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HOOKIPA Pharma Announces First Person Dosed in Phase 1b Clinical Trial of HB-500 for the Treatment of HIV

Posted: July 2, 2024 at 2:33 am

NEW YORK and VIENNA, July 01, 2024 (GLOBE NEWSWIRE) -- HOOKIPA Pharma Inc. (NASDAQ: HOOK, HOOKIPA), a company developing a new class of immunotherapeutics based on its proprietary arenavirus platform, today announced that the first person has been dosed in a Phase 1b clinical trial of HB-500, an investigational therapeutic vaccine for the treatment of HIV. As a result, HOOKIPA achieves a $5 million non-dilutive milestone payment under its collaboration and license agreement with Gilead.

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Alvotech Issues New Shares to Holders of Convertible Bonds

Posted: July 2, 2024 at 2:33 am

Alvotech (NASDAQ: ALVO), a global biotech company specializing in the development and manufacture of biosimilar medicines for patients worldwide, today issued new shares to holders of subordinated convertible bonds, originally issued by Alvotech on November 16, 2022, and December 20, 2022, with maturity on December 20, 2025 (the “Convertible Bonds”).

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Ipsen expands collaboration and license agreement for development of Cabometyx® in advanced neuroendocrine tumors based on positive CABINET Phase III…

Posted: July 2, 2024 at 2:33 am

PARIS, FRANCE, 2 July 2024 - Ipsen (Euronext: IPN; ADR: IPSEY) announced today confirmation of an expanded collaboration and license agreement with Exelixis, Inc. for the development of Cabometyx® (cabozantinib) in advanced pancreatic neuroendocrine tumors (pNETs) and advanced extra-pancreatic neuroendocrine tumors (epNETs). The agreement is based on positive outcomes from the CABINET Phase III trial, led by the Alliance for Clinical Trials in Oncology and sponsored by the National Cancer Institute (NCI), which investigated Cabometyx versus placebo in people living with advanced pNETs or advanced epNETs whose disease had progressed after prior systemic therapy. An independent Data and Safety Monitoring Board recommended to stop accrual to the study, unblind patients and allow crossover from placebo to Cabometyx. This was due to early efficacy demonstrated at an interim analysis in both of the trial’s cohorts, with clinically meaningful improvements in progression-free survival (PFS).1

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Medsenic, BioSenic’s subsidiary, signed a new set of licensing and commercialization agreements with Phebra PTY Ltd.

Posted: July 2, 2024 at 2:33 am

PRESS RELEASE – PRIVILEGED INFORMATION

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Limitations in next-generation sequencing-based genotyping of breast cancer polygenic risk score loci | European … – Nature.com

Posted: June 24, 2024 at 2:41 am

Missing loci & convertibility to hg38

For four BC PRS loci, no variants were listed at the specified genomic position in gnomAD v2.1.1, namely rs572022984, rs113778879, rs73754909, and rs79461387. gnomAD v3.1.2 also reported no variants for three of these four loci for corresponding loci in hg38 as defined by dbSNP [23] (Supplementary Table2). Locus rs572022984 was listed but with an overall allele count of zero in NFE samples (Table2).

For two loci, conversion to hg38 resulted in a change in alleles, namely for rs143384623 (hg19: 1-145604302-C-CT; hg38: 1-145830798-C-CA) and rs550057 (hg19: 9-136146597-C-T; hg38: 9-133271182-T-C). For rs143384623, the change of the alternative allele from CT to CA did not result in a noticeable shift in AFs observed in gnomAD NFE samples (5142/13304 (0.39) in v2.1.1 versus 24316/64610 (0.38) in v3.1.2, two-sided Fishers exact test p=0.14). For rs550057, the observed AFs appeared exactly opposite, i.e., 3786/14828 (0.26) for allele T in gnomAD v2.1.1 and 49878/67552 (0.74) for allele C in gnomAD v3.1.2. Therefore, 149878/67552 was assumed as the gnomAD v3.1.2 effect AF at this bi-allelic site.

