Genetic Testing Market Overview

According to the report, the global genetic testing market was valued for$15.5 Billionin 2022 and is estimated to reach$40.9 Billionby 2032, exhibiting a CAGR of 10.2% from 2023 to 2032.

CAGR: 10.2% Current Market Size: USD 40.9 Billion Forecast Growing Region: APAC Largest Market: North America Projection Time: 2023- 2032 Base Year: 2023

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Genetic Testing Market Drivers

The increasing prevalence of genetic disorders like Down syndrome, sickle cell disease, phenylketonuria, and others, along with the rising adoption of genetic testing for cancer diagnosis, is expected to drive market growth. However, the high costs associated with genetic tests may impede market expansion. Conversely, the market is expected to witness growth due to the significant presence of manufacturers producing genetic testing devices and growing public awareness about the benefits of genetic testing during the forecast period.

The molecular testing segment to maintain its leadership status throughout the forecast period.

In 2022, the molecular testing segment dominated the global genetic testing market, capturing over half of the revenue, and is expected to remain at the forefront throughout the forecast period. This is primarily due to the widespread adoption of molecular testing for diagnosing genetic diseases. Conversely, the cytogenetic testing segment is forecasted to achieve a notable compound annual growth rate (CAGR) of 10.8% from 2023 to 2032.

North Americato maintain its dominance by 2032.

In 2022, North America led the global genetic testing market in revenue, capturing over two-fifths of the total revenue. This is attributed to the significant presence of genetic testing device manufacturers in the region and the increasing prevalence of chronic diseases such as cancer that require genetic testing.

However, the Asia-Pacific region is anticipated to experience the fastest compound annual growth rate (CAGR) of 10.9% from 2023 to 2032, likely becoming the dominant market during the forecast period. This growth can be attributed to the rising population affected by cancer and the growing awareness of genetic testing in the region.

Leading Market Players:

Recent Developments in the Genetic Testing Market

In November 2022, Myriad Genetics, Inc., a leader in genetic testing and precision medicine, announced that it has acquired Gateway Genomics, LLC, a personal genomics company and developer of consumer genetic tests including the No. 1 selling SneakPeek Early Gender DNA Test. SneakPeek reveals a babys gender at six weeks into pregnancy, the only at-home test to do so with 99% accuracy and the earliest method available.

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Genetic Testing Market to Generate USD 40.9 Billion by 2032, 10.2% (CAGR ) Annual Growth Predicted D - PharmiWeb.com

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Ever wondered which breed your dog really is or how your cat's dental health is going? Wonder no more thanks to these great deals on pet DNA kits during the last day of Amazon Pet Day.

Best pet DNA kits at Amazon Pet Day

Best pet DNA kit for dogs

Best dog DNA kit for fast results

Best dog DNA kit for locating relatives

Knowing your dog or cat's genetic makeup might initially seem like just scratching a curiosity itch, but the knowledge they reveal can be useful. Genetic testing for your pets can give you insights into their behavior, common health issues of their specific breed, and introduce you to their relatives. If any of this piques your interest, Amazon Pet Day has some great deals on pet DNA kits for both cats and dogs.

Hurry to grab these deals because Amazon Pet Day ends tonight at 11:59 p.m. PT.

Here are the best pet DNA kit deals we've found so far:

The AncestryDNA dog breed identification kit is simple to use: collect a saliva sample, pack it up, and send it off. In about two to six weeks, you'll get detailed online results that give specifics about your dog's genetic makeup. Ancestry will list which breed (or breeds) are present in your dog with percentages of each. These details can help explain not only physical characteristics but personality traits and common behaviors, too.

Mashable Top Stories

While swabbing your cat's gums and teeth might not sound like the best day ever, the results are worth the effort. The Basepaws cat DNA test comes back with results that'll indicate your cat's breed (or breeds), as well as common traits. Basepaws scans for over 21 breeds and 25 genetic traits.

