STIs Reached Record Highs, Then COVID-19 Hit. Test Makers Now Ponder What’s Next – GenomeWeb

NEW YORK As the COVID-19 pandemic pushes into a third year, public health experts and PCR-based assay makers are eager to know whether an increase in sexually transmitted infections is on the horizon.

Rates of STIs in the US had been rising for a decade until lockdowns in the early days of the pandemic led to a sharp reduction in STI testing and screening. Although levels have since recovered, experts are uncertain whether missed cases will lead to increased STI rates, or whether the impact will be offset by pandemic-related changes in sexual behavior.

For developers of STI tests, that uncertaintycouldpresentchallenges as they try to prepare and prioritize for the future.

STIs aren't usually fatal, but they can still create a myriad of personal and public health issues. Chronic, untreated infections in women cause pelvic inflammatory disease in about 12 percent of cases according to one estimate, and approximately 1 in 5 PID cases will result in infertility. Even an asymptomatic STI can increase a person's risk of picking up other infections, like HIV, and STIs can lead to complications for pregnant people and their babies, including infant death in cases of congenital infection.

The majority of people infected with two of the most prevalent STIs chlamydia and gonorrhea have no symptoms whatsoever. For chlamydia, an estimated 90 percent of cases in males are asymptomatic, as are between 70 and 95 percent of cases in females. Gonorrhea infections are also asymptomatic in a little more than half of all cases.

To tamp down rising rates of these infections, the US Centers for Disease Control and Prevention recommends annual screening using nucleic acid amplification tests, and screening up to four times per year for some high-risk groups. Symptomatic cases are also frequently tested using molecular diagnostics, with gonorrhea in particular also isolated and cultured for national reporting and surveillance.

But not only did the pandemic lead to shortages in sample collection and preparation devices, testing reagents, and healthcare and lab staff, it also saw fewer people willing to step into a clinician's office for routine screening.

Overall, STI rates in the US had already risen to an all-time high in 2019, continuing a trend that had been going on for six consecutive years, according to the CDC.

The agency's 2019 STD Surveillance Report, published in April 2021, noted that there were 1,808,703 cases of Chlamydia trachomatis and 616,392 cases of gonorrhea reported to the CDC in 2019. In total, there were 2.5 million reported cases of chlamydia, gonorrhea, and syphilis the three most commonly reported STDs that year which represented a nearly 30 percent increase between 2015 and 2019, CDC said.

Then, COVID happened, resulting in a dramatic reduction in the number of detected STI cases, according to local as well as national data.

BarbaraVan Der Pol runs a clinical microbiology lab at the University of Alabama, Birmingham, and specializes in STIs. The precarious state of plastic consumables and sample prep supplies for molecular testing led her lab, and many others, to restrict STI testing to symptomatic cases and pivot their resources to COVID testing.

"There were three months where nobody in the country could get collection devices, because the same swab used for STI specimens was being used to collect nasal swabs," Van Der Pol said in a recent interview. Just as her lab had restarted its full-capacity STI clinical lab testing a few months ago,the Delta variant came roaring in, and now some of the supplies the lab needs for STI testing are in short supply again, she said.

In a study published online in theAmerican Journal of Preventive Medicine in May, Van Der Pol and colleagues from Quest Diagnostics and Pennsylvania State University reported CT/NG screening hit a low point in April 2020.

The team saw a 59 percent decrease in screening for female patients and a 63 percent decrease for male patients. Further, the results correlated with increases in positivity rates for both chlamydia and gonorrhea.

Overall, the team estimated that in their sample, there were 27,659 cases of chlamydia and 5,577 chases of gonorrhea missed between March and June of 2020.

Philip Chan, a physician and researcher at Brown University who treats STIs in Rhode Island, noted that staff in health departments and publicly funded clinics shifted from STIs to COVID-19 response. Data recently published by him and his team also showed that the pandemic significantly reduced the average number of STI clinic visits per week.

Nationally, reported STI diagnoses dropped significantly, then resurged in 2020 due to COVID-19 lockdowns, according to the CDC. Specifically, a CDC study in Sexually Transmitted Diseases showed that during the most restrictive period of pandemic lockdowns in early April 2020, chlamydia cases dropped nearly 50 percent compared to the same time in 2019. Reported gonorrhea infections fell more than 71 percent, and syphilis infection diagnoses dropped nearly 64 percent. In April 2020 alone, the CDC estimated that roughly 30,000 gonorrhea cases went undetected.

Through a spokesperson, the CDC said three factors likely contributed to the initial decrease in reported cases: reduced screening, limited resources, and stay-at-home orders which may have influenced sexual behaviors and reduced STD transmission.

Yet, by the end of 2020 infection detection resurged, according to CDC data, and the missed cases were mostly recouped. The cumulative total number of cases in the US was just 1 percent lower for syphilis and 14 percent lower for chlamydia for the year than expected, and cumulative numbers of gonorrhea infections were more than 7 percent higher compared to 2019, implying a resurgence of cases.

Commercial perspectives

In September 2020, the CDC issued guidance to healthcare providers on ways to prioritize testing during a national shortage of chlamydia and gonorrhea molecular diagnostic test kits and laboratory supplies.

"The shortages affect multiple diagnostic companies, public health and commercial laboratories, and impact several components of the specimen collection and testing process," the agency wrote in the Dear Colleague Letter.

The agency provided an update in June 2021, noting that "one of the main manufacturers of chlamydia/gonorrhea diagnostic test kits is reporting to CDC that their STI diagnostic test supplies are readily available at normal levels," while other manufacturers reported "continued improvement" in product availability and anticipated "near normal levels" of supplies in the coming months.

Major suppliers of molecular diagnostic tests for STIs including Roche, Hologic, and Becton Dickinson report that STI assay ordering dropped off significantly last year, but that ordering and availability have mostly returned to pre-pandemic rates.

Cindy Perettie, head of Molecular Labs at Roche, said that early in the pandemic the firm invested to scale up production capacity across its core diagnostics business, but that "Roche observed a core business decline, including STIs, at the start of the pandemic," with declines corresponding to peaks in COVID-19 across the world.

But now, Roche has seen an increase in STI testing demand across several of its lab customers, and the firm plans to support higher testing volumes, Perettie said. "We work closely with our country affiliates and customers to plan, as accurately as possible, future demands, in order to secure and safeguard our manufacturing and distribution capabilities," she added.

Kevin Thornal, Hologic's division president of diagnostic solutions, said that Hologic also experienced a significant drop in demand for its STI assays in the spring and summer of 2020 due to the COVID-19 pandemic. "This allowed us to divert some of our manufacturing capacity to production of our Aptima SARS-CoV-2 assay to help meet the rapidly escalating public health need for COVID testing," he said.