For 39 of the 320 PRS loci listed with AF>0 in gnomAD v3.1.2, at least one observation of technical artifacts was reported: 38 loci were flagged as being located in low-complexity regions, 3 as being localized at a low-quality site, and 1 failed the allele-specific VQSR filter (Supplementary Table2).

Due to the absolute difference threshold 0.016 (Supplementary Fig.1), 24 loci were determined as showing deviating AFs compared to CanRisk (Fig.1, Table2). Absolute differences ranged from 0.03 to 0.71, and for 21 out of these 24 loci (87.5%), technical artifacts were reported in gnomAD v3.1.2.

Extremely deviating AFs with an absolute difference>0.016 are indicated by red markers.

All 49 PRS loci for which a noticeably deviating AF was observed in at least one of the data sets provided by the five participating GC-HBOC centers are listed in Table3.

For the IMGAG DRAGEN data, 0.052 was calculated as threshold to determine noticeably deviating AFs (Supplementary Fig.2), resulting in 18 loci affected (Table3, Fig.2). Of these, 16 were previously also identified as missing or showing noticeably deviating AFs in gnomAD v3.1.2. The exceptions were rs62485509 and rs9931038. For IMGAG freebayes data, 0.036 was calculated as threshold (Supplementary Fig.2), resulting in 16 loci from the BCAC 313 BC PRS determined as showing a noticeably deviating AF. Of these, 11 loci were also identified as showing deviating AF in IMGAG DRAGEN data, and all but rs12406858 and rs11268668 were previously identified as missing or showing deviating AFs in gnomAD v3.1.2.

Data were provided by the Institute of Medical Genetics and Applied Genomics (IMGAG) at University Hospital Tbingen, Institute for Clinical Genetics (ICG) at University Hospital Carl Gustav Carus Dresden, by the Department of Medical Genetics (DMG) at University Hospital Mnster, by the Center for Familial Breast and Ovarian Cancer (CFBOC) at University Hospital Cologne, and by the Institute of Human Genetics (IHG) at the University of Regensburg.

Considering genotyping data provided by the ICG based on 585 samples, 23 of the overall 324 PRS loci did not meet the minimum quality criteria (read depth20) in more than 25% of samples and were discarded (Supplementary Table3). Additionally, GATK reported read depth <20 for >25% of samples for rs56097627 and rs143384623. For 260 of the remaining 299 PRS loci (86.96%), forced genotyping with GATK and freebayes resulted in the observation of identical AFs. For both ICG GATK and freebayes data, 0.063 was calculated as threshold to determine noticeably deviating AFs (Supplementary Fig.3). Using this threshold, 11 loci showed noticeably deviating AFs in the GATK data set (including two loci exclusive for BCAC 313 BC PRS) and 14 loci in the freebayes data set (including three loci exclusive for BCAC 313 BC PRS), respectively, with an overlap of 7 (Table3, Fig.2).

The DMG provided GATK- and DRAGEN-based BRIDGES 306 BC PRS genotyping data of 545 samples. Locus rs138179519 did not meet the quality criteria, and additionally rs774021038 using DRAGEN. Of the remaining 304 loci, 252 (82.89%) showed identical AFs (Supplementary Table3). Using a threshold of 0.052 (Supplementary Fig.4), resulted in 20 loci showing deviating AFs in GATK data and14 loci in DRAGEN data, respectively,with an overlap of 9 loci.

For the CFBOC data based on 412 samples, a threshold of 0.047 was calculated (Supplementary Fig.5). The loci of the BRIDGES 306 BC PRS were considered, 243 (79.41%) of which showed identical AFs for both callers applied (Supplementary Table3). Overall 25 loci (all of which are included also in the BCAC 313 BC PRS) showed deviating AFs: 16 loci in GATK and 19 loci in freebayes data, with an overlap of 10 loci.

The IHG provided GATK- and CLC-based BRIDGES 306 BC PRS genotyping data of 251 samples (Supplementary Methods). Four loci did not meet the quality criteria in both settings, and additional four in the CLC setting. Of the remaining 298 loci, 228 (76.51%) showed identical AFs (Supplementary Table3). Using a threshold of 0.063 (Supplementary Fig.6), resulted in 23 loci showing noticeably deviating AFs in GATK data, respectively 19 loci in CLC data, with an overlap of 10 loci.

