Monthly Archives: June 2022

VectorY presents new pre-clinical data at the European Network for the Cure of ALS (ENCALS) – Business Wire

Posted: June 4, 2022 at 2:16 am

AMSTERDAM--(BUSINESS WIRE)--VectorY, a biotech company focusing on the development of innovative gene therapy approaches for the treatment of neurodegenerative and muscular disorders through vectorized antibodies, today announced new data at the 2022 ENCALS meeting in Edinburgh.

The poster, which was presented on 1 June in McEwan Hall, d114, was titled Reduction of oxidized phospholipids or misfolded protein aggregates by AAV-VecTabs in ALS pre-clinical models (Sogorb-Gonzalez, M. et al). The new data demonstrate the therapeutic potential and versatility of VectorYs VecTab platform technology, enabling development of secreted or intracellular antibody fragments that are AAV-vectorized and delivered to neurons and/or astrocytes, in vitro and in vivo.

TAR-DNA binding protein-43 (TDP-43) is necessary for the correct processing and transport of multiple mRNAs that are essential for neuronal survival. Cytoplasmic mis-localized, misfolded or aggregated TDP-43 has been implicated in the pathogenesis of >97% of ALS patients. VectorY has confirmed that iPS-derived ALS motor neurons exhibit TDP-43 pathology. The Company has developed a library of single-chain variable fragments (scFv), named VecTabs, which are designed to exclusively bind to the misfolded, toxic TDP-43, while leaving the native TDP-43 functional. Todays new data show that TDP-43-targeting intracellular VecTabs can effectively clear (large) TDP-43 aggregates from U2OS cells. In addition, iPS-derived ALS motor neurons were efficiently transduced with AAV to express TDP-43 VecTabs in a dose-dependent manner.

TDP-43 pathology and mitochondrial dysfunction are closely linked to formation of oxidized phospholipid (oxPL) species, which are highly toxic to motor neurons. Recent in vivo and ex vivo data indicate a pivotal role for oxPL in axonal damage and motor neuron death in ALS.

VectorY has developed AAV-expressed secreted VecTabs that specifically bind and neutralize oxPL, thereby protecting iPS-derived motor neurons from oxPL-mediated cell death. The biodistribution and expression of VecTabs in the central nervous system were investigated in mice and pigs. The results presented today demonstrated transduction of the target spinal cord and cortical motor neurons and expression of the therapeutic scFv antibodies. Multiple humanized and optimized constructs were generated, leading to the selection of VTx-001 and VTx-002 for further development in ALS.

For more information or to meet the VectorY team at ENCALS, please contact info@vectorytx.com.

Notes to Editors

About VectorY

VectorY combines the therapeutic potential of antibodies and gene therapy to develop long-lasting therapeutic solutions for neurodegenerative and -muscular diseases with high unmet medical need. Founded in October 2020 and based in the Amsterdam Science Park, VectorY is a fully integrated gene therapy company focused on the development of innovative therapeutics based on a novel AAV gene therapy platform, antibody-based targeted protein degradation technologies and proprietary manufacturing technology. While focusing initially on neurodegenerative and -muscular diseases, VectorY's synergistic technologies may be applied across a wide range of indications. VectorYs manufacturing capabilities will include a state-of-the-art multi-product GMP facility in the Netherlands.

For more information, see http://www.vectorytx.com.

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VectorY presents new pre-clinical data at the European Network for the Cure of ALS (ENCALS) - Business Wire

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BDO’s BioProcess Technology Group to Present at the 2022 BIO International Convention – Business Wire

Posted: June 4, 2022 at 2:16 am

BOSTON--(BUSINESS WIRE)--BDOs BioProcess Technology Group (BPTG), the world leader in providing strategic, technical, regulatory, and business consulting services to the biopharmaceutical industry, will be presenting at the upcoming 2022 BIO International Convention, June 13-16, 2022 in San Diego. The annual convention, hosted by Biotechnology Innovation Organization (BIO), is the worlds largest industry gathering and has returned live and in person for the first time since the start of the COVID-19 pandemic.

Patti Seymour, managing director in BDOs BioProcess Technology Group, will chair the panel about cell and gene therapy development, on Tuesday, June 14, from 12:00 PM to 12:45 PM at the Emerging Therapies Track within the BPI Theater. Her discussion will focus on factors impacting the decision to develop one therapy type over another.

Nick Vrolijk, Ph.D., managing director in BDOs BioProcess Technology Group, will chair a panel about business and manufacturing strategies for therapeutic cell and gene therapy modalities, on Wednesday, June 15, at the Emerging Therapies Track, within the BPI Theater from 12:00 PM to 12:45 PM. The roundtable discussion will cover new technologies to compress timelines, increase capacity and reduce costs for cell and gene therapy development.