Simultaneously, Hologic also initiated aggressive efforts to increase its molecular diagnostics capacities at its San Diego and Manchester, UK, manufacturing sites.

"As of today, demand for STI assays has reverted to nearly pre-pandemic levels, while the need for COVID testing remains high," Thornal noted. But, because of the increased production capacities, the firm is "currently meeting demand on all fronts."

Hologic is also continuing its manufacturing expansion. "We are on track to have the capacity to make at least 75 million tests a quarter across our portfolio by early 2022, which is more than triple our pre-pandemic capacity," Thornal also said.

At Becton Dickinson, Chris Beddard, business line leader of core MDx and women's health and cancer at BD Integrated Diagnostic Solutions, said the firm focused on COVID-19 assay manufacturing in the early months of the pandemic.

However, BD manufactures STI tests on multiple platforms, including the BD Viper XTR and BD Viper LT Systems, and the firm has been able to maintain asteady supply consistent with customer demands, and "unimpacted by COVID-19 test manufacturing," Beddard said.

Nevertheless, BD also experienced declines in demand for STI assays early in the pandemic "as laboratories were, of course, focusing resources to support the urgent need for COVID-19 testing," Beddard said, while people were perhaps reluctant to visit physicians for routine and non-COVID-related health concerns.

BD has since seen demand return to pre-COVID levels.

"It is difficult to predict the immediate- and long-term impact on STI rates that the pandemic may have, but BD continues to have the capacity to expand, to innovate, and to address customer demands and changes in the healthcare landscape," Beddard said.

Resistance, risk, and innovation

What the ultimate consequence of missed cases will be on future STI rates is unknown, and may not be known for years to come.

Charles Cartwright, senior VP of clinical affairs at SpeeDx, suggested in an interview that an explosion in cases may not happen, but even so, "it will take three or four years before that is actually able to be analyzed," he said, in part because surveillance data takes a long time to be published.

Rates of antimicrobial resistance, particularly for gonorrhea, may also potentially be impacted.

SpeeDx offers a gonorrhea diagnostic test that supports "resistance-guided therapy," essentially detecting whether an infection is susceptible to a second-tier antibiotic. The firm partnered with Roche last year to offer its resistance assays, and Perettie said Roche saw this as a unique opportunity to extend an important test to its customers, as AMR STIs are "now listed by the Centers for Disease Control and Prevention as key threats due to the rising resistance and the limited available treatment options." In the long term, Roche sees the partnership as "a key strategic goal" to extend solutions to customers as quickly as possible, Perettie also said.

The increase in reported gonorrhea cases in the CDC's preliminary 2020 data is "definitely concerning," the agency spokesperson said, and it is something the CDC is continuing to study.

Past data suggest that about half of all gonorrhea cases are resistant to at least one antibiotic. CDC has not completed analysis for its 2020 resistance monitoring data, but indications are that there could be an upswing in antibiotic resistance.

For example, without treatment, people may have been carrying gonorrhea infections for a longer time, which increases the chance of the bacteria developing resistance. Asymptomatic oral and rectal infections, in particular, may have gone undetected due to decreased screening, and these sites are more prone to encourage resistance development through horizontal gene transfer with other commensal bacteria.

The newest CDC STI guidelines recommend gonorrhea be treated with an injectable antibiotic, and it is possible there were more cases treated with a less effective oral treatment at times when access to clinical settings was limited or difficult, a CDC spokesperson also said. This is particularly relevant for oral gonorrhea because almost all of these infections are asymptomatic and so, detection depends on screening.

The CDC confirmed that its Gonococcal Isolate Surveillance Project, or GISP,was able to continue surveillance throughout the pandemic.

However, some participating clinics were closed, STI patients were not always the highest priority, health department staff were moved to COVID-19 duties, and testing facilities were short on test kits and lab equipment. The ways all these factors ultimately impacted the GISP program are still being evaluated.

Different groups may also be differently impacted by STIs due to a historic lack of care, aversion to seeking treatment, or prevalence of practices that better enable certain transmission events.

The CDC noted in its report on 2019's rates that certain populations had already been disproportionately impacted by STIs. Many nonwhite populations, for example, have significantly higher rates of STIs, including African Americans, whose STI rates were as much as eight times higher than for non-Hispanic white people. Meanwhile, American Indian, Alaska Native, Native Hawaiian and other Pacific Islander populations were as much as five times higher, and Hispanics had STI rates twice as high.

Also, gay and bisexual men who have sex with men made up nearly half of the total number of primary and secondary syphilis cases in 2019, and in some areas of the US, rates of gonorrhea among this group were 42 times that of heterosexual men.

And young people specifically ages 15 to 24 are also disproportionately impacted, making up 61 percent of the nation's chlamydia cases in 2019 and 42 percent of all gonorrhea cases.

The CDC noted in its assessment that pre-existing disparities in STI services may now also be impacted by staff returning to STI testing labs burnt out by stints of COVID testing. On the other hand, the CDC identified "several new and innovative ways STD services can meet more people where they are" during the COVID-19 pandemic and in the future.

These include walk-in STD express clinics, partnerships with pharmacies and retail health clinics, telehealth, and new support for self-testing and patient-collected specimens.

Prognostication

At least one epidemiological model of chlamydia screening suggests that there would have been twice as many infections if there had been no screening programs and notification of partners between 2000 and 2015. Nevertheless, it is possible thatmissed cases and limited testing resources during the pandemic will be offset by the stay-at-home orders, which may have affected sexual behaviors.

Given that STIs have been increasing for the past decade, Brown's Chan said they will likely continue to increase. But, although clinics saw fewer patients, "on the flip side, I think people were socializing less ... and less socializing means, in many cases, less sex." Anecdotally, Chan said some of the higher-risk patients he sees, who typically have many sexual partners, seem to have picked a single partner with whom to get through the pandemic.

Point-of-care STI tests are also on the cusp of making an impact, and Chan noted that the research that went into COVID diagnostics could hopefully translate to other public health concerns, like STIs.

SpeeDx's Cartwright also highlighted that COVID-19 led many firms, includingSpeeDx, to dramatically scale up manufacturing capacity across the spectrum of molecular diagnostics. "Big instrument companies have placed a lot of instruments, and companies like ours have greatly increased our ability to manufacture large amounts of product to meet demand," he said.

