiation, plans, timing of our future clinical trials for AK-OTOF, and the potential benefit of AK-OTOF. The words anticipate, believe, contemplate, continue, could, estimate, expect, intend, might, may, plan, potential, predict, project, should, target, will, would, and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: our limited operating history; uncertainties inherent in the development of product candidates, including the initiation and completion of nonclinical studies and clinical trials; whether results from nonclinical studies will be predictive of results or success of clinical trials; the timing of and our ability to submit applications for, and obtain and maintain regulatory approvals for, our product candidates; our expectations regarding our regulatory strategy; our ability to fund our operating expenses and capital expenditure requirements with our cash, cash equivalents, and marketable securities; the potential advantages of our product candidates; the rate and degree of market acceptance and clinical utility of our product candidates; our estimates regarding the potential addressable patient population for our product candidates; our commercialization, marketing, and manufacturing capabilities and strategy; our ability to obtain and maintain intellectual property protection for our product candidates; our ability to identify additional products, product candidates, or technologies with significant commercial potential that are consistent with our commercial objectives; the impact of government laws and regulations and any changes in such laws and regulations; risks related to competitive programs; the potential that our internal manufacturing capabilities and/or external manufacturing supply may experience delays; the impact of the COVID-19 pandemic on our business, results of operations, and financial condition; our ability to maintain and establish collaborations or obtain additional funding; and other factors discussed in the Risk Factors included in the Companys Quarterly Report on Form 10-Q for the quarter ended June 30, 2022, filed with the Securities and Exchange Commission on August 15, 2022, and in other filings that the Company makes with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company expressly disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.

Contacts

Media:Katie Engleman, 1ABkatie@1abmedia.com

Investors:Courtney Turiano, Stern Investor RelationsCourtney.Turiano@sternir.com

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Akouos Receives FDA Clearance of its IND Application for AK-OTOF, a Gene Therapy Intended for the Treatment of OTOF-mediated Hearing Loss - Yahoo...

At 44, Janet Waterhouse should have been the picture of health; a former Division I soccer player, she taught yoga, enjoyed running, and didnt drink alcohol. Despite her healthy and active lifestyle, over a span of decades she experienced a number of unexplained symptoms.

Her symptoms continued to worsen into her 20s when she began to sporadically lose function of her hands and experience severe bouts of vertigo. Most doctors attributed her symptoms to stress and anxiety. During this time, Waterhouse was seeing a pain management specialist, who was concerned enough about her worsening symptoms to run a blood test, where he found irregularly shaped blood cells, called acanthocytes.

A series of serendipitous referrals led Waterhouse to Ali Hamedani, an assistant professor of neurology and ophthalmology in the Perelman School of Medicine. Based on her symptoms and exam, he suspected a genetic condition called chronic progressive external ophthalmoplegia (CPEO) and referred her to Laynie Dratch, a certified genetic counselor in the Penn Neurogenetics Therapy Center, for genetic testing.

In May of 2022, Dratch gave Waterhouse what she had been chasing for decades: a diagnosis. When the genetic counselor told me they found the genetic mutation they were looking for, I cried for a solid five minutes out of relief, Waterhouse says.

Waterhouses case of CPEO was found to be caused by a variation on her RRM2B gene, which affects the mitochondria in her cells. While the condition is very rare and can sometimes take years to locate and diagnose, Hamedanis hunch about the gene mutation led them right to it.

Because little is known about CPEO, treatment options are limited. Most people would be discouraged by the uncertainty, she says, but it thrills me to get to be the blueprint. I get to show people how to live with this.

Launched in March 2020, the Penn Neurogenetics Therapy Center has a team of clinicians, nurses, genetic counselors, and clinical research staff who are devoted to the care of patients with inherited neurological disorders and to participating in clinical trials of novel gene and molecular therapies.

The programs mission is twofold: first, they utilize the expertise of clinicians and researchers throughout the department of Neurology and across Penn Medicine to achieve a genetic diagnosis for as many patients like Waterhouse as possible, creating a database of eligible patients for new treatments and clinical trials. Second, they work to establish clinical trials using novel gene and molecular therapies for patients with genetically-based neurological disorders.

Our genetics counselors are some of the best in the country, and are incredibly effective at diagnosing patients and matching them with effective treatments and clinical trials, says Steven Scherer, a professor of neurology and director of the Neurogenetics Therapy Center. Now we can utilize this expertise to design tomorrows therapies.

Read more at Penn Medicine News.