BPTG has presented at this conference in 2019, and for several years prior, before being acquired by BDO. The four-day convention presents an opportunity for BPTG to demonstrate its expertise in bioprocess manufacturing to senior biotech executives, business development leaders and investors.

BDOs BioProcess Technology Group is proud to exhibit and speak alongside biotech and pharmaceutical organizations showcasing groundbreaking development at this years BIO Convention, said Patti Seymour, Managing Director of BDOs BioProcess Technology Group. These organizations are on the frontiers of medical breakthroughs, and were excited to show how we can partner to help them solve their greatest challenges including CMC product development, quality, regulatory and compliance, and manufacturing strategy and operations.

To learn more about BDOs Life Sciences practice and our services, connect with BPTG professionals at booth 1414 and Biodefense professionals at booth 5113.

Visit our website for a complete overview of BDOs presence at BIO 2022.

About BDOs BioProcess Technology Group

BPTG is the recognized worldwide leader in biologics CMC consulting, providing a full range of technical, regulatory, and strategic assistance to pharmaceutical and biotechnology companies in the development and commercialization of biopharmaceutical products. The company works with clients globally to find and apply solutions to the challenges of biopharmaceutical product development from clone to commercial. BPTG helps develop manufacturing processes and strategies that enhance the overall value of client companies, de-risk biopharmaceutical development, and accelerate the advancement of new products from clone to clinic. BPTG also helps investors, service providers, and companies make informed decisions by providing technical due diligence services and business evaluations of new products as well as technologies for product discovery, development, or commercialization. Learn more about BPTG and our services here. Follow us on Twitter.

About BDO USA

BDO is the brand name for BDO USA, LLP, a U.S. professional services firm providing assurance, tax, and advisory services to a wide range of publicly traded and privately held companies. For more than 100 years, BDO has provided quality service through the active involvement of skilled and committed professionals. The firm serves clients through more than 65 offices and over 740 independent alliance firm locations nationwide. As an independent Member Firm of BDO International Limited, BDO serves multi-national clients through a global network of more than 91,000 people working out of over 1,600 offices across 167 countries.

BDO USA, LLP, a Delaware limited liability partnership, is the U.S. member of BDO International Limited, a UK company limited by guarantee, and forms part of the international BDO network of independent member firms. BDO is the brand name for the BDO network and for each of the BDO Member Firms. For more information please visit: http://www.bdo.com and follow us on LinkedIn.

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BDO's BioProcess Technology Group to Present at the 2022 BIO International Convention - Business Wire

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Lysogene Provides Clinical Update on AAVance Phase 2/3 Clinical Trial with Gene Therapy LYS-SAF302 for the Treatment of MPS IIIA at the 25th Annual…

Posted: June 4, 2022 at 2:16 am

PARIS--(BUSINESS WIRE)--Regulatory News:

Lysogene (Paris:LYS) (FR0013233475 LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, provided updated clinical data from the ongoing AAVance phase 2/3 clinical trial with the investigational gene therapy LYS-SAF302 for the treatment of MPS IIIA (NCT03612869) during the 25th Annual Meeting of the ASGCT (American Society for Gene & Cell Therapy) in Washington, DC.

The preliminary results from the AAVance study evaluating LYS-SAF302 in patients with Sanfilippo syndrome type A (MPS IIIA) who have been followed for at least two years demonstrated improvement, stabilization, or slowing down of decline in cognitive developmental age (DA) in about half of the patients. Notably, in all 6 patients enrolled under the age of 30 months, a persistent increase or stabilization of the cognitive, language and motor domains of the BSID-III was observed in the 24 months period post-treatment. Two (2) of these 6 patients, including 1 patient over 4 years of age, have already exceeded by several months the highest cognitive DA (35 months) observed in the natural history cohort. The 2 patients reached 41 and 42 months of cognitive DA in the 24 months period post-treatment with an increase of 25 and 17 months compared to their baseline, respectively.

These results, which suggest that younger patients are more responsive to the gene therapy treatment, need to be corroborated and confirmed by the final statistical analyses. They will also include secondary behavioral and imaging endpoints as well as data from the observational study in children treated with LYS-SAF302, using video and parent interviewing (also called the Patient Reported Outcome Videos [PROVide] study). Full results will be available in Q3 2022.

Considering these promising efficacy signals and the observed general trend towards stabilization and/or decrease of the total white matter lesional volume at injection sites, from 12 months post-treatment onwards, with no apparent clinically significant consequences, the company will plan further discussions with regulatory authorities to determine next steps.