Given the number of COVID tests being run daily, "there will be a huge amount of excess capacity in laboratory testing" when the pandemic ends. Combined with an influx in cash, Cartwright said diagnostics companies may become less nervous about menu expansion intoSTIs, which would in turn help to grow the market.

Still, whether diseases that tend to more strongly impact marginalized groups can ever be given enough attention and funding to be fully controlled is an open question. Van Der Pol noted that STI funding is "orders of magnitude less" than funding for HIV research, for example.

Similarly, antibiotic resistance in gonorrhea is a growing problem, and STIs continue to be especially problematic for women who are of reproductive age. Van Der Pol suggested that this demographic is most likely to be impacted by missed cases during the pandemic. "I think we are going to see a lot of infertility coming out of this, since people will have had untreated infections that have gone on for a year or more," she said.

The solution, ultimately, is to declare that "these are important infections that we should always be paying attention to," and for society to get over the stigma and learn to talk about STIs, Van Der Pol said.

"Hiding them is part of why we are not getting rid of them," she said.

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STIs Reached Record Highs, Then COVID-19 Hit. Test Makers Now Ponder What's Next - GenomeWeb

Huntingtons disease: northern Scotland has one of world’s highest rates and rising sharply here’s why – The Conversation UK

Huntingtons disease (HD) is a devastating inherited neurodegenerative condition that causes a slow but relentless decline in mental health, thinking processes, speech, swallowing and balance, resulting in uncontrollable jerky movements.

Those who have the condition will eventually lose the ability to walk, talk, eat, drink, make decisions or care for themselves. A fatal condition, it typically takes between 15 and 25 years from a person developing symptoms until they die. These symptoms generally begin between the ages of 30 and 60 but can appear before or after this range.

The disease is caused by the faulty Huntingtin gene that expresses a toxic protein, (also called Huntingtin) which builds up and causes early brain-cell death. The child of someone with Huntingtons has a one in two chance of inheriting it, so people at risk grow up experiencing the impact on family members as they become aware of their own likelihood of getting the disease.

Although worldwide research is taking place, there is currently no cure for Huntingtons disease. However, the effects of many of its symptoms can be reduced with a combination of medication, dietary advice and non-medical therapies, including physiotherapy and speech therapy as well as appropriate social care and support.

Nearly 40 years ago, 9.94 per 100,000 people in the north of Scotland had symptoms of Huntingtons, compared to 5.4 per 100,000 elsewhere in the UK. Now our latest research has revealed that 14.6 people per 100,000 in this northernmost region have Huntingtons. The current figure for rest of the UK is 12.4 per 100,000, illustrating that rates are rising generally, but remain particularly high in Scotland.

Our research team decided to re-run a 1984 study that had examined the prevalence of Huntingtons in the Grampian region. We used medical records from labs and HD clinics across the Highlands and Grampian regions to count the number of people across the north of Scotland tested and diagnosed with the condition in the intervening years. Remarkably, we found that cases have increased by 46% since 1984.

In addition, over the last four years, 23% more people with no symptoms but a personal or family history of HD have had a genetic test to see if they will develop the condition possibly in the hope of taking part in trials that might slow the disease.

Our findings mean that northern Scotland has among the highest rates of Huntingtons disease in the world. Its prevalence is almost three times greater than reported elsewhere in Europe (4.7 per 100,000); North America (4.1-5.2 per 100,000); Japan (0.1 per 100,000); Australia (5.70 per 100,000 people) and more than five times the estimated worldwide rate of 2.71 per 100,000 people.

Since the identification of the Huntingtons gene in 1993 made testing for the condition possible, awareness has increased and a diagnosis can now be made in people unaware of their family history of the condition. Along with better care and hope of treatment trials, this has led to more people coming forward for testing.

We believe the high rates of HD diagnosis in Grampian and Highland are due to a combination of underlying genetic susceptibility in ancestral populations dating back to the rule of the Picts; increased awareness that diagnosis will lead to better care and support services; and the region having one of the oldest specialist Huntingtons research clinics in the world. Interestingly, there is also a higher incidence of of multiple sclerosis (MS) in the north of Scotland, though no equivalent genetic explanation for some of the cases.

We did note that the rates of Huntingtons also vary between the different health board regions in the north of Scotland, with more cases in Highland and fewer in Orkney and Shetland compared with Grampian. This local variation in rates could have major drug cost and service delivery implications for the NHS, especially if expensive, complexly administered therapies prove successful.

Although the condition is particularly common in northern Scotland, our data clearly shows that there is far more Huntingtons diagnosed now than the previous prevalence studies suggest, and more people with the HD gene are testing before the appearance of symptoms to access better care and research trials.

The increased diagnosis rate for Huntingtons is likely to be even greater in regions around the world where there has been no long-term focus on the diagnosis and management of the disease.

Health care providers worldwide should now assess local need for specialist services in the expectation that Huntingtons is more common than previously thought. They also need to plan for therapies that can help with the condition, making them part of routine clinical care by improving local services, providing Huntingtons clinics, and studying the prevalence of the disease in their country.

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Huntingtons disease: northern Scotland has one of world's highest rates and rising sharply here's why - The Conversation UK

Greater Gift, AGTC, and 2020 On-site Partner To Honor & Celebrate Clinical Trial Participants and – GlobeNewswire

WINSTON SALEM, N.C., Aug. 24, 2021 (GLOBE NEWSWIRE) -- Greater Gift of Winston Salem, a non-profit that celebrates participation in clinical trials today announced a partnership with Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company developing genetic therapies for the treatment of rare inherited retinal diseases (IRDs), and mobile vision provider 2020 On-site, a company involved in IRD clinical research and provides complimentary eye exams and facilitates the delivery of eyeglasses to students in need.

The pandemic has shone a bright light on the need for clinical trial participation as never before in history, said Lilly Skok Bunch, Executive Director of Greater Gift. Greater Gift has been celebrating and acknowledging clinical trial participants for more than 10 years; this is the first time that a program will recognize trial participants in a way that also yields benefits for students with vision challenges.

Greater Gifts programs celebrate clinical trial participants by making donations on behalf of those individuals to reinforce the impact of their contribution on the advancement of clinical research. Celebrations of clinical trial participants have provided an additional benefit of 130,000 vaccines and meals to children in need through prior programs.

AGTC is thrilled to partner with Greater Gift to honor clinical trial participants and their families for their contribution to the clinical research community, said Halley Losekamp, Director of Patient Enrollment at AGTC. "Through this partnership, our goal is to enhance awareness within the community about the value of clinical trial participation in advancing the science and understanding of gene therapies in IRDs while underscoring AGTCs commitment to support the underserved communities that have limited access to vision care services.