Another benefit is the medical read-out that comes with the analysis. Basepaws screens for over 40 genetic diseases and gives you an oral health report. Both of these elements can help with early detection of potential future health issues and clue you in on the mouth health of your pet.

With a return time between two and four weeks, the Embark Breed & Health dog DNA test can be a great option if you're looking for fast results. Embark developed the test in partnership with Cornell University College of Veterinary Medicine to use research-grade genotyping. The test scans for relations to over 350 dog breeds and even includes dingoes, coyotes, and wolves.

The DNA test also looks at over 250 genetic health conditions to help you and your vet better care for your dog's needs. And if you have questions about the results, check in with one of Embark's vets or genetic experts.

Finding out a distant cousin lives in Paris can be a pretty exciting discovery. Finding your dog's sibling lives one town over is arguably even more exciting. The Wisdom Panel Breed Discovery dog DNA kit scans a large breed database and then allows you to see photos, compare genetic testing results, and even send messages (imagine the playdate possibilities). Wisdom Panel says the company has found relative matches for over 99.9% of the 3 million dogs they've tested.

The test also gives a clear breakdown of breeds present and tests for medical sensitivities, which can let you know if your dog is more likely to have a reaction to certain medications.

Read more here:
Save up to $60 on a pet DNA kit during the final hours of Amazon Pet Day - Mashable

Recommendation and review posted by Bethany Smith

Pictured: 3D illustration of the CRISPR-Cas9 system editing a stretch of DNA/iStock,Meletios Verras

Excision BioTherapeutics attempt to use a CRISPR-based gene editing therapy to cure HIV has failed an early-stage study, according to several media reports on Friday.

Results from the Phase I trial of five patients showed that Excisions CRISPR therapeutic did not strongly suppress the HIV virus. Three patients who were taken off of antiretroviral therapy soon developed viral rebound and needed to resume conventional treatments, according to reporting by STAT News.

Still, Excisions approach did show signs of promise. One patient was able to keep the virus at bay for 16 weeks after stopping antiretroviral treatment before rebounding. Typically, HIV levels resurge after around four weeks. The experimental CRISPR treatment also appears to have lowered the number of infected cells in this patient, according to STAT.

In response to the disappointing outcome, CEO Daniel Dornbusch told STAT that Excision is encouraged by the safety data in the Phase I study, which suggests that in-vivo CRISPR can be done in a safe way in humans. The company will use these findings to work on a new gene editing candidate that it can apply to other chronic viral infections, such as herpes and hepatitis B.

According to its website, Excisions lead candidate is EBT-101, a CRISPR-based gene editor that works by cutting out the HIV pro-viral DNA from all infected cells. The candidate is delivered via an AAV9 vector and uses two guide RNAs that can home in on three target sites in the HIV genome, minimizing the likelihood of viral escape.

The company is positioning EBT-101 as a potentially curative treatment for chronic HIV and is rapidly advancing toward clinical trials.

In October 2023, Excision released safety data from its first-in-human Phase I/II study of EBT-101, showing that there were no serious adverse events or dose-limiting toxicities in all three patients that had been dosed with the gene editor at the time. The study found four mild toxicities that could be potentially related to EBT-101, though all were resolved without intervention.

The early data also showed that EBT-101 was present at detectable levels in the blood four weeks after treatment.

In addition to targeting HIV, Excision is also leveraging its proprietary CRISPR-based gene-editing platform against herpes 1 and 2, for which it is advancing EBT-104, and hepatitis B, for which it is developing EBT-107.

Several biotech companies have responded to the HIV crisis, including industry powerhouses Gilead and GSK. Gilead owns the oral antiretroviral pill Biktarvy (bictegravir/emtricitabine/tenofovir alafenamide), while GSK and partner ViiV Healthcare market the oral pill Tivicay (dolutegravir) and long-acting injectable Cabenuva (cabotegravir/rilpivirine).