Karen Aiach, Founder and CEO of Lysogene said: "We are pleased to share these promising efficacy data in patients treated with LYS-SAF302, after a 2-year follow-up period. The data show encouraging signals of efficacy on cognitive, language and motor development in young patients. We look forward to confirming and supporting these observations by analyzing all the data collected since the beginning of the study, including secondary behavioral and imaging endpoints and real-life behaviors assessed through the video study. Following this full analysis expected in Q3 this year, we will have all the necessary elements to discuss the path forward with the regulatory authorities. Karen Aiach added: "These promising data will support our effort to raise funds, which remains Lysogenes top priority. In a context of challenging market conditions for innovative life sciences companies, Lysogene is reviewing on an ongoing basis all options available to extend its cash runway.

AAVance is an open-label single-arm multicenter trial aimed at evaluating the effectiveness of a one-time intracerebral delivery of a recombinant adeno-associated virus vector rh.10 carrying the N-sulfoglucosamine sulfohydrolase (SGSH) gene (LYS-SAF302, olenasufligene relduparvovec) in children with MPS IIIA. MPS IIIA is caused by mutations in the SGSH gene, which produces an enzyme involved in the catabolism of heparan sulfate. LYS-SAF302 is intended to deliver a functional copy of the SGSH gene and allow the brain to secrete the missing enzyme.

About Lysogene

Lysogene is a gene therapy Company focused on the treatment of orphan diseases of the central nervous system (CNS). The Company has built a unique capability to enable delivery of gene therapies to the CNS to treat lysosomal diseases and other disorders of the CNS. A phase 2/3 clinical trial in MPS IIIA is ongoing. An adaptive clinical trial in GM1 gangliosidosis is also ongoing. Lysogene is also developing an innovative AAV gene therapy approach for the treatment of Fragile X syndrome, a genetic disease related to autism. The Company also entered into an exclusive worldwide license agreement with Yeda, the commercial arm of the Weizmann Institute of Science, for a novel gene therapy candidate for neuronopathic Gaucher disease and Parkinson disease with GBA1 mutations. http://www.lysogene.com.

Forward Looking Statement

This press release may contain certain forward-looking statements, especially on the Companys progress of its clinical trials and cash runway. Although the Company believes its expectations are based on reasonable assumptions, all statements other than statements of historical fact included in this press release about future events are subject to (i) change without notice, (ii) factors beyond the Companys control, (iii) clinical trial results, (iv) increased manufacturing costs, (v) potential claims on its products. These statements may include, without limitation, any statements preceded by, followed by or including words such as target, believe, expect, aim, intend, may, anticipate, estimate, plan, objective, project, will, can have, likely, should, would, could and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Companys control that could cause the Companys actual results, performance or achievements to be materially different from the expected results, performance or achievements expressed or implied by such forward-looking statements. A further list and description of these risks, uncertainties and other risks can be found in the Companys regulatory filings with the French Autorit des Marchs Financiers, including in the 2021 universal registration document, registered with the French Markets Authorities on April 19, 2022, and future filings and reports by the Company. Furthermore, these forward-looking statements are only as of the date of this press release. Readers are cautioned not to place undue reliance on these forward-looking statements. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future. If the Company updates one or more forward-looking statements, no inference should be drawn that it will or will not make additional updates with respect to those or other forward-looking statements.

This press release has been prepared in both French and English. In the event of any differences between the two texts, the French language version shall supersede.

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Lysogene Provides Clinical Update on AAVance Phase 2/3 Clinical Trial with Gene Therapy LYS-SAF302 for the Treatment of MPS IIIA at the 25th Annual...

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Once a Taboo Topic, Menopause Is (Finally) Having Its Wellness Moment – Vogue

Posted: June 4, 2022 at 2:13 am

This past spring, a small group of women gathered at the Beaverbrook estate in Surrey, England, a 470-acre country idyll outside London that once received guests including Elizabeth Taylor and Ian Fleming. The sprawling property, which has become a destination for wellness programs featuring everything from lectures on traditional Chinese massage to Wim Hofs cold plunges, had lured attendees for a womens health retreat. The topics included abdominal massage therapy and guided talks on depleted estrogen. This is the first time there have been so many of us, gut-health expert Amanda Porter relayed to the group, adding the staggering statistic that nearly 1 billion women are projected to experience the onset of symptoms including hot flashes and brain fog by 2025. They are the many, the confused, the perimenopausal.