In summary, for four loci, deviating AFs were reported in all GC-HBOC real-world settings examined, namely for rs56097627, rs113778879, rs57589542, and rs3988353. Further four loci, namely rs574103382, rs73754909, rs3057314, and rs57920543, were reported with deviating AFs in all settings except for one (Table3).

However, there were also 16 loci that were conspicuous in a single setting exclusively, namely five in IHG GATK data (rs1511243, rs4880038, rs1027113, rs12709163, rs1111207), three each in ICG freebayes data (rs34207738, rs147399132, rs199504893) and in IHG CLC data (rs10975870, rs11049431, rs144767203), two in DMG GATK data (rs10644978, rs66987842), and one each in IMGAG DRAGEN (rs9931038), IMGAG freebayes data (rs12406858), and CFBOC freebayes data (rs140702307). Another three loci (rs10074269, rs55941023, rs35054928) showed AF deviations in only one center, but these were concordant.

Considering the loci non-existent in gnomAD v3.1.2, rs113778879 was not observed with expected AF in any GC-HBOC center, and rs73754909 only with forced DRAGEN calling in DMG data. For rs79461387, expected AFs were reported consistently when using freebayes, but not by unforced DRAGEN calling and in two settings using forced GATK. Of note, rs572022984 with zero allele count in gnomAD v3.1.2 NFEs and an expected AF of 0.0364 in CanRisk, was consistently not observed at all or with a maximum AF of 0.0037 (Supplementary Table3).

Five loci showing aberrant AFs in gnomAD v3.1.2 NFEs (Table2) were not reported with deviating AF by any of the participating GC-HBOC centers, namely rs78425380, rs62331150, rs60954078, rs10862899, and rs112855987.

Without further information and assuming a standardized PRS at the 50th percentile, the estimated 10-year risks of developing primary BC of cancer-unaffected women of 20, 40, and 60 years of age were 0.1%, 1.5%, and 3.4% according to CanRisk (Supplementary Table4). Percentiles of PRSs from artificial VCF files with aberrant dosages (see Materials and Methods) ranged from 47.5% (IHG CLC, BRIDGES 306) up to 55.7% (ICG freebayes, BCAC 313). The risk of 0.1% for a 20-year-old woman was concordantly unchanged in all scenarios including artificial PRSs. For a 40-year-old woman, estimated 10-year risks were increased by 0.1% in seven scenarios, and for a 60-year-old woman by up to 0.2% in eight scenarios.

Estimated remaining lifetime risks of developing primary BC assuming an average PRS (50th percentile) of cancer-unaffected women aged 20, 40, and 60 years are 11.3%, 10.9%, and 7.1% according to CanRisk (Supplementary Table4). When using PRSs from artificial VCF files with aberrant dosages, estimated lifetime risks ranged from 11.1% up to 11.9% for a 20-year-old woman, from 10.6% up to 11.4% for a 40-year-old woman, and from 7.0% up to 7.4% for a 60-year-old woman. The lowest estimates were obtained with the BRIDGES 306 BC PRS based on IHG CLC data with 19 artificial dosages imputed, and the highest with the BCAC 313 BC PRS based on ICG freebayes data with 14 artificial dosages imputed.

For 20 PRS loci showing noticeably deviating AFs in at least one real-world NGS data set, alternative alleles or overlapping variants with minimum AF 0.01 in NFEs were reported in gnomAD v3.1.2 (Supplementary Table5). For rs73754909 and rs79461387, both SNVs and non-existent in gnomAD v3.1.2, deletions were reported with comparable AFs to the ones expected by CanRisk. For both deletions, the adjacent downstream nucleotide of the reference sequence was identical to the substituted nucleotide of the expected effect allele (Fig.3). For rs113778879, which is also an SNV not contained in gnomAD v3.1.2, a similar observation could be made (Supplementary Fig.7), but the reported AF exceeds the expected one by more than 0.1 (0.5762 versus 0.6818).

Both alternative alleles are deletions with the adjacent downstream nucleotide identical to the expected substituted one.