Greater Gift partnered with 2020 On-site because of the organizations commitment to the underserved through their relationship with the Boston Public School System. This donation will allow 2020 On-site to continue the longstanding eye exam program and to double the impact of the gift, as they conduct their annual exam program and delivery of eyeglasses to public school students in need in Boston.

We are honored to be part of this partnership with AGTC and Greater Gift. Both companies align with our mission to make eye care more accessible and to increase trust in and awareness of clinical trials. Now, because of this relationship, we will be able to support even more Boston area school children to help them succeed in the coming school year and beyond, said Jessica Mays, Senior Director of Life Sciences for 2020 On-site, which uses their fleet of Mobile Vision Clinics to provide eye care both routinely and as part of ongoing clinical trials.

About Greater Gift (www.greatergift.org):Greater Gift is a non-profit organization, founded in 2010, with a mission to increase awareness of clinical research, especially among underrepresented communities, to improve global health. Greater Gift builds bridges with underrepresented communities to engage them in research, ensure equal representation in clinical research and to increase access to research as an option for medical care. Since its founding, Greater Gift has honored 130,000 clinical trial volunteers by making donations of vaccines and meals to children in need in their honor.

About AGTC: AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. AGTCs most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM B3 and ACHM A3). Its preclinical programs build on the Companys industry leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders. In recent years AGTC has entered into strategic partnerships with companies including Otonomy, Inc., a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology, and Bionic Sight, LLC, an innovator in the emerging field of optogenetics and retinal coding.

About: 2020 On-site: 2020 On-site has been revolutionizing how vision care benefits are being delivered to companies and patients for 7 years. Its state-of-the-art mobile vision clinics serve over 450 companies in the greater Boston area. The company has also expanded its services to support BioPharma companies, CROs, and clinical sites to develop programs to assess their patients close to home, or even right at their front door. To learn more about how 2020 On-site is supporting clinical research, visit their website.

Contact: David Harrison david@harrisoncommunications.net 410-804-1728

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Greater Gift, AGTC, and 2020 On-site Partner To Honor & Celebrate Clinical Trial Participants and - GlobeNewswire

X-linked Adrenoleukodystrophy Market to Eyewitness Massive Growth by 2028 | Sanofi, Pfizer, J&J, Abbott, Merck, GSK UNLV The Rebel Yell – UNLV The…

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The Global X-linked Adrenoleukodystrophy Market is expected to grow at a CAGR of 7.10% in the forecast period of 2021 to 2028.

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As per the market report analysis, X-linked Adrenoleukodystrophy (X-ALD) is a rare genetic disorder. This condition is caused due to the abnormality in HEALTHCARED1 gene which is present on the X chromosome. It further results in the accumulation of very-long-chain fatty acids (VLCFAs) in the brain, nervous system, and adrenal gland. This accumulation in tissues leads to the damage of myelin which causes neurological problems in patients.

Some of the most significant key factors driving the growth of the Global X-linked Adrenoleukodystrophy Market are increase in the prevalence of X-linked adrenoleukodystrophy, inevitable inheritance of the disease, advancement of therapies for treatment, and increase in the awareness towards genetic diseases coupled with genetic counselling.

Regional analysis, North America dominates the X-Linked Adrenoleukodystrophy Market due to the existence of major key players, high prevalence rate, increasing social awareness, well-developed healthcare sector, and increase in the government support for research & development in this region. Global X-Linked Adrenoleukodystrophy Market in the APAC is expected to grow during the forecast period, because of growing focus of pharmaceutical companies on advancement of therapies for treatment of rare diseases, growing government support, and rising healthcare expenditure.

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Major Key Players:

1 agtc

2 bluebird bio

3 MINORYX THERAPEUTICS SL

4 SwanBio Therapeutics, Inc

5 Viking Therapeutics

6 Novartis AG

7 Sanofi

8 Pfizer Inc

9 Sumitomo Corporation

10 Johnson & Johnson Private Limited

11 AstraZeneca

12 Cipla Inc

13 Abbott

14 Bayer AG

15 Merck KGaA

16 GlaxoSmithKline plc and More.

Global X-linked Adrenoleukodystrophy Market Segmentation:

Market Segment by Type:

1 Adrenomyeloneuropathy (AMN) 2 Adult Cerebral ALD 3 Childhood Cerebral ALD 4 Addisons-Only ALD

Market Segment By Symptoms:

1 Paraparesis 2 Adrenocortical Insufficiency 3 Psychiatric Disorders 4 Dementia Urinary and Genital Tract Disorders 5 Others

Market Segment By Treatment:

1 Stem Cell Transplant 2 Adrenal Insufficiency Treatment 3 Gene Therapy 4 Physical Therapy 5 Corticosteroids 6 Lorenzos Oil 7 Others

Market Segment By Diagnosis:

1 Blood Testing 2 MRI 3 Vision Screening 4 Skin Biopsy 5 Fibroblast Cell Culture

Market Segment By End-Users:

1 Clinics 2 Hospitals 3 Diagnostic Centres 4 Others

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1 Global Adrenoleukodystrophy Treatment Market Industry Trends and Forecast to 2027

2 Global X-Linked Hypophosphatemia (XLH) Treatment Market Industry Trends and Forecast to 2027

3 Global Hypophosphatemia Treatment Market Industry Trends and Forecast to 2027

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X-linked Adrenoleukodystrophy Market to Eyewitness Massive Growth by 2028 | Sanofi, Pfizer, J&J, Abbott, Merck, GSK UNLV The Rebel Yell - UNLV The...

Alpha Thalassemia Market 2021 Industry Analysis, Segmentation, Share, Size, Opportunities and Forecast to 2027 UNLV The Rebel Yell – UNLV The Rebel…

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Key Market Players: Bluebird Bio, Novartis, Kiadis Pharma, Acceleron Pharma

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Gene Therapy

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Private Clinics

Other

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Bedford Biotech Restores "Meaningful Vision" in Blind Patients With Gene Therapyand May Soon Go Public Dallas Innovates -…

Update 6/23/21: Nanoscope Therapeutics announced today that it has received FDA approval of its Investigational New Drug application for its Phase 2b clinical trial of MCO-010, an ambient-light activatable optogenetic monotherapy to restore vision in patients with advanced retinitis pigmentosa (RP). Its randomized, double-blind, sham-controlled Phase 2b trial will begin this month in locations across the U.S.