Think of it like puberty, says Jen Gunter, MD, a Canadian American gynecologist and the author of The Menopause Manifesto. There isnt really a hard start date, and you dont know that youre in it until youre really in it. She goes on to describe an often undiagnosed panoply of symptoms associated with perimenopause, which precedes menopause (when youve gone 12 consecutive months without menstruating) and can start as early as our mid-30s: cycle irregularities, increased anxiety and depression, insomnia, night sweats, weight gain, a decimated sex drive. A lack of visibility doesnt help. When I turned 43, feeling wholly unequipped for my own experience with mood swings and spontaneously heavy periodsand with no guidance from my doctorI found myself googling flash periods after an episode of And Just Like That (the one where Charlotte memorably bleeds through a white jumpsuit at a charity paint party). There was just this lack of information and support, agrees the actor Naomi Watts when we spoke about the topic earlier this year. Id had enough. This September, Watts will join an increasingly vocal group of entrepreneurs, celebrities, and medical providers determined to change that dynamic when she launches a new perimenopause- and menopause-focused wellness brand with Bay Area biotech company Amyris.

Its true that hormonestheir unpredictability, and their ripple effect on how we look and feelare increasingly part of the beauty conversation. The first part of perimenopause is typically characterized by estrogen levels that fluctuate, but are still pretty high, and decreasing progesterone levels. And then the second part has to do with very rapidly declining estrogen, explains Anna Barbieri, MD, an ob-gyn at New Yorks Mount Sinai Hospital. Hormone replacement therapy (HRT), in which synthetic versions of these hormones can bolster dwindling supplies, remains a tried-and-true course of treatment. Every single one of us whos entering this transition or going through it should know about the option of hormone therapy and its risks and benefits, says Barbieri, who is also the founding physician of Elektra Health, a digital womens health platform offering virtual one-on-one menopause care with accredited providers and accountability counselors known as menopause doulas. But now there are also products such as Kindras hot flash and night sweatmitigating Cool Down Mist; Wiles Drinking Your Feelings, a stress-reducing adaptogen powder that can be mixed into your daily matcha; and the skin-calming Meditation Gel Cream from Knours, an entire skin-care range tailored to fluctuating cycles. Veracity, a six-piece skin-care line formulated to support tangential issues, including excess oil production and thinning skin, recently inspired a Hormone Balancing Facial at The Well, Manhattans buzzy destination for mindful beauty. A 60-minute treatment left my skin far less inflamed than usual, and my soul soothed.

Despite an uptick in home diagnostic tests from Thriva and Everlywell, there is no clinically proven way to test for perimenopause. Somebody who tells you we can just measure your estrogen and progesterone is bullshitting you, says Alicia Jackson, PhD, the founder of Evernow, a telehealth company that aims to provide patients with 24/7 online perimenopause support as well as virtual hormone therapy plans. Thats because our hormone levels during this transition vary wildly from day to day, so testing them to get a quantifiable diagnosis isnt reliable. Theres no established value for them, agrees Barbieri. What hormone testing can help with, she says, is to rule out other conditions (like thyroid abnormalities, for instance) that can often pose as perimenopause. Somi Javaid, MD, a Cincinnati-based ob-gyn, uses hormone tests at HerMD, the medical practice she runs in Ohio and Kentucky, for a different reason: If women see that their FSH [follicle-stimulating hormone], which is responsible for controlling our menstrual cycles, is going up and their estrogen and testosterone are going down, I may not be able to tell them theyre perimenopausal, but it helps them understand that somethings going on, says Javaid, adding that this kind of validation is extremely important: So many women who complain about perimenopause symptoms are being dismissed.

According to a recent report published in The New York Times, female patientsand particularly women of colorare far more likely than men to experience a kind of medical gaslighting (in which legitimate symptoms are invalidated by doctors); when it comes to perimenopause, that brush-off may be even more widespread. Stacy London experienced this firsthand. The longtime fashion stylists hormonal transition at age 46 had a number of typical markers. I started to feel anxiety all the time, my skin got worse, my joints felt sore, I had insomnia and terrible brain fog, says London. I thought my brain was short-circuiting, because I couldnt remember words. Londons doctor recognized her symptoms but provided little recourse for treatment. She said, Its just menopause, youll get through it, London recalls. The experience compelled her to join State Of, a skin- and body-care line that she became the CEO of in 2021, which aims to give the menopause transition a makeover, one cheerily packaged tube of joint cream at a time.

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Caitlyn Jenner can still hit golf ball nearly 300 yards after transitioning to female – Washington Times

Posted: June 4, 2022 at 2:13 am

Caitlyn Jenner is 72, but if she ever decides to compete on the womens golf tour, watch out.

The transgender celebrity, who won the gold medal in the 1976 Olympic decathlon as Bruce Jenner, said Wednesday she can still hit a golf ball nearly 300 yards, seven years after undergoing a gender transition that included years of hormone therapy.