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Solving medical mysteries with genetics: The Penn Neurogenetics Therapy Center | Penn Today - Penn Today

SEATTLE, Sept. 12, 2022 (GLOBE NEWSWIRE) -- Data Bridge Market Research Published Latest Global Pharmacogenetics Testing in Psychiatry/Depression Market Study by in-depth analysis of the current scenario, the Market size, demand, growth pattern, trends, and forecast. This Pharmacogenetics Testing in Psychiatry/Depression market report studies the market and the healthcare industry comprehensively by considering several aspects. The report helps in achieving sustainable growth in the market, by providing well-versed, specific, and most relevant product and market information. Inputs from various industry experts, essential for the detailed market analysis, have been employed very carefully to generate this finest market research report. The company profiles of all the top market players and brands are listed in the Pharmacogenetics Testing in Psychiatry/Depression report which puts light on their moves like product launches, product enhancements, joint ventures, mergers and acquisitions and their effect on sales, import, export, revenue and CAGR values.

Data Bridge Market Research analyzes that the global pharmacogenetics testing in psychiatry/depression market is expected to reach the value of USD 2,133.49 million by 2029, at a CAGR of 9.5% during the forecast period. Anxiety accounts for the largest type segment in the market due to the increasing depression rate among the global population. This market report also covers pricing analysis, patent analysis, and technological advancements in depth.

Access PDF Sample Report (Including Graphs, Charts & Figures) @ https://www.databridgemarketresearch.com/request-a-sample/?dbmr=global-pharmacogenetic-testing-in-psychiatry-depression-market

Market Insights:-

Pharmacogenetic testing helps medical professionals by providing information on how a person metabolizes a medication. This information can help doctors and others avoid prescribing antidepressants that could produce undesirable outcomes. Pharmacogenomics has shown promise for predicting antidepressant response and tolerability in major depressive disorder (MDD). Pharmacogenomics can improve clinical outcomes by guiding antidepressant selection and dosing. The growing biotechnology sector, and increasing health expenditure, have accelerated the demand for pharmacogenetic testing in psychiatry/depression.

The growing prevalence of cancer disease, novel technology in the treatment of depression and/or other psychiatric conditions are increasing the adoption of pharmacogenetics testing in psychiatry/depression devices and procedures, and the rising preference for non-surgical procedures are the major drivers which propelled the demand for the market in the forecast period. However, the high cost associated with the tests, stringent regulation, and lack of awareness may expect to hamper the pharmacogenetics testing in psychiatry/depression market growth in the forecast period.

Some of the major players operating in the global pharmacogenetics testing in psychiatry/depression market are

Recent Developments

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Opportunity

Advances in pharmacogenomics have introduced an increasing number of opportunities to bring personalized medicine into clinical practice for psychiatric disorders. Personalized medicine may be defined as a comprehensive, prospective approach to preventing, diagnosing, treating, and monitoring disease in ways that achieve optimal individual health care decisions. Over 100 medications now contain United States Food and Drug Administration (FDA) labelling related to potentially applicable pharmacogenomic biomarkers with technological advancements in healthcare. Also, new and advanced methods are being developed to promote pharmacogenetics testing in depression-like disorders. These tests use advanced genetic testing methods to give precise results to form a treatment regimen. The improvements in technology supporting tests improved accessibility of testing options, and the growing number of resources that help clinicians understand how to use this information when it is available are making this aspect of personalized or precision medicine a reality. Thus, providers need to become more aware of the scientific and clinical relevance of pharmacogenomic tests.

The tests also help to establish a meaningful relationship between a drug and the individual genetic makeup. This helps in deciding the drugs to be administered to the patient to treat major depressive disorders and other psychiatric conditions.

Market Dynamics:-

Drivers

Depression is a common illness worldwide, with an estimated 3.8% of the population affected, including 5.0% among adults and 5.7% among adults older than 60 years. Depression can become a serious health condition of mild to extreme severity, affecting the person to suffer greatly and can lead to suicide in the worst cases. Although over 45 antidepressants are available, suboptimal response poses a challenge and is considered a result of genetic variation, psychiatry/depression. Depending on the severity and pattern of depressive episodes over time, healthcare providers may offer psychological diagnosis such as behavioral activation, cognitive behavioral therapy, interpersonal psychotherapy, and/or antidepressant medication such as selective serotonin reuptake inhibitors (SSRIs) and tricyclic antidepressants (TCAs). Different drugs are used for this kind of mental disorder.