While these virus-suppressive treatments are effective, a true cure remains elusive and the field continues to face several substantial challenges. In March 2024, the World Health Organization warned of a growing virus resistance against Tivicay.

Meanwhile, gene therapies which have strong curative potential still pose potential safety concerns, particularly unintended edits and off-target effects.

Tristan Manalac is an independent science writer based in Metro Manila, Philippines. Reach out to him on LinkedIn or email him at tristan@tristanmanalac.com or tristan.manalac@biospace.com.

Excerpt from:
Excisions CRISPR-Based HIV Treatment Fails to Show Curative Potential in Early Study - BioSpace

Recommendation and review posted by Bethany Smith

BALTIMORE An ambitious effort to cure HIV with CRISPR genome editing fell short in an early clinical trial, investigators announced Friday morning.

In the study, run by Excision BioTherapeutics, researchers tried to use the gene editing tool to address a chief reason HIV has been so hard to cure. While antiviral drugs can clear patients of replicating virus, HIV is able to worm its way into a patients own DNA in certain cells. If the patient ever stops taking medicines, those cells start pumping out HIV particles and the infection roars back.

Researchers hoped they could send CRISPR to those cells and, by cutting the HIV DNA lurking there at two spots, slice out the virus. In the Phase 1 trial, investigators administered the treatment to five patients. They then took three of them off conventional antiviral treatment.

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Trial using CRISPR to edit HIV out of cells disappoints - STAT - STAT

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Cell culture

MT4CCR5 cells are a human CD4+ T cell-line modified to stably express CCR5 co-receptor after being transduced with a lentiviral vector expressing human CCR5 under the control of SFFV promoter. These cells are susceptible to both R5-and X4-tropic HIV-1. The MT4CCR5 cells were kindly provided by Dr. Koki Morizono (UCLA, Los Angeles). These cells were cultured in RPMI-1640 medium (Gibco) supplemented with 10% fetal bovine serum (Gibco), 2mM L-glutamine, 100 units penicillin, and 100g/ml streptomycin (Gibco). The cell cultures were maintained in a humidified incubator at 37C and 5% CO2. HEK293T cell line (Clontech) was used for viral production and lentiviral titer. The cells were cultured in Dulbeccos Modification of Eagles Medium (Hyclone) with the same supplements as MT4CCR5 cells.

The third-generation lentiviral vector, pLVX-AcGFP1-N1 (Clontech, Cat# 632154), was used as the backbone vector to transfer the C46 HIV-1 fusion inhibitor gene into the target cells. The C46 C-peptide fusion inhibitor sequence obtained from the publication of Felix G. Hermann et al., 200950 were gene synthesized and cloned into pUC57 plasmid (Integrated DNA Technologies, Inc). The C46 HIV-1 fusion inhibitor sequence was designed to flank XhoI and EcoRI restriction sites to clone in the lentiviral vector, resulting pLVX-C46-AcGFP1. The pLVX-C46 plasmid was cloned by amplifying C46 HIV-1 fusion inhibitor gene without the expression of AcGFP1 from the synthesized pUC57 plasmid with the forward primer having the XhoI restriction site and the reverse primer adding a stop codon before the EcoRI restriction site. All construct lentiviral plasmids were confirmed to contain the C46 HIV-1 fusion inhibitor by restriction enzyme digestion and DNA sequencing.

VSVG-pseudotyped lentiviral vector particles were produced in HEK293T cells, using four separate plasmids and the calcium phosphate transfection method, as previously described51,52,53. HEK293T cells were seeded on 10-cm cell culture dishes, and co-transfected with the construct lentiviral vector with the third-generation packaging plasmid system (Addgene plasmid #12251 (pMDLg/pRRE), #12253 (pRSVREV), #12259 (pMD2.G). Vector particles were harvested from the culture supernatant collected at 24 and 48h. The viral vector titers, determined by infection of HEK293T cells with serial dilution of the samples, were expressed as the percentage of AcGFP1+positive cells evaluated by flow cytometry.