Ive been on for seven years of full transition, been on hormone replacement, and I can still hit the golf ball 280, 290 yards. Theres a lot left over, said Ms. Jenner on Fox News.

The average driving distance on the LPGA tour is about 250 yards. The longest driver on the tour is Emily Kristine Pedersen of Denmark, who averages 282 yards, according to the LPGA. On the mens tour, according to the PGA, the average drive for the 2021 season was 295.3 yards.

Ms. Jenners comment came in a discussion about former University of Pennsylvania swimmer Lia Thomas, who became in March the first male-born athlete to win an NCAA Division I womens title with a victory in the 500-yard freestyle.

Ive got a lot left over, and I think Lia Thomas obviously has a lot left over, said Ms. Jenner.

The debate over male-born athletes in womens sports reemerged Tuesday after Thomas said in interviews that transgender competitors are no threat to womens sports.

Im not a medical expert, but theres a lot of variation among cis female athletes, Ms. Thomas told ABC News. There are cis women who are very tall and very muscular and have more testosterone than another cis woman, and should that then also disqualify them?

Ms. Jenner has long argued, with medical support, that trans women who have undergone puberty have unfair physical advantages over their female competition.

She pinned the blame on the NCAA, which permitted Ms. Thomas to compete on the womens team in the 2021-22 season after undergoing more than a year of testosterone suppression.

Ms. Thomas had swam previously on the Penn mens team for three years, without coming close to winning any national titles.

Right now were living in this woke world that allows her to compete and win medals against biological women, Ms. Jenner said. Lia Thomas played by the rules, I have no problem with her, but what I have been urging from the beginning is that the NCAA change their policies to protect women in sports.

In January, the NCAA announced it would defer to national sports governing bodies on transgender competitors but then backtracked for the 2021-22 championships, saying it would be unfair to change the rules on athletes mid-season.

When you transition, you have to have some integrity, and I think in some cases that may be lacking here, Ms. Jenner said. But she [Thomas] did play by the rules.

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Clarus Therapeutics Holdings, Inc. (NASDAQ:CRXT) Receives $6.88 Consensus PT from Analysts – Defense World

Posted: June 4, 2022 at 2:13 am

Shares of Clarus Therapeutics Holdings, Inc. (NASDAQ:CRXT Get Rating) have been assigned an average recommendation of Buy from the six ratings firms that are presently covering the company, MarketBeat Ratings reports. Four analysts have rated the stock with a buy recommendation. The average 1-year price objective among analysts that have covered the stock in the last year is $6.88.

A number of brokerages have issued reports on CRXT. Maxim Group decreased their price objective on Clarus Therapeutics to $1.50 in a research report on Thursday. Zacks Investment Research raised Clarus Therapeutics from a hold rating to a buy rating and set a $1.75 target price on the stock in a research note on Monday, April 4th. Needham & Company LLC dropped their price objective on Clarus Therapeutics to $3.00 and set a buy rating on the stock in a research note on Tuesday, May 17th. Finally, Truist Financial dropped their target price on Clarus Therapeutics from $7.00 to $3.00 and set a na rating on the stock in a research report on Tuesday, May 17th.

A number of hedge funds have recently modified their holdings of CRXT. Verition Fund Management LLC acquired a new stake in Clarus Therapeutics during the 3rd quarter worth about $160,000. UBS Group AG acquired a new stake in Clarus Therapeutics during the 3rd quarter worth about $206,000. Telemetry Investments L.L.C. acquired a new stake in Clarus Therapeutics during the 3rd quarter worth about $240,000. Powell Investment Advisors LLC grew its position in Clarus Therapeutics by 48.8% during the 4th quarter. Powell Investment Advisors LLC now owns 64,000 shares of the companys stock worth $156,000 after purchasing an additional 21,000 shares during the period. Finally, Citadel Advisors LLC acquired a new stake in Clarus Therapeutics during the 4th quarter worth about $31,000. Institutional investors own 19.50% of the companys stock.

Clarus Therapeutics (NASDAQ:CRXT Get Rating) last posted its earnings results on Monday, May 16th. The company reported ($0.61) EPS for the quarter. The company had revenue of $4.01 million during the quarter. On average, equities research analysts anticipate that Clarus Therapeutics will post -1.22 EPS for the current year.

Clarus Therapeutics Company Profile (Get Rating)

Clarus Therapeutics Holdings, Inc, a pharmaceutical company, focuses on the development and commercialization of oral testosterone replacement therapy in the United States. It offers JATENZO, a soft gel oral formulation of testosterone undecanoate for treating hypogonadal men. The company has a licensing agreement with HavaH Therapeutics for product to treat androgen therapies for inflammatory breast disease and certain forms of breast cancer; and license agreement with The Royal Institution for the Advancement of Learning/McGill University to develop and commercialize McGill's proprietary technology designed to treat conditions associated with CoQ10 deficiencies in humans.