With the growth in the prevalence of depression, the demand for pharmacogenetics testing is also increasing as it studies the effect of genetic variants intending to furnish tailored diagnosis. The market is expected to grow in foresting period.

Pharmacogenetics test aids the medical professional in choosing the best medicine for the person because the test searches for the gene variant that may be responsible for influencing the effect of the drug.

Medicine is becoming personal, and patients gradually express interest in improved outcomes and less adverse effects with personalized medications. Personalized medicine has the potential to tailor the therapy with a high safety margin and the best response. This trend is largely driven by genome sequencing improvements.

The move toward personal healthcare means changes in the manufacturing of medicines. Manufacturers are moving from creating small molecules to the combination of small molecule and gene therapies. Sponsors focus on replacing inefficient large-scale batch production with investment in new technology and producing personalized drugs.

Critical Insights Related to the Pharmacogenetics Testing in Psychiatry/DepressionIncluded in the Report:

Restraint

Most clinicians still lack confidence in pharmacogenetic (PGx) testing and subsequent data interpretation, indicating insufficient knowledge in this field. It emphasizes the need to improve literacy among healthcare professionals regarding expertise in and understanding of pharmacogenetic (PGx) testing.

Lack of practitioners awareness about the possibilities of pharmacogenetics and poor or insufficient explanation of the test results also reduce personalization technologies for patients. In addition to developing thematic training courses at medical universities, including the educational cycles in continuing professional education systems, and free placement of information for practicing doctors are required: academic internet portals, webinars, etc. A clinical pharmacologist plays a crucial role in the implementation of pharmacogenetic testing.

The competence of a clinical pharmacologist in the field of pharmacogenetics is critical: he or she is the one who organizes the application of genotyping in clinical practice, interprets tests, and informs doctors about the possibilities of pharmacogenetics for patients with specific nosologies, that is, acts as the main link between the scientific world, the healthcare system and practicing physicians in the process of introducing pharmacogenetics.

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Challenge

The concerns regarding the efficacy and safety of products have caused most governments to develop regulatory agencies and policies to look after the development of new medical products or tests. The use of these medical products can be done after passing stringent regulatory standards, which ensure the product is safe, well studied, and has no adverse reactions.

The recent guidelines and the amendment have adequate guidance for manufacturers. International regulations such as food, drug, and administration play a major role in the new launch of medical products or tests into the market. Thus, it can be a major restraint for the market. Therefore, strict government regulation on new products and instrument approval will likely impact the market.

Segmentation: Pharmacogenetics Testing in Psychiatry/Depression Market

Global pharmacogenetics testing in psychiatry/depression market is segmented into type, test type, gene type, patient type, product, end user, and distribution channel. The growth among segments helps you analyze niche pockets of growth and strategies to approach the market and determine your core application areas and the difference in your target markets.

By Type

By Test Type

By Gene Type

By Patient Type

By Product

By End User

By Distribution Channel

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Global Pharmacogenetics Testing in Psychiatry/Depression Market Regional Analysis/Insights

The pharmacogenetics testing in psychiatry/depression market is analyzed, and market size information is provided by the type, test type, gene type, patient type, product, end user, and distribution channel.

The countries covered in pharmacogenetics testing in psychiatry/depression market report are U.S., Canada, Mexico, Germany, France, U.K., Italy, Spain, Russia, Turkey, Netherlands, Switzerland, Hungary, Austria, Lithuania, Ireland, Norway, Poland, and the rest of Europe, China, Japan, India, South Korea, Singapore, Thailand, Malaysia, Australia, Vietnam, Philippines, Indonesia and rest of Asia-Pacific, UAE, Israel, South Africa, Egypt, Kuwait and rest of the Middle East and Africa, Brazil, Argentina, Peru and, rest of South America.

In 2022, North America is dominating due to the presence of key market players in the largest consumer market with high GDP. The U.S is expected to grow due to the rise in technological advancement in pharmacogenetics testing.

North America is dominating the market. The increasing investment in R&D and increasing adoption of pharmacogenetics testing as an option for formulating treatment regimens is expected to boost the market growth. The U.S. dominates the North American region due to the strong presence of advanced technology providers such as Eurofins Scientific, Illumina, Inc., and others. The U.K. dominates Europe due to the increasing demand from emerging markets and the expansion of pharmaceutical industries. China dominates the Asia-Pacific region due to increased healthcare expenditure.