When you have retinitis pigmentosa, the world slowly goes dark. Most patients with RP lose most of their sight by young adulthood and are often legally blind by age 40. The genetic disorder affects around one in 4,000 people, causing the retinas photoreceptive cells to degrade over time. But a new gene therapy is giving hope and producing life-changing resultsand it could lead to treatments for far more common retinal diseases.

Bedford-based Nanoscope Therapeutics is trying to turn the light back on for RP patientsby developing gene therapies using light-sensitive molecules that could re-sensitize the retina to detect low light levels. That could restore vision in millions of visually impaired people who suffer from RP and other retinal degenerative diseases, like Stargardt disease and dry age-related macular degeneration (AMD). The startups gene therapies, called optogenetics, aim to bring sight to the blind. Nanoscope is one of a number of teams and companies exploring optogenetics as a treatment for RP.

There is no treatment for the diseases that were working on, Nanoscopesco-founder, president, and chief scientific officer, SamarendraMohanty, told Dallas Innovates. So theres a real unmet need that we are trying to fill.

Samarendra Mohanty [Photo: Nanoscope Therapeutics]

Nanoscope, a clinical-stage biotech company, announced earlier this month that its Phase 1/2a clinical study using Multi-Characteristic Opsin (MCO) on RP patients had restored clinically meaningful vision. Significant dose-dependent improvement of visual acuity was demonstrated at 16 weeks, and continued through one year in patients suffering from severe RP, the company said.

The study included 11 patients with advanced RP who had either no light perception or just perception of light in the study eye and no better ability than counting fingers in their other eye. The studys initial positive results were reported at the American Academy of Ophthalmologys 2020 annual meeting last November.

After MCO treatment, the patients reported long-lasting improvements in outdoor light sensitivity and daily activities, saidthe principal investigator, Dr. Santosh Mahapatra, an ophthalmologist and eye surgeon, in a statement. We were pleasantly surprised that after eight weeks of treatment, some subjects could attend their follow-up visits during the study without the assistance of a chaperone. Some of the patients even gained the ability to read letters on a wall or even the large text in a newspaper, use a cell phone, watch television, and could even thread a needle.

Another benefit of the treatment: Vision restoration was produced without the need of stimulating retinal implants or explants (goggles).

Nanoscope co-founder and CEO Sulagna Bhattacharya said the trial studys impact on patients lives has been powerful.

Their quality of life improves significantly, she told us. This is a relief to patients family members, healthcare systems, and society as a whole.

Sulagna Bhattacharya [Photo: Nanoscope Therapeutics]

We expect to begin the first randomized, placebo-controlled, double-masked Phase 2b multi-center optogenetic trial in the U.S. this summer to further validate our gene therapys ability to improve clinically meaningful vision in RP patients, Bhattacharya said in the statement. If successful, it will be the first-ever restorative drug for millions of RP patients worldwide.

On June 23 the company announced it has received FDA approval of its application for the 2b trial, and specified that the trial would begin this month. The trial will involve eight to 10 centers in the U.S., from Beverly Hills to Florida to other locations on the east coast.

[Image: Ivan-balvan/istockphoto]

Nanoscopes RP gene therapy has received orphan drug designation from the FDA. It uses a proprietary AAV2 vector to deliver MCO genes into the retina. This mutation-independent gene therapy is delivered via a single injection through the eye administered in a doctors office.

All 11 subjects participating in the trial had objective and subjective improvement in functional vision, Nanoscope reports. Shape discrimination accuracy improved more than 90% in all the subjects compared to baseline. Further, the performance in two different mobility tests improved by 50%, including the reduction in time to touch a lighted panel. Nanoscope says the test outcomes were highly correlated with improved patient-reported outcomes.

Nanoscopes co-founder, Mohanty, is the inventor of the technology used in the trial.

Optogenetics is a powerful research tool, he said in a statement, but had limited scope of clinical benefit because the opsins had a narrow band of activation, unlike natural light environment. MCO is sensitive to broadband light and activatable by ambient light, thus eliminating the risk of photo-toxicity from long-term continuous use of external intense light stimulation devices.

Nanoscope Therapeutics got a $2 million grant from the National Eye Institute in June 2020 and closed anoversubscribed Series A funding roundin July 2020 to help fund its clinical trial.The startup, a TechFW client, is a spinout from Nanoscope Technologies, serving as a commercialization partner for the R&D enginesMCO vision restoration work. It has 12 employees and a group of consultants and advisors.

Nanoscope Technologies, meanwhile, has received around$10 million in grants from theNational Institutes of Health to help fund its R&D,Bhattacharya said.

The CEO added that the RP study could lead to pivotal results.

Were very excited about our Phase 1/2a results, Bhattacharya said. This trial has the potential to become pivotal, which will allow our product to be available in the clinics to treat millions of blind individuals for whom there is no treatment so far.

Beyond the RP trials, Mohanty says his company placing a big focus on dry age-related macular degeneration (AMD),

Thats a big indication that we are targeting, Mohanty said of AMD, since unlike RP, AMD is super prevalent. According to the National Eye Institute, 11 million Americans have AMD.

Mohanty said that AMD is very rapidly progressing as we are aging and worldwide there are major concerns.

Nanoscope plans to initiatemultiple trials to treat both dry AMD and Stargardt disease, another inherited retina disorder.

Nanoscope Technologies was founded in 2009 by Mohanty. Bhattacharya joined the company as co-founder in 2013. Mohanty and CFO Anthony Togba told Dallas Innovates they are open-minded to the option of going public with their company, and doing due diligence to prepare for that potentiality.

CFO Anthony Togba [Photo: Nanoscope Therapeutics]

Togba says the company is performing readiness activities, undertaking internal processes, and putting in place the structures to be ready to go public.

The timeline? By late Q4 or before, well have a better idea about where were headed, Togba said. The upcoming clinical trial is the preoccupation now to make sure that we have a flawless execution to obtain those results that we expect.

Quincy Preston contributed to this report. It was updated on 6/23/21 to reflect FDA approval of Nanoscopes Phase 2b trial application.

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The investment was led by Advantech Capital, a PE fund based in China that focuses on TMT, pharmaceuticals, and healthcare. This combined with the support from the Cancer Prevention and Research Institute of Texas (CPRIT), which granted OncoNano $9.97 million last year, will support Phase 3 clinical trials for the biotech's technology that can diagnose and treat cancer with high specificity.

Taysha Gene Therapies, which rocketed from a UTSW spinout to a $157 million IPO in under six months last year, has gone global with rights to TSHA-120, apromising AAV9 clinical-stage genetherapy. It's a historic announcement. There are no current treatments for giant axonal neuropathy, or GAN,a severe, progressive disease that affects the central and peripheral nervous systems.TSHA-120 is the first-ever successful in-human intrathecal (spinal) gene transfer.