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Clarus Therapeutics Holdings, Inc. (NASDAQ:CRXT) Receives $6.88 Consensus PT from Analysts - Defense World

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Juvenile Macular Degeneration Treatment Market Set to Grow According to Forecasts 2020 2030 Industrial IT – Industrial IT

Posted: June 4, 2022 at 2:13 am

Juvenile Macular Degeneration Treatment: Introduction

Juvenile macular dystrophy, also known as juvenile macular degeneration, refers collectively to a group of rare and genetic inherited eye disorders that can affect children and young adults. Juvenile macular dystrophy involves the deterioration of the eyes macula; however, it is different from age-related macular degeneration (AMD).

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AMD occurs as part of the bodys natural aging process and affects older adults; however, juvenile macular dystrophy is an inherited condition, which primarily targets the vision of children, teens, and young adults.

Key Drivers and Restraints of Global Juvenile Macular Degeneration Treatment Market

Increase in R&D activities, rise in number of patients suffering from diseases, and rapid expansion of health care and biopharmaceutical industries in developed and developing countries are anticipated to propel the global advancements in therapies in the juvenile macular degeneration treatment market during the forecast period. In June 2018, Lin BioScience granted orphan drug designation to LBS-008, a first-in-class oral therapy, for the treatment of Stargardt Disease by the European Medicines Agency.

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Juvenile macular degeneration is currently an untreatable inherited condition that causes permanent vision loss in children during childhood and adolescence. Stargardt disease is the most common form of juvenile macular degeneration that affects approximately 37,000 individuals in the U.S. and 66,000 in the EU. Currently, there is no approved treatment for Stargardt patients. The disease is caused due accelerated formation and accumulation of toxic vitamin A dimers in the retina caused due mutation in the ABCA4 gene, which cause progressive retinal cell death. Consequently, this process eventually leads to legal blindness with only peripheral vision remaining.

Increase in the number of eye care centers has been recorded, since primary eye care is presently taken care of in optical shops. New optical shops have the latest technological devices that improve user friendliness. Rise in the number of optical shops, eye care centers, and hospitals has increased the availability of treatment.

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In terms of end-user, the global juvenile macular degeneration treatment market can be categorized into hospitals, ophthalmic clinics, and ambulatory surgical centers. The hospitals segment accounted for a major share of the global macular degeneration treatment market. The segment is likely to maintain its leading position during the forecast period. A large population prefers to visit a hospital first rather than an ophthalmic clinic for treatment, as the cost of therapy at a hospital is lower. An ambulatory surgical center is a relatively less preferred option for the treatment of AMD.

Asia Pacific to Lead Global Juvenile Macular Degeneration Treatment Market

In terms of geography, the global juvenile macular degeneration treatment market can be segmented into five key regions: North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. North America and Europe are expected to hold major share of the global juvenile macular degeneration treatment market during the forecast period, owing to developed health care infrastructure and large patient population along with increasing technological development in these regions.

Increase in government intervention in emerging markets has led to a rise in awareness about diseases and improvement in health care infrastructure. Additionally, socioeconomic development has been observed in emerging economies such as Asia, Africa, and Latin America. These factors contribute to the overall increase in spending capacity of the population. Moreover, an increase in the foreign direct investment has been observed in these countries. This has led to various international players who are presently entering into these markets.

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Key Players Operating in Global Juvenile Macular Degeneration Treatment Market

Manufacturers in the global juvenile macular degeneration treatment market are increasingly investing in research & development of new and innovative drugs such as Alkeus Pharmas lead compound, ALK-001; Katairo GmbH lead product, Remofuscin; Gene Therapy to Activate ABCA4 for Stargardt Disease and Recombinant Enzyme for Age Related Macular Degeneration and Stargardt Disease. These players are also focused on offering highly efficient and patient compliant products.

Leading companies operating in the global juvenile macular degeneration treatment market are:

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ClinVar Miner

Posted: June 4, 2022 at 2:12 am

The content on this website is current as of 28 February 2022. On that date, ClinVar had 1,943,225 submissions on 1,194,065 variants. When referencing data from this website, please cite the 2018 Human Mutation article "ClinVar Miner: Demonstrating utility of a Webbased tool for viewing and filtering ClinVar data" .