MT4CCR5 cells and MT4CCR5 CRISPR/Cas9 knockout CCR5 cells (1106) were transduced with lentiviral vectors at a multiplicity of infection (MOI) of 0.1 with 8g/ml polybrene for 24h. The transduced cells were seeded in a 6-well plate and incubated at 37C for 3days before determining the percentage of transduced cells by flow cytometry. The transduced cells were enriched for those containing the lentiviral vectors by treating them with 1g/ml puromycin (SigmaAldrich) in culture medium and refreshing the medium every 34days.

3xNLS-SpCas9 plasmid (Addgene plasmid#114365), was transformed into ClearColi BL21(DE3) E.coli cells for protein expression. The recombinant protein was expressed and purified as previously published54. In detail, the cells were cultured in LuriaBertani medium supplemented with 100g/ml of ampicillin at 37C until the cell density reached 0.6 at OD600 nm. Subsequently, protein expression was induced by adding 0.4mM isopropyl--D-thiogalactopyranoside (IPTG), and the culture was maintained at 18C in a shaking incubator for 24h. After induction, cells were centrifuged, and the cell pellets were stored at20C. To purify the Cas9 protein, the cell pellets were lysed in a buffer containing 20mM TrisHCl pH 7.5, 200mM NaCl, 0.5mg/ml lysozyme, and 1mM DTT by incubating on ice for 10min. The cells were then physically lysed by sonication and centrifuged at 15,000rpm for 1h at 4C. The supernatant was applied onto the first 1ml of HisTrap FF prepacked column (Cytiva). After extensive washing with binding buffer (20mM TrisHCl pH 7.5, 200mM NaCl) and binding buffer containing 20mM Imidazole for 5ml, the partially purified Cas9 protein was eluted with a buffer containing 200mM imidazole. For the second purification step, hydrophobic interaction chromatography (HIC) column (phenyl sepharose HP) was utilized. The HIC column was pre-equilibrated with 20mM TrisHCl pH 7.5, 200mM NaCl, and 2M NaCl buffer. The elution sample from the first column was also supplemented with salt up to 2M to promote protein binding. After applying the sample onto the column, Cas9 protein was eluted with a buffer containing 20mM TrisHCl pH 7.5, 200mM NaCl, and 1M NaCl. Finally, the Cas9 protein was further purified using gel filtration chromatography (superdex 200 increase 10/300 GL) as a third purification step. The purity of the final protein sample was evaluated by SDS-PAGE. The protein concentration was determined by the Bradford method using BSA as protein standard.

The sgRNA1# sequences, 5-ACTATGCTGCCGCCCAGT-3; the sgRNA2# sequence 5-CAGAAGGGGACAGTAAG-314. The sgRNAs targeting to CCR5 gene were synthesized with chemically modified nucleotides at the terminal positions at both the 5 and 3 ends were purchased from Integrated DNA Technologies, Inc. Ribonucleoprotein (RNP) complex was made by incubating 6g (36.81pmol) or 10g (61.35pmol) of the in-house purified Cas9 protein, 2g (61.86pmol) or 4g (123.72pmol) of sgRNA#1 or sgRNA#2 at room temperature for 20min. Resuspended 1106 cells in 20l of SF cell line nucleofector solution, mixed with the RNP complex, and transferred into the Nucleocuvette (Lonza), program DC100. The nucleotransfection protocol was followed according to the manufacturers recommendations (Amaxa 4D-Nucleofector, Lonza).