In this weekly roundup of executive moves in North Texas, you'll also find news from Liberty Capital Bank, Krista Software, Tuesday Morning, Trive Capital, Cantey Hanger, UNT, JUNO, NuZee, Jaunt Air Mobility, Korbyt, and ID90 Travel.

The Series C funding brings Allied BioSciences total to more than $80 million, the team told us. It will be used to grow the biotech's flagship product, SurfaceWise2, which is an active surface coating that can continuously destroy 99.9 percent of viruses on surfaces. Last year, SurfaceWise2 was the first and only surface coating that the EPA approved for continuous protection against COVID-19 with a single application.

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Bedford Biotech Restores "Meaningful Vision" in Blind Patients With Gene Therapyand May Soon Go Public Dallas Innovates -...

‘Like turning back the clock’: Windsor dad with cystic fibrosis among patients seeking access to new therapy – CBC.ca

Windsor, Ont., dad Rian Murphy has had cystic fibrosis since childhood, and wants to live to see his year-old son grow up. Murphy has hope he and other people with the respiratory disease will get access to a new drug, Trikafta, which was approved Friday by Health Canada.

Rian Murphy was diagnosed with cystic fibrosis as a child andnever expected to live into his 30s, but Health Canada's recent approval of a new breakthrough drug treatmenthas theWindsor, Ont., dad hopefulhe'll be spending many more years with hisson.

"It's a massive step going forward for cystic fibrosis patients such as myself," said Murphy about the triple-combination therapy Trikafta. "At the end of the day, it's a big, big window of opportunity for us to look down the road, future-wise."

On Friday, Trikaftawasapproved for use in patientsage 12 and over who have aminimum of one of the CF F508del gene mutations.

Cystic Fibrosis Canada (CFC) calls Trikaftaa "transformational" therapy that couldtreat up to 90 per centof Canadianswith theprogressive, genetic disease, whichaffects the lungs and digestive system, and is the most common fatal genetic disease in children. TheCFCestimates one in every 3,600 children is born with the disease, and over4,370 Canadiansattend specialized clinics.

Over time, the CFC says, Trikafta could reduce severe lung disease by 60 per cent andthe number of deaths by 15 per cent,and increase life expectancy by several years, the CFC says in quoting research.Clearing the airways from mucus buildup is important in CF care.

In the last three years, half of Canadians who died of cystic fibrosis were under age 34.

"I never thought about retirement. Inever thought about those things because my whole life I was told you're never going to make it until you're 20, you're 30," said Murphy, who with wife Diane are parents to their year-old son Logan.

Three years ago, Murphy lost significant lung function andwas hospitalized for threeweeks at St. Michael's Hospital in Toronto. He hadbeen on and off intravenousantibiotics for months.

"On an average day, I'm doing about two to three hours of masks, and vests and physiotherapy, not including all the pills I take," he said.

"I'm 34 years old. If I can obtain this drug [Trikafta] and take it for the recommended period of time to get the results, it would be like me turning back the clock."

There's no cure for CF. While other therapies work to address the symptoms, Trikafta helps the defective protein function more effectively.

With Health Canada's approval, doctors can now prescribe Trikafta.

But aswith a couple of other drugs for CF, provincial insurance coverage for Trikaftaremains a concern for patients, Kelly Grover, president and chief executive officer of CFC, saidin a release.

"We turn to the provinces next. They must immediately fund Kalydeco and Orkambi, which have been in negotiations for more than a year, and fund Trikafta as soon as possible. Provincesend the wait and save lives."

The pan-Canadian Pharmaceutical Alliance (pCPA) isa regulatory body that negotiates drug prices on behalf of the provinces.

In astatement released shortly after Health Canada announcedapproval of Trikafta, thepCPAsaid it has agreed to negotiate prices for the CF drugsOrkambi and Kalydeco, and Trikafta might be added to the agreements, pending a positive regulatory and health technology assessment recommendation.

CBC reached out to the Ontario government for comment, and in an email, the Ministry of Health said the province "recognizes that the cystic fibrosis community is anxious for access to new and effective treatments such as Trikafta," and "will continue to work productively through the established review and pCPA processes."

In the meantime, Murphy and his wife have started a fundraiser, hoping to raise enough money so hecan eventually access the drug.

"If I can get a couple months, that's huge," he said.

His wife Diane, who'sactively involved with CFC, as well as a petition and Instagram groups calling for the Ontario government to fund Trikafta, encourages the public to send letters to the province.

Shehas hopes of her husband "watching our child grow up."

Link:
'Like turning back the clock': Windsor dad with cystic fibrosis among patients seeking access to new therapy - CBC.ca

Germline genetic testing can benefit all cancer patients as a routine practice in cancer care – PRNewswire

"Cancer is a disease of genetics, yet clinical practice has struggled to keep pace with rapid advancements in research, particularly with respect to the role of germline genetics. Testing guidelines and medical policy often codify barriers, further lengthening the path to adoption of widespread testing and in some cases restricting access to precision therapies and clinical treatment trials," said Ed Esplin, M.D., Ph.D., FACMG, FACP, clinical geneticist at Invitae. "Research presented at ASCO shows that cancer-linked genetic changes are common across cancer types and when patients do receive germline testing, over two thirds of those with positive results are eligible for changes to their treatment plans. It's clear that incorporating germline testing alongside tumor profiling can help oncologists better tailor treatment for each patient."

Data from 250 pancreatic cancer patients from the landmark INTERCEPT study conducted at the Mayo Clinic found that nearly one in six patients with pancreatic cancer (n=38) showed cancer-linked genetic changes and, importantly, receiving germline testing was associated with improved survival.

A separate study of prostate cancer patients confirmed similar findings in other cancer types that limiting testing deprives patients and clinicians of actionable information. In the first-ever presentation of the PROCLAIM study, which was conducted primarily in community urology clinics, of patients diagnosed with prostate cancer, a significant number of cancer-linked variants were missed if testing was done based on NCCN guidelines. Of the 532 patients with clinician-reported data, nearly half, 45% (n=239), did not meet NCCN criteria. Overall, 59 patients had a cancer-linked variant; one in 10 of them did not meet the criteria (9.6%, n=23), and 12.3% (n=36) of patients met the criteria. When a 12-gene panel was used, only 29 patients were found to have a cancer-linked variant and one third of these patients were missed by guidelines.