ClinVar Miner was developed by Alex Henrie in the Eilbeck Lab with support from the NHGRI's ClinGen Resources (grants U41 HG006834-01A1 to Heidi Rehm and U01 HG007437-01 to Jonathan Berg). Use of the NCBI logo represents use of NCBI data but does not constitute an approval or endorsement of this application. Source code is available on GitHub under the GNU General Public License.

The information on this website is not intended for direct diagnostic use or medical decision-making without review by a genetics professional. Individuals should not change their health behavior solely on the basis of information contained on this website. Neither the University of Utah nor the National Institutes of Health independently verfies the submitted information. If you have questions about the information contained on this website, please see a health care professional.

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ClinVar Miner

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Genetics and hypertension: What is the link? – Medical News Today

Posted: June 4, 2022 at 2:12 am

Hypertension, or high blood pressure, means that the force of blood against the artery walls is higher than recommended levels. People with a family history of hypertension are more likely to develop the condition.

In adults, typical blood pressure measures 120/80 millimeters of mercury. Almost half of all adults in the United States have high blood pressure, which puts them at risk of cardiovascular disease, including stroke and heart attack.

Certain factors increase an individuals risk of developing hypertension, including age, weight, diet, and genetics.

Hypertension is a widespread problem affecting 47% of adults in the U.S. a figure that amounts to 116 million people. The condition affects 50% of males and 44% of females. However, only 1 in 4 have their hypertension under control. In 2019, the condition caused or contributed to more than 500,000 deaths.

Keep reading to learn more about hypertension, including the role genetics play in its development and other potential risk factors.

If an individual has family members with hypertension, they have an increased risk of developing the condition.

Hypertension can run in families because of shared genes, environments, and lifestyle habits. The risk increases even more if an individual with a genetic link to hypertension also engages in unhealthy lifestyle choices.

According to some research, when biological parents and grandparents have hypertension, there is a risk of the same condition occurring in their children and grandchildren. Specifically, the biggest risk is when individuals in a family develop hypertension before the age of 55 years. And this is independent of lifestyle factors, such as physical activity, alcohol intake, and a diet high in salt.

Research in twins and families has suggested that up to 3050% of the variance in blood pressure readings could be due to family history. It seems that genes play a role in hypertension, and family members can pass these genetic traits from one generation to the next.

However, genes are only part of the reason for heredity hypertension. Another factor is that people who live in common environments may eat the same non-nutritious diet or share habits such as smoking or drinking excessive alcohol. Along with genetics, these factors increase the likelihood of developing hypertension.

If an individual has a family history of hypertension, they should understand the risk factors for the condition and reduce them wherever possible.

Doctors refer to risk factors that someone can change as modifiable. An individual can work with their doctor to lower these risk factors as much as possible. This could involve maintaining a moderate weight, eating a nutritious diet, quitting cigarette smoking if applicable, and drinking less alcohol. Research has found that adhering to a healthy lifestyle has associations with lower blood pressure, regardless of underlying genetic risk.

Besides taking these steps, a doctor may suggest regular blood pressure monitoring so they can assess and address any changes in blood pressure over time.

Hypertension can occur in people for various reasons. It is important to note that in about 95% of cases, the cause of the condition is unknown.

Health experts do not fully understand what causes primary hypertension, which they also refer to as essential hypertension. In this type, people can develop high blood pressure over time. Additionally, there is no underlying cause for this increase. In contrast, secondary hypertension can result from other disorders that increase blood pressure.

Essential hypertension is complex many environmental and genetic factors contribute to its development. There are many genetic variations in humans that have links to essential hypertension. Examples might include genes that play a role in regulating the following:

This plays a role in regulating blood pressure in the body. Scientists believe that genetic changes in a person might impair their bodys ability to control blood pressure, and hypertension can occur.

Any changes to the genes that contribute to the typical functioning of the lining can lead to a difference in blood vessel structure and impair its abilities. Blood vessels might become narrower, which can result in high blood pressure.

Environmental factors can also contribute to the development of hypertension in some people.

Learn more about the causes of secondary hypertension.

Besides a family history of hypertension, various risk factors can increase an individuals likelihood of developing the condition.

The lifestyle risk factors for hypertension include:

Other risk factors for hypertension include:

A doctor will likely recommend lifestyle changes and medications if they diagnose someone with hypertension.

Lifestyle changes include:

An individual may also benefit from antihypertensive medications. They may use a single medication or combine two or more to control their blood pressure.

Depending on the individuals overall medical health, a doctor may prescribe one or more of the following:

Learn more about blood pressure medications.

Hypertension, or high blood pressure, is a common condition that can lead to serious health complications without treatment.

Experts believe there is a link between a family history of hypertension and the condition occurring. However, many factors contribute to the risk, including lifestyle, age, race or ethnicity, and other health conditions.