For 28 out of the 49 loci showing noticeable deviating AFs in at least one real-world data set, proxies in 1000G GRCh37 microarray data, 1000G GRCh38 High Coverage WGS data, or TOPMED European data could be identified (Supplementary Table6). For rs113778879, rs73754909, and rs79461387, LDpair based on GRCh38 reported the same alternative alleles as gnomAD v3.1.2 (Supplementary Table5), where the original PRS loci are non-existent.

Proxies and alternative alleles showing AFs in gnomAD v3.1.2 comparable to expected CanRisk AFs, i.e., an absolute deviation <0.016, were considered as possible workarounds for improved PRS genotyping, and further evaluated with respect to observed AFs in IMGAG freebayes data (Table4). For 19 of these 21 PRS loci, absolute differences between expected and observed AFs in IMGAG freebayes data remained below the previously defined IMGAG freebayes-specific threshold of 0.036. The exceptions were the substitutions of rs12406858 and rs79461387. The latter is noteworthy because the original PRS locus, which is an SNV, was correctly called by freebayes in forced and unforced mode (Table3), whereas GATK HaplotypeCaller seemed to call an overlapping deletion of sequence GAG in DMG and CFBOC data. Also noteworthy are the potential replacements of rs73754909 and rs111833376, as both variants were called with noticeably deviating AFs in most real-world data sets.

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Y chromosome is evolving faster than the X, primate study reveals – Livescience.com

Posted: June 24, 2024 at 2:41 am

The Y chromosome in primates including humans is evolving much more rapidly than the X chromosome, new research on six primate species suggests.

For instance, humans and chimpanzees share upwards of 98% of their DNA across the whole of the genome, but just 14% to 27% of the DNA sequences on the human Y chromosome are shared with our closest living relatives.

The finding surprised scientists, given that humans and chimpanzees diverged just 7 million years ago a blip in evolutionary terms.

"I expect my genome to be very different to that of bacteria or insects because a lot of time has elapsed, evolutionarily speaking," study co-author Brandon Pickett, a postdoctoral fellow at the National Human Genome Research Institute (NHGRI) at the National Institutes of Health, told Live Science. "But from other primates, I expect it to be pretty similar."

Related: Genomes of 51 animal species mapped in record time, creating 'evolutionary time machine'

It's not clear exactly why the Y chromosome is evolving so rapidly. For starters there is only a single copy of the Y chromosome per cell in primates, females carry two copies of the X chromosome, while males carry an X and a Y chromosome the Y chromosome plays a critical role in sperm production and fertility. Having only a single copy of the Y chromosome presents a vulnerability if changes happen to occur, there is no second chromosome to act as a backup.

And changes are likely to occur due to something called mutation bias. The Y chromosome may be so prone to change because it generates many sperm. This requires lots of DNA replication. And every time DNA is copied, there's a chance for mistakes to creep in.

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Scientists have previously sequenced the primate genome for all 16 representative families.

In the new study, published May 29 in the journal Nature, scientists compared the sex chromosomes of five great ape species chimpanzees (Pan troglodytes), bonobos (Pan paniscus), western lowland gorillas (Gorilla gorilla gorilla) and Bornean and Sumatran orangutans (Pongo pygmaeus and Pongo abelii) and one more distantly related to humans, siamang gibbons (Symphalangus syndactylus).

The team studied the chromosomes using telomere-to-telomere (T2T) sequencing. T2T can accurately sequence repetitive elements, including the protective telomere "caps" of chromosomes that have proven difficult to read in the past, Pickett said. The researchers used computing software to make comparisons between the sequencing results, by creating alignments to reveal which parts of the chromosome had changed and which parts had stayed the same.

The chromosomal X and Y sequences of each of the six species were also compared to the human X and Y chromosome, already sequenced in an earlier studywith the T2T method.

The findings revealed that across all the studied species, the Y chromosome evolved rapidly. Even species in the same genus have very different Y chromosomes to one another. For instance, chimpanzees and bonobos diverged just 1 million to 2 million years ago, yet there is a dramatic difference in their Y chromosome lengths, said Christian Roos, a senior scientist at the Primate Genetics Laboratory, German Primate Center, who was not involved in the study.