A third study showed simply changing medical policy is not enough to drive changes in clinician adoption. In a review of two independent datasets, including commercially insured and Medicare Advantage enrollees, only 3% (n=1,675) of the 55,595 colorectal cancer patients received germline genetic testing, despite medical policy recommending germline genetic testing for all colorectal cancer patients (consistent with the INTERCEPT colorectal cancer study). Of the patients who received testing, 18% (n=143) had a cancer-linked variant and two thirds, or 67% (n=96), of those patients were potentially eligible for precision therapy and/or clinical trials.

"The data have been available for years that show knowing what changes patients have in their genes is beneficial to treating their cancer. Yet the oncology community has been slower to adopt germline testing than tumor profiling, for reasons that are not entirely clear. These data presented at ASCO highlight the need for oncologists to embrace germline genetic testing as routine practice for all cancer patients," said Robert Nussbaum, M.D., chief medical officer at Invitae. "A positive germline genetic result may enable patients to enroll in clinical trials or gain access to new precision medicines. And equally important, the discovery of an inherited variant can alert relatives to seek out earlier cancer screening, helping avoid later-stage diagnoses and offering a treatment benefit if cancer develops."

Invitae aims to help overcome obstacles to the adoption of genetic testing by providing physicians with clinical consults to help interpret results and reducing cost as a barrier to genetic information. Invitae also provides patients direct access to genetic counselors, helping to integrate routine genetic testing into patient care with GIA, a HIPAA-compliant chatbot. Family members are also able to receive no-charge genetic testing if a positive result is found.

Details of the 2021 ASCO presentations:

Oral Abstract Session: Prevention, Risk Reduction, and Hereditary Cancer

Poster Discussion Session: Prevention, Risk Reduction, and Hereditary Cancer

Poster Session: Prevention, Risk Reduction, and Hereditary Cancer

Poster Session: Gastrointestinal Cancer--GastroesophageaI, Pancreatic, and Hepatobiliary

About Invitae Invitae Corporation(NYSE: NVTA) is a leading medical genetics company whose mission is to bring comprehensive genetic information into mainstream medicine to improve healthcare for billions of people. Invitae's goal is to aggregate the world's genetic tests into a single service with higher quality, faster turnaround time, and lower prices. For more information, visit the company's website atinvitae.com.

Safe Harbor Statement This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements relating to the benefits of germline testing and genetic information; and that the data presented at ASCO highlight the need for increased germline testing in all cancer patients regardless of medical policy. Forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially, and reported results should not be considered as an indication of future performance. These risks and uncertainties include, but are not limited to: the company's history of losses; the company's ability to compete; the company's failure to manage growth effectively; the company's need to scale its infrastructure in advance of demand for its tests and to increase demand for its tests; the company's ability to use rapidly changing genetic data to interpret test results accurately and consistently; security breaches, loss of data and other disruptions; laws and regulations applicable to the company's business; and the other risks set forth in the company's filings with the Securities and Exchange Commission, including the risks set forth in the company's Quarterly Report on Form 10-Q for the quarter ended March 31, 2021. These forward-looking statements speak only as of the date hereof, and Invitae Corporation disclaims any obligation to update these forward-looking statements.

Contact: Laura D'Angelo [emailprotected] (628) 213-3283

SOURCE Invitae Corporation

http://www.invitae.com

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Germline genetic testing can benefit all cancer patients as a routine practice in cancer care - PRNewswire

Seattle Cancer Care Alliance is an Authorized Treatment Center for Ide-cel CAR T-Cell Therapy – StreetInsider.com

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Cancer center among the first in the nation to offer the first approved CAR T-cell therapy for adults with multiple myeloma

SEATTLE--(BUSINESS WIRE)-- Seattle Cancer Care Alliance (SCCA), the only National Comprehensive Cancer Network designated cancer center in Washington State, today announced that it is an authorized treatment center for the new B-cell maturation antigen (BCMA) targeted chimeric antigen receptor (CAR) T-cell therapy, idecabtagene vicleucel, also known as ide-cel.

Ide-cel was approved by the U.S. Food and Drug Administration (FDA) on March 26, 2021, and is indicated for the treatment of adult patients with relapsed refractory multiple myeloma after four or more prior lines of therapy including a proteasome inhibitor, an immunomodulatory therapy and an anti-CD38 antibody. It is the first cell-based gene therapy approved by the FDA for the treatment of multiple myeloma and is being marketed under the brand name Abecma.

We are pleased to offer this new advanced therapy to patients who are suffering from relapsed or refractory multiple myeloma, said Nancy Davidson, MD, president and executive director of Seattle Cancer Care Alliance. We are committed to delivering personalized care to our patients and improving patient outcomes and excited to be among the first cancer centers in the nation to offer this treatment to adult patients with multiple myeloma.

Multiple myeloma is a cancer of plasma cells in which abnormal plasma cells build up in bone marrow and limit the bodys ability to make enough healthy blood cells, thus resulting in low blood counts. Multiple myeloma is also associated with bone and kidney damage as well as a weakened immune system. There are over 140,000 people in the United States living with this cancer and according the American Cancer Society approximately 34,920 new cases will be diagnosed in 2021, and 12,410 deaths among those with multiple myeloma will occur.

Ide-cel is a one-time therapy that is created from a patients own white blood cells, which have been modified to recognize and attack myeloma cells. As an anti-BCMA CAR T-cell therapy, ide-cel recognizes and binds to BCMA, a protein that is nearly universally expressed on cancer cells in multiple myeloma, leading to the death of BCMA-expressing cells.

In the clinical study that supported its approval, ide-cel was shown to be safe and effective. Approximately 72% of patients partially or completely responded to the treatment with 28% of patients showing complete response. An estimated 65% of this group remained in complete response to ide-cel for at least 12 months.

The FDA approval of this novel therapy is a significant milestone in the advancement of new, innovative therapies for multiple myeloma, said David Maloney, MD, PhD, medical director for cellular immunotherapy at the Bezos Family Immunotherapy Clinic at Seattle Cancer Care Alliance. We are excited about the continued expansion of CAR T-cell treatment options available to our patients, and the potential ide-cel offers to extend the lives of those who have multiple myeloma.

Our clinical trials at the SCCA have provided us with extensive experience using BCMA CAR T-cells for multiple myeloma. The new FDA approval allows our to leverage this knowledge and safely bring a promising therapy to a wider population of adult patients with multiple myeloma, said Damian Green, MD, Seattle Cancer Care Alliance and Associate Professor, and who leads translational myeloma research programs at Seattle Cancer Care Alliance and the Fred Hutchinson Cancer Research Center.