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Pharmacogenetics Testing in Psychiatry/Depression Market Will Rise at a CAGR 9.5% by 2029 and is segmented by Test Type, Gene Type, Patient Type,...

image:The research is led by professor UrFU Dmitri Alexandrov. view more

Credit: UrFU

Physicists and mathematicians from the Ural Federal University (UrFU) have created a complex mathematical model that calculates the distribution of nanoparticles (in particular, viruses) in living cells. The mathematical model helps finding how the nanoparticles cluster (merge into a single particle) inside cells, namely in cellular endosomes, which are responsible for sorting and transporting proteins and lipids. These calculations will be useful for medical purposes because, on the one hand, they show the behaviour of viruses when they enter cells and seek to replicate. And on the other hand, the model allows to accurately calculate the amount of medication needed for therapy, so that the treatment is as effective as possible and with minimal side effects. The model description and the results of calculations, the scientists publishedin the journal Crystals.

The processes in cells are extremely complex, but in simple words, the viruses use different variants to reproduce. Some of them deliver the genetic material directly to the cytoplasm. Others use the endocytosis pathway: they deliver the viral genome by releasing it from the endosomes. If viruses linger in the endosomes, the acidity increases and they die in the lysosomes, says Dmitri Alexandrov, Head of the Laboratory of Multiscale Mathematical Modeling at UrFU. So, our model has allowed to find out, first, when and which viruses "escape" from endosomes in order to survive. For example, some influenza viruses are low pH-dependent viruses; they fuse with the endosome membrane and release their genome into the cytoplasm. Secondly, we found that it is easier for viruses to survive in endosomes during clustering, when two particles merge and tend to form a single particle.

As the scientists explain, the mathematical model will also be useful in tumor targeting therapy: many cancer therapies depend on when and how nanoparticles of a drug saturate cancer cells. And the model will help to calculate this parameter.

In addition, understanding the behavior of viruses in cells is important for the development of vaccines and drugs, as well as for gene therapy, which treats diseases that conventional medicine cannot cope with. For example, various adenovirus-based vectors and lipid particles are used as a platform for gene delivery to treat the disease. But their limited ability to "slip out" of the endosomes also limits their use as deliverers.

Nanoparticles smaller than 100 nanometers are becoming increasingly important tools in the modern medicine. Its applications range from nanodiagnostics to radiation therapy for cancer. For example, pH-sensitive nanoparticles mimicking viruses are used for targeted delivery of antitumor drugs. This is how drugs are delivered from whole organs to individual cells, says Head of the Laboratory of Stochastic Transport of Nanoparticles in Living Systems (UrFU) Eugenya Makoveeva.

Reference

Endosome is a membrane intracellular organelle. It is responsible for sorting and transporting proteins and lipids; occur during the fusion and development of endocytic vesicles. In the process of maturation, the endosome goes through several stages, as a result of which it turns into a lysosome. Mature endosomes reach sizes of 300-400 nanometers.

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Scientists have created a mathematical model for the dynamics of nanoparticles and viruses in cells - EurekAlert

MENLO PARK, Calif. & DALLAS--(BUSINESS WIRE)--ReCode Therapeutics, a genetic medicines company using superior delivery to power the next wave of mRNA and gene correction therapeutics, announced today that Fierce Biotech has named it as one of 2022s Fierce 15 biotechnology companies, designating it as one of the most promising early-stage biotechnology companies in the industry.

In recent years, weve seen major advances in mRNA and gene correction therapeutics. However, their utility has been limited by current delivery systems. Thanks to the pioneering science behind ReCodes novel selective organ targeting (SORT) lipid nanoparticle (LNP) technology, we are poised to transform the paradigm for genetic medicines with the ability to precisely target and deliver any form or combination of genetic cargo to specific organs and cells, beyond the liver, said Shehnaaz Suliman, M.D., MBA, M.Phil., chief executive officer and board member, ReCode Therapeutics. It is an honor to be recognized among this distinguished group of Fierce 15 industry innovators.

ReCode is differentiated by its first-in-class modular genetic medicines delivery platform. The companys selective organ targeting (SORT) lipid nanoparticle technology (LNP) platform is the foundation for its pipeline. Pioneered by co-founder Professor Daniel J. Siegwart, Ph.D., of the University of Texas, and described by Nature as one of the Seven Technologies to Watch in 2022, ReCodes SORT LNP platform is an innovation beyond the lipid delivery system used by the mRNA COVID vaccines and novel RNA and gene correction therapeutics.

LNPs are used to package and deliver genetic cargo such as mRNA. When delivered into the blood, first-generation LNPs are primarily taken up by the liver, which limits their utility for broad therapeutic applications. ReCodes SORT LNPs are engineered with a biochemically distinct fifth lipid to help the body sort and direct the LNPs to other targeted organs such as the lung and spleen, with the ability to bypass the liver, if desired.