In some cases the difference in length caused by chromosome losses or duplications that occur when DNA is copied amounted to up to about half of the observed differences. For example, the Y chromosome from the Sumatran orangutan is twice as long as the gibbon's Y chromosome.

In contrast, the study found that the X chromosome was highly conserved across the primate species, as might be expected for a structure with a critical role in reproduction.

One reason the Y seems to have thrived despite such a high rate of mutation is that across all the studied species, it contains stretches of highly repetitive genetic material, such as palindromic repeats, where the sequence reads the same forward and backward. Nestled within these stretches of repeating DNA are genes. So the repeated DNA may safeguard important genes from replication mistakes and thereby preserve essential biological material, the researchers wrote in their paper.

The study did have limitations though; it looked at only a single representative for each primate species, and it couldn't say how much the Y chromosome would vary within animals of the same species, Pickett said.

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Mythbusting menopausal hormone therapy | InSight+ – MJA Insight

Posted: June 24, 2024 at 2:41 am

Despite advances in our understanding of the risks and benefits of menopausal hormone therapy, outdated research continues to raise concerns for both patients and their clinicians.

Menopausal hormone therapy (MHT) has seen dramatic perception shifts over the past 70 years. From initially being lauded for cardiovascular protection, studies in the early 2000s caused widespread concern by highlighting both increased cardiovascular and breast cancer risks.

However, recent research reveals a more nuanced perspective and how there are many health and lifestyle benefits to MHT (also known as hormone replacement therapy or HRT), especially when started earlier.

A research article published in Research, Fertility and Development outlined the metamorphosis of MHT and explained how the risks and benefits are not fully understood or communicated.

The history of menopausal hormone therapy

According to author Professor Bronwyn Stuckey from the University of Western Australia, MHT used to be seen as largely beneficial for cardiovascular risk reduction.

When you talk to patients, wed say its better for bones, its better for cardiovascular, it reduces weight gain, and the only downside is [the risk of] breast cancer. And then wed talk about ways to mitigate that, she said.

However in 2002, the Womens Health Initiative (WHI) study was released, which reported both an increase in breast cancer and an increase in cardiovascular disease in women on MHT.

Initially, the statement was made that the increased risks for cardiovascular disease and invasive breast cancer were present across age strata. Results are likely to be generally applicable to healthy women in this age range. Professor Stuckey wrote in her article.

There was a huge media hype at the time and it caused a shift in attitudes of both doctors and patients away from MHT.

Even one of the co-authors of the WHI study mentioned that the treatment of menopausal symptoms has been derailed by the interpretation and editorial [of the WHI study], she told InSight+.

When the WHI study came out, the first author said that there was risk for cardiovascular disease across all age strata, which is not a correct statement. When you actually look at the WHI findings, the only group in which there was a significantly increased risk of cardiovascular events was the women who were more than 20 years past the menopause, and who had continuous combined HRT, Professor Stuckey explained.

In 2007, the WHI investigators published a more nuanced analysis of their data, which revealed the difference between the oestrogen-only and the oestrogen plus progestogen (E + P) MHT, and the difference between starting early after menopause and starting later, Professor Stuckey wrote.

There have also been several more studies analysing the risks and benefits of HRT, the timing of HRT and the types of therapies.

Current MHT therapies

According to the Royal Australian and New Zealand College of Obstetricians and Gynaecologists councillor, Dr Anna Clare, this recent research, including a 15 year follow-up to the WHI study, that showed neither cardiovascular mortality nor all-cause mortality were increased by MHT, is reassuring to clinicians and their patients.

I think from the point of view of starting hormonal therapy around the time of menopause, I think practitioners would feel pretty confident to reassure women that youre not at increased risk of cardiovascular disease, and actually risk is probably reduced by MHT, she said.

However, Professor Stuckey highlighted that much of this research hasnt been integrated into practice partly because the Monthly Index of Medical Specialties (MIMS) directions havent caught up with the evidence.

She told InSight+ that this evidence includes:

1) That there are no stopping rules for MHT. It can continue for as long as there is an indication.

2) Oestrogen is just as effective as an anti-resorptive agent to prevent fractures as drugs like bisphosphonate and denosumab, and much more appropriate for the young woman.