SCCA is home to several of the worlds leading immunotherapy experts whose research has contributed to the foundation of many immunotherapies currently used to treat cancer. SCCAs Bezos Family Immunotherapy Clinic, which opened in 2016, is a state-of-the-art center dedicated to offering the newest cellular immunotherapy clinical trials and FDA approved treatments.

About Seattle Cancer Care Alliance

Seattle Cancer Care Alliance brings together the leading research teams and cancer specialists from Fred Hutch, Seattle Childrens and UW Medicine one extraordinary group whose sole purpose is the pursuit of better, longer, richer lives for our patients. Based in Seattles South Lake Union neighborhood, Seattle Cancer Care Alliance has nine clinical care sites in the region, including a medical oncology clinic at EvergreenHealth in Kirkland; hematology/medical oncology and infusion services at Overlake Medical Center in Bellevue, medical and radiation oncology clinics at UW Medical Center - Northwest Seattle and medical oncology services at SCCA Issaquah, as well as Network affiliations with hospitals in five states. For more information about SCCA, visit seattlecca.org.

View source version on businesswire.com: https://www.businesswire.com/news/home/20210513005357/en/

Karina San Juan, ksanjuangu@seattlecca.org or (206) 606-1926 Heather Platisha, hplatisha@seattlecca.org or (206) 606-7239

Source: Seattle Cancer Care Alliance

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Seattle Cancer Care Alliance is an Authorized Treatment Center for Ide-cel CAR T-Cell Therapy - StreetInsider.com

Discovery of a new genetic cause of hearing loss illuminates how inner ear works – India Education Diary

A gene calledGAS2plays a key role in normal hearing, and its absence causes severe hearing loss, according to a study led by researchers in the Perelman School of Medicine at the University of Pennsylvania.

The researchers, whose findings arepublished online today inDevelopmental Cell, discovered that the protein encoded byGAS2is crucial for maintaining the structural stiffness of support cells in the inner ear that normally help amplify incoming sound waves. They showed that inner ear support cells lacking functionalGAS2lose their amplifier abilities, causing severe hearing impairment in mice. The researchers also identified people who haveGAS2mutations and severe hearing loss.

Anatomists 150 years ago took pains to draw these support cells with the details of their unique internal structures, but its only now, with this discovery aboutGAS2, that we understand the importance of those structures for normal hearing, said study senior authorDouglas J. Epstein, PhD, professor of genetics at Penn Medicine.

Two to three of every 1,000 children in the United States are born with hearing loss in one or both ears. About half of these cases are genetic. Although hearing aids and cochlear implants often can help, these devices seldom restore hearing to normal.

One of the main focuses of the Epstein laboratory at Penn Medicine is the study of genes that control the development and function of the inner eargenes that are often implicated in congenital hearing loss. The inner ear contains a complex, snail-shaped structure, the cochlea, that amplifies the vibrations from sound waves, transduces them into nerve signals, and sends those signals toward the auditory cortex of the brain.

Unraveling the role ofGas2in hearing

A few years ago, Epsteins team discovered thatGas2, the mouse version of humanGAS2, is switched on in embryos by another gene known to be critical for inner ear development. To determineGas2s role in that development, the team developed a line of mice in which the gene had been knocked out of the genome and called themGas2-knockout mice.

Alex Rohacek, PhD, a former graduate student in the Epstein lab, was puzzled to observe that theGas2-knockout mice had inner ears with cells and structures that seemed quite normal. However, the animals, when tested, turned out to be severely hearing-impaired, with deficits at high sound frequencies of up to 50 decibelsequivalent to a loss of 99.999 percent of the normal acoustic energy.

Tingfang Chen, PhD, a postdoctoral fellow and co-first author on the study, determined thatGas2is normally active within inner-ear support cells called pillar cells and Deiters cells. In these cells, the protein encoded by the gene binds to flexible, tube-like structures called microtubules in a way that bundles and stabilizes them, effectively stiffening the cells.

With help from the collaborating team ofBenjamin L. Prosser, PhD, assistant professor of Physiology at Penn Medicine and an expert on microtubules, the researchers discovered that when pillar cells and Deiters cells lackGas2, their microtubule bundles tend to come apart, dramatically reducing the stiffness of the cells.

That turns out to have dire implications for hearing. Within the inner ear, pillar cells and Dieters cells help form the basic structure of the cochlea and serve as physical supports for cells called outer hair cells. The outer hair cells move in response to incoming acoustical vibrationsessentially to provide a crucial amplification of that sound energy. The experiments revealed that the pillar and Deiters cells loss of stiffness, due to the absence ofGas2, severely degrades the sound-amplifying properties of the outer hair cells they support.

We observed that some of Deiters cells in theGas2-knockout mice even buckled under the tension of the rapid movements of the outer hair cells, Epstein said.

The experiments included sophisticated imaging of propagating sound waves in the inner ears of liveGas2-knockout and normal mice, conducted by collaboratorJohn Oghalai, MD, chair and professor of otolaryngology-head and neck surgery at the Keck School of Medicine of USC, and his team.

GAS2also causes human hearing loss

Curiously, the researchers could find no reports ofGAS2-associated congenital hearing loss in the medical literature. Even when they canvassed colleagues around the world who run hearing-loss clinics, they came up empty-handed.

Then one day,Hannie Kremer, PhD, professor and chair of molecular otogenetics at Radboud University Medical Center in the Netherlands, emailed Epstein. She and her team had been studying a Somalian family in which four of the siblings had severe hearing loss from early life. The affected family members had no mutations in known hearing-loss genesbut each carried two mutant copies ofGAS2.

The study therefore establishesGAS2as a very probable new hearing loss gene in humansthe first one known to affect the mechanical properties of inner ear support cells.

The prevalence of hearing loss in people due toGAS2mutations remains to be determined, but Epstein noted that this type of congenital hearing loss is nevertheless an attractive target for a future gene therapy.

In many genetic hearing loss conditions, the affected cells are permanently damaged or die, but in this one, the affected cells are intact and conceivably could be restored to normal or near-normal by restoringGAS2function, he said.

He added that such a gene therapy might be useful not only in more obvious cases of hearing loss in early childhood, but also in casesperhaps more numerousin which inherited mutations lead to a slower development of hearing loss in adulthood.

Funding was provided by the National Institutes of Health (R01 DC006254, R01 DC014450, R01 DC013774, R01 DC017741, R01 HL133080), the Boucai Innovation Fund in Auditory Genomics, the National Science Foundation (15-48571), and the Heinsius Houbolt Foundation.

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Discovery of a new genetic cause of hearing loss illuminates how inner ear works - India Education Diary