Beyond its highly selective targeting capability, ReCodes SORT LNP platform is further distinguished by its versatility in both mode of administration (including IV, inhaled, subcutaneous, intramuscular and intrathecal), and the diversity of genetic cargo that can be delivered (including mRNA, siRNA, DNA, gene correction components and mixed cargoes). Together, these qualities offer vast opportunities to address a wide range of unmet medical needs through a precision medicine approach that delivers the right medicine to the right organs and cells using the optimal mode of administration.

In June, ReCode announced the close of a Series B extension financing round which infused a total investment of $200M to fund the diversification of ReCodes pipeline into central nervous system, liver, and oncology indications, while continuing to advance ReCodes lead mRNA programs for primary ciliary dyskinesia and cystic fibrosis into the clinic.

The Fierce 15 celebrates the spirit of being fierce championing innovation and creativity, even in the face of intense competition. This is Fierce Biotechs 20th annual Fierce 15 selection. An internationally recognized daily report reaching a network of over 450,000 biotech and pharma industry professionals, Fierce Biotech provides subscribers with an authoritative analysis of the day's top stories. Every year Fierce Biotech evaluates hundreds of early-stage companies from around the world for its annual Fierce 15 list, which is based on a variety of factors such as the strength of its technology, partnerships, venture backers and a competitive market position.

About Fierce Biotech

Fierce Biotech is the biotech industry's daily monitor, an email newsletter and web resource providing the latest biotech news, articles, and resources related to clinical trials, drug discovery, FDA approval, FDA regulation, patent news, pharma news, biotech company news and more. More than 450,000 top biotech professionals rely on Fierce Biotech for an insider briefing on the day's top stories. Signup is free at http://www.fiercebiotech.com/signup.

About ReCode Therapeutics

ReCode Therapeutics is a genetic medicines company using superior delivery to power the next wave of mRNA and gene correction therapeutics. ReCodes selective organ targeting (SORT) lipid nanoparticle (LNP) platform is a next-generation, genetic medicines technology that enables precise delivery to target organs and cells beyond the liver. The SORT LNP platform is the foundation for ReCodes pipeline of disease-modifying mRNA and gene correction therapeutics. ReCodes lead programs are focused on primary ciliary dyskinesia and cystic fibrosis. ReCode is leveraging its SORT LNP platform and nucleic acid technologies to expand its pipeline with therapeutics that use mRNA-mediated replacement and gene correction in target organs with precision targeting of disease-relevant cells. For more information, visit http://www.recodetx.com and follow us on Twitter @ReCodeTx and on LinkedIn.

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Fierce Biotech Names ReCode Therapeutics as One of its Fierce 15 Biotech Companies of 2022 - Business Wire

NEW YORK Roche's Foundation Medicine announced on Monday that it will collaborate with Relay Therapeutics to develop FoundationOne CDx as a companion diagnostic for Relay's RLY-4008, an investigational FGFR2 inhibitor.

The drug is currently being evaluated for use in patients with FGFR2-mutated cholangiocarcinoma, or bile duct cancer, and other solid tumors, the companies said in a statement. If the therapy and FoundationOne CDx as a companion diagnostic are approved, the test would be used to identify patients with FGFR2 fusions and select rearrangements in cholangiocarcinoma who may be appropriate for treatment with the drug.

FGFR2 is a receptor tyrosine kinase that is often altered in certain cancers, and RLY-4008 is currently being evaluated in a clinical trial in patients with advanced or metastatic FGFR2-altered solid tumors, the companies said.

"FGFR2-mutated cholangiocarcinoma is an aggressive condition that's generally diagnosed in advanced stages when prognosis is poor and treatment options are limited," Don Bergstrom, president of R&D at Relay Therapeutics, said in a statement. "We're proud to partner with the leader in companion diagnostic approvals as we work to advance this potentially life-changing therapy and create access to it once approved."

Next-generation sequencing-based FoundationOne CDx is used as a companion diagnostic for a variety of drugs and detects substitutions, insertion and deletion alterations, and copy number alterations in 324 genes and select gene rearrangements, along with genomic signatures such as microsatellite instability and tumor mutational burden using DNA isolated from formalin-fixed, paraffin-embedded tumor tissue specimens.

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Foundation Medicine, Relay Therapeutics to Develop FoundationOne CDx for FGFR2 Inhibitor - GenomeWeb