3) That the so-called window of opportunity does not mean that one cannot start MHT after 10 years past the last menstrual period. It just means the cardiovascular benefit may not be as great, but the benefit for vasomotor symptoms and quality of life will be just as great.

When informing patients about post-menopausal health, clinicians should compare the risk of breast cancer to the greater risk of cardiovascular disease and the effect of MHT on preserving bone density and decreasing the risk of type 2 diabetes. However, most importantly, is its impact on quality of life.

There are an increasing number of menopausal women in the workplace, increasing number of menopausal women in leadership roles. And there are more women internationally who are championing this, Michelle Obama, Davina McCall. Theyre saying menopause is a real thing, Dr Clare highlighted.

This resurgence of speaking about the problem combined with compelling research shows other women that they dont have to suffer in silence.

For many years, women have suffered menopause symptoms, which can be pretty debilitating and can really affect quality of life. And now more people are less worried about treating the symptoms with HRT because we have robust safety data, Dr Clare said.

When prescribing HRT, Professor Stuckey said although there are a few rules, a personalised approach is crucial.

There are a few overall rules, like no uterus = no progestogen because of the lower risk of breast cancer and the greater cardiovascular benefit with oestrogen-only MHT. However, transdermal oestrogen is preferred for people with risk of venous thrombosis or who have had a venous thrombosis, or for women with migraine which is sensitive to hormonal fluxes. Women who are very close to their last menstrual period will do better with cyclical MHT rather than continuous combined.

One of my colleagues once described choosing MHT formulae or preparations as a bit like trying on a dress to see which one suits you. One formulation will not suit everyone, she concluded.

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Rod Stewart Helped Wife Penny Lancaster with ‘Inferno’ of Menopause – PEOPLE

Posted: June 24, 2024 at 2:41 am

Penny Lancaster is opening up about going through the menopause.

While speaking to The Times in an interview published Thursday, June 20, Lancaster, 53, discussed some of her symptoms and revealed how her husband Rod Stewart helped her cope after she was originally misdiagnosed with depression.

The model and TV personality, who was 49 when she first started experiencing menopause symptoms, recalled how just before the COVID-19 pandemic started in March 2020, she suddenly, "Found that one night after another, I was waking up, sweating head to toe."

She and Stewart, 79 who tied the knot in 2007 after starting dating in 1999 were at their home in Florida at the time.

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Lancaster, who thought she had COVID-19 at the time, explained, It wasnt just a little discomfort in the night. I was on top of the covers, feeling like this inferno was rising, as if I was standing in a pit of fire."

The heat started in my feet and rose up my legs. It was as if I could feel it going through my blood vessels, my blood was boiling. I could feel it rising, closer and closer to my heart and into my head," she went on.

"You think, How much hotter can I actually get? So Id wake up to the heat, to the fire bearing in mind we had air conditioning, it wasnt hot in the house find myself on top of the bedcovers, finally cooling down, and then, of course, waking up again, freezing cold because my temperature had settled, because my skin was damp and then cold with the air-con. Then back under the covers. And this was a vicious cycle for about four times a night, the policewoman added.

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At the time, Lancaster's fellow panelists on the U.K. talk show Loose Women suggested it might be menopause,but she thought she was too young.

At the end of March 2020, Lancaster, Stewart, and their two sons Alastair, 18, and Aiden, 13, returned to their U.K. home, which is when her symptoms got worse.

I had a few what I could classify as mental breakdowns, " she told the outlet, remembering how the family got chickens at the time and she became "the crazy chicken woman," hanging out in the animals' area.

One night at dinner, Lancaster revealed that father-of-eight Stewart and the two boys took a while to come down, and she ended up throwing the plates "across the kitchen."

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"I couldnt contain it anymore. I didnt want to hurt anyone. I didnt want to hurt myself. But I felt like it was getting to that point, she shared.

"I collapsed on the floor in a heap, burst into tears, shaking in the corner. Of course, it was an absolute shock to Rod and the boys. Rod just went, Boys, in the other room. Leave Mummy, " Lancaster said of her husband. "He came over and said, Its all right, darling. Its OK, its OK. I was shaking. I cant do this. What the hell is going on with me? I thought I was going mad. Rod said, Right, weve got to get you to the doctors. You cant carry on like this. Theres got to be an answer. Theres got to be something.