Making healthy lifestyle choices, such as maintaining a moderate weight, eating a nutritious diet, and getting regular exercise, can help reduce the risk of developing hypertension. If lifestyle changes are not enough to control blood pressure, a doctor may prescribe medication.

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Genetics and hypertension: What is the link? - Medical News Today

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IVF: heres how genetics may be affecting its success new insights – The Conversation Indonesia

Posted: June 4, 2022 at 2:12 am

It has been almost 44 years years since the first in vitro fertilisation (IVF) procedure was successfully performed in 1978 in Lancashire, England. Since then, more than 8 million babies have been born worldwide to assisted reproductive technologies, such as IVF.

But despite its increasing use, the success rate of IVF still remains relatively low, at around 30%. There may be a number of reasons for this. In our recent paper, we argue that this low rate is partially due to the many unfavourable genetic changes that we carry in our DNA.

Genetic changes happen when mutations in our genes replace, insert or delete sections of DNA. More of these mutations are occurring now in humans because were having babies at a later age. As we get older, more mutations are likely to accumulate meaning older parents are more likely to pass on genetic mutations to their children than younger parents. Mutations may also be caused by environmental factors (such as ultraviolet radiation in sunlight), or lifestyle choices (for example, smoking).

All of the genetic changes we inherit or develop throughout our lifetime constitute whats known as our genetic load. This genetic load can impact our ability to reproduce. And as our study suggests, this may also affect our ability to reproduce via methods such as IVF.

Genetic mutations make evolution possible. They provide the new material for natural selection that allows species to adapt and evolve. While most of these mutations have no effect, some are slightly harmful. Such harmful mutations may cause diabetes or breast cancer, for example or they may disrupt the healthy development of an embryo.

Human DNA carries more than 1,000 harmful mutations, most of which happened many generations ago. Yet, even though they are harmful, they have not (yet) been removed, because natural selection is a very slow process.

In addition to the large number of old mutations, new mutations also enter the population every generation. On average, every person acquires approximately 70 new mutations during their lifetime. But since some of these mutations are harmful, they need to be removed by natural selection, so that they arent passed on to future offspring. One of the most important times this happens is during natural conception.

When a child is conceived naturally, the body has many mechanisms in place to remove some of these harmful mutations.

For example, the female reproductive system is designed in such a way that only the fittest sperm cells can reach the egg for fertilisation. Although evidence is scarce, animal studies suggest that the sperm that reach the fertilisation site have a better DNA quality and potentially fewer mutations.

Mature eggs also undergo a sort of quality check during fertilisation. This too helps purge some of the genetic load. The implantation stage (where a fertilised embryo implants itself in the mothers womb) is also important, as many embryos with severe genetic abnormalities tend to be lost naturally during pregnancies.

However, IVF bypasses some of these natural mechanisms. During IVF, multiple eggs are harvested from the womans ovaries and fertilised with sperm in a laboratory. After they have been fertilised, the embryos are then returned to the womb. This reduces the opportunity for natural selection, which may therefore make IVF less efficient in reducing the genetic load. This could potentially increase the likelihood that harmful variants of genes may be passed onto the next generation.

So, the genetic load has two big implications for human reproduction. First, the genetic load of parents affects their ability to successfully reproduce. This is true both for natural conception, as well as for IVF. Second, by relaxing natural selection, IVF may let more mutations slip through the net. As such, it could slowly increase our genetic load in future generation. But there may be a solution.

Fertility rates have suffered an unprecedented decline in recent decades. In fact, sperm count has fallen by about 50 to 60% between 1973 and 2011. Its unclear why this is, but if this trend continues it could mean more people turn to IVF to conceive.

Yet we still know surprisingly little about human reproduction and the selective processes operating during natural conception. We must understand natural conception first if we want to improve assisted reproduction methods, including IVF. But recent technological advances in assisted reproductive technologies mean that we may soon be better able to counteract some of the genetic load in humans. For example selection at sperm level in the IVF process has been shown to improve the offspring fitness in animal models. In particular, selection of longer-lived sperm in zebrafish results in healthier offspring that live longer.

Advances in genomic technologies also have the potential to affect human evolution. Already, genomic data is effectively being used in clinical care, and the genomic bases of thousands of human diseases are now known. Furthermore, changes to our environment and our lifestyle are affecting the genetic load and human health. Most often, these changes have a negative effect, which makes these technological advances ever more important. As new advances are made, it will also be important to consider the potential consequences of using assisted reproductive technologies if these become the norm.

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IVF: heres how genetics may be affecting its success new insights - The Conversation Indonesia

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