Lancaster said she was misdiagnosed with depression by one doctor, who gave her antidepressants The medication made Lancaster, who has no history of mental health issues, feel worse and just "numb," according to the outlet.

It was only when the Loose Women panelists performed an intervention and recommended Dr. Louise Newson a menopause specialist that she finally got some answers.

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I burst into tears because of the good news," Lancaster told the outlet. Thats what it was. Im not going mad. I can save my marriage. I can keep my family together. I thought everything was falling apart.

Lancaster said Stewart "dealt with it very well.

Dr. Newson eventually weaned her off the antidepressants and prescribed hormone replacement therapy (HRT) instead, with Lancaster saying she felt better "almost immediately."

"Within a week or two, I slept through the night and my bedsheets werent wet. And then I could function during the day, and then I was just uplifted. I had more energy, more confidence," she said. "I wanted to start exercising again. The chicken hammock went into retirement. Thats not there any more. But that was a moment, definitely an important time to reflect on and to be able to talk about, like I am.

According to the Mayo Clinic, the menopause is the time that marks the end of your menstrual cycles. Symptoms include mood changes, hot flashes, mood changes, sleep problems, weight gain and thinning hair.

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How to pay for individualized genetic medicines – Nature.com

Posted: June 24, 2024 at 2:40 am

Competing interests

J.M.Y.P. is an associate at Atlas Venture. J.V. is a co-founder of EveryONE Medicines and the N=1 Collaborative. W.X.Y. is a founder, employee and shareholder of Arbor Biotechnologies. A.W.L. reports personal investments in private biotechnology companies, biotechnology venture capital funds and mutual funds; is a co-founder and principal of QLS Advisors LLC, a healthcare investments advisor, and QLS Technologies LLC, a healthcare analytics and consulting company; a director of AbCellera, Annual Reviews, Atomwise, BridgeBio Pharma, Uncommon Cures and Vesalius Therapeutics; an advisor to Apricity Health, Aracari Bio, BrightEdge Impact Fund, Enable Medicine, FINRA, Health at Scale, MIT Proto Ventures, Quantile Health, Roivant Social Ventures, Swiss Finance Institute, Thals, Think Therapeutics and xCures; and during the most recent 6-year period has received speaking/consulting fees, honoraria, or other forms of compensation from AbCellera, AlphaSimplex, Annual Reviews, Apricity Health, Aracari Bio, Atomwise, Bernstein Fabozzi Jacobs Levy Award, BridgeBio, Cambridge Associates, CME, Enable Medicine, Journal of Investment Management, Lazard, MIT, New Frontier Advisors, Oppenheimer, Princeton University Press, Q Group, QLS Advisors, Quantile Health, Research Affiliates, Roivant, SalioGen Therapeutics, Swiss Finance Institute, Think Therapeutics, Vesalius Therapeutics and WW Norton. T.W.Y. has received research funding from EveryOne Medicines, has served as a scientific consultant to Biomarin and Servier Pharmaceuticals, is a board member of the Oligonucleotide Therapeutics Society and serves as a volunteer scientific advisor to several nonprofit rare disease foundations. J.M.Y.P., J.V., T.W.Y. and W.X.Y. are volunteers with the N=1 Collaborative, a non-profit organization.

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Sarepta Therapeutics Stock Soars on FDA Approval – sharewise

Posted: June 24, 2024 at 2:40 am

Sarepta Therapeutics (NASDAQ: SRPT) is leading the game in precision genetic medicine and biotechnology. Sarepta Therapeutics' stock price has witnessed a surge of over 40% after receiving expanded FDA approval for its Duchenne muscular dystrophy (DMD) gene therapy, Elevidys. This pivotal decision marks a turning point in treating this debilitating disease and underscores Sarepta's commitment to developing groundbreaking therapies for rare diseases.

Duchenne muscular dystrophy (DMD) is a severe genetic disorder characterized by progressive muscle degeneration and weakness. Primarily affecting boys, DMD is caused by mutations in the DMD gene, which provides instructions for creating dystrophin, a protein crucial for maintaining muscle cell structure and function. The absence of functional dystrophin leads to progressive muscle wasting, loss of mobility, and, ultimately, life-threatening complications. With an estimated incidence of 1 in 3,500 male births worldwide, DMD presents a significant unmet medical need, making the development of effective therapies an urgent global health priority.

Founded in 1980, Sarepta Therapeutics has emerged as a pioneer in genetic medicine, particularly in developing therapies for DMD and other rare neuromuscular disorders. Driven by a mission to engineer precision genetic medicines that transform patients' lives, Sarepta has built a diverse and robust pipeline of over 40 programs. The company's innovative approach is powered by its multi-platform Precision Genetic Medicine Engine, which encompasses cutting-edge gene therapy, RNA technology, and gene editing technologies. This strategic focus has enabled Sarepta to become a leader in developing targeted therapies for previously untreatable diseases.

Elevidys is a single-dose, adeno-associated virus (AAV)-based gene therapy administered via intravenous infusion. It addresses the underlying genetic cause of DMD by delivering a functional version of a shortened dystrophin gene (micro-dystrophin) directly into muscle cells. This innovative approach aims to enable the production of essential dystrophin protein, potentially slowing or halting the progression of muscle degeneration.

The FDA's recent decision to expand Elevidys' approval represents a significant milestone for Sarepta and the DMD community. Initially granted accelerated approval for ambulatory DMD patients aged 4 and 5 in 2023, Elevidys is now approved for all DMD patients aged four and above, regardless of ambulatory status. This expanded label encompasses two key distinctions: traditional approval for ambulatory patients and accelerated approval for non-ambulatory patients.

Based on robust clinical data demonstrating Elevidys' efficacy in improving muscle function in ambulatory patients, the traditional approval reinforces the therapy's value proposition. The accelerated approval, granted for non-ambulatory patients, recognizes the critical need for treatment options for this patient population, with continued approval contingent on confirmatory clinical trials.

Sarepta's earnings report for Q1 2024 highlights Elevidys's transformative impact on the company's growth trajectory. The company reported a 55% year-over-year increase in net product revenue, reaching $359.5 million, with Elevidys generating an impressive $133.9 million in net revenue during the quarter. Since its initial approval, Elevidys has achieved cumulative sales surpassing $334 million, surpassing the combined performance of other gene therapies approved in recent years.

Sarepta's financial performance is further reflected in its profitability, with GAAP earnings of $36.1 million and non-GAAP earnings of $78.2 million reported for Q1 2024. These positive financial indicators demonstrate Elevidys's significant commercial potential and underscore Sarepta's successful execution of its strategic vision.

In anticipation of increased demand for Elevidys, Sarepta has undertaken strategic initiatives to bolster its operational capabilities. The company recently announced hiring nine new employees, signaling its commitment to expanding its manufacturing, commercialization, and research activities. These strategic investments reflect Sarepta's proactive approach to ensuring it can meet the anticipated surge in demand for Elevidys following its expanded approval.

Sarepta Therapeutics presents a compelling investment opportunity for investors seeking exposure to the rapidly evolving field of genetic medicine and the pharmaceutical sector. The company's dominant position in the DMD market, driven by the expanded approval of Elevidys and its robust pipeline of innovative therapies, positions it for sustained growth. Sarepta's strong financial performance, including impressive revenue growth and profitability, further strengthens its investment appeal.

However, investors should carefully consider the inherent risks associated with biotech investments. Regulatory hurdles, competition within the gene therapy landscape, and the volatility inherent to the industry are factors that could impact Sarepta's future performance.

The FDA's expanded approval of Elevidys marks a pivotal moment for Sarepta Therapeutics and the DMD community. The company's commitment to developing groundbreaking therapies positions it as a frontrunner in the race to conquer rare diseases. As Sarepta continues to innovate and expand access to its life-changing therapies, it holds the potential to deliver substantial value to patients and investors.

Source MarketBeat

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Sarepta Therapeutics Stock Soars on FDA Approval - sharewise

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