Cells were stained with monoclonal antibodies against human CCR5 (2D7: PECy7 labeled, eBioscience, Cat#557752), human CXCR4 (12G5: APC labeled, Biolegend, Cat#306510), human CD4 (OKT4: PE labeled, Biolegend, Cat#317410), according to the manufacturers instructions. C46 HIV-1 fusion inhibitor expression was measured by staining with 0.2g of anti-HIV-1 gp41 Monoclonal (2F5) (NIH AIDS Reagent Program, Cat#1475) followed by 50l of 1:500 of the secondary antibody (PE-anti-human Fc, Jackson Immunoresearch, cat#109-115-098). Isotype control antibodies were included with mouse IgG2b control-PE (ImmunoTools, Cat#21275534), mouse IgG1 control-APC (ImmunoTool, Cat#2185016), and mouse IgG1 control-PECy7 (Biolegand, Cat#400126). CCR5, CXCR4, CD4, C46 HIV-1 fusion inhibitor, and AcGFP1 expression were measured by flow cytometry using the CytoFLEX Flow Cytometer (Beckman Coulter). A live cell population defined by 7AAD- staining (Biolegend, cat#420404) was subjected to single-cell population analysis for each antibody staining. Additionally, a positive control for dead cells was established by exposing MT4CCR5 cells to heat at 56C for 30min before staining and determined by the 7AAD+ population. Data analysis for gene expression was performed with FlowJo version 10 software (BD Biosciences).

The genomic DNA was isolated from cells using Invitrogen Purelink Genomic DNA Mini Kit (Invitrogen, cat#K182001) according to the manufacturers instructions. 25ng of genomic DNA per reaction was validated with real-time PCR reactions set up in duplicate in 25l reaction volumes using Agilent Brilliant II master mix (Invitrogen) in a 96-well plate on CFX Connect Thermal Cycler platform (Bio-Rad). Taqman primer and probe sequences for this assay were as follows: C46 probe: 5 6-FAM/CA CTC CAC G/ZEN/C AGC ACT TCC GCT CG/IABkFQ 3, C46 forward primer: 5 CAC AGC CTG ATC GAG GAG AG 3, C46 reverse primer: 5 GTC CTG CCA CTG GTG GTG 3, -globin probe: 5 HEX/CT CCT GAG GAG AAG TCT GCC GTT ACT GCC /BHQ-2 3, -Globin forward primer: 5 CAA CCT CAA ACA GAC ACC ATG G 3, -Globin reverse primer: 5 TCC ACG TTC ACC TTG CCC 3. Thermocycling conditions were 50C 2min, 95C 10min, 40(95C 15s; 60C 1min)18.

Cells were washed with ice-cold PBS and lysed in radioimmunoprecipitation assay (RIPA) lysis buffer (Thermo-Fisher Scientific). The total protein concentration in the cell lysates was measured using the bicinchoninic acid assay (Thermo-Fisher Scientific). Subsequently, 30g of total protein was separated by SDS-PAGE and transferred onto a PVDF membrane (Thermo-Fisher Scientific). Blocking was carried out using 5% skim milk (Sigma) in 0.05% PBST for 24h, followed by overnight incubation with the primary antibody (anti-CCR5 antibody; CUSABIO TECHNOLOGY, cat#CSB-PA006994). After washing the blots five times with 0.05% PBST, they were probed with the secondary antibody (Goat pAb to Rb IgG (HRP); AbCam, cat#ab205718) for 1h and subsequently detected using ECL Western Blotting Substrate (Bio-Rad, cat#1705060) and scanned using the Odyssey InfraRed Imaging System (LI-COR BioSciences, Lincoln, NE). The blots were then stripped with mild stripping buffer (Thermo-Fisher Scientific), re-blocked, and re-probed with anti--Actin antibody (Abcam, cat#ab8227).

Genomic DNA was extracted from cells using Invitrogen Purelink Genomic DNA Mini Kit (Invitrogen, cat#K182001) according to the manufacturers instructions. The target sequence was amplified using the Phusion high-fidelity DNA polymerase (Thermo Scientific, cat#F-530XL) and XhoI-CCR5 forward primers 5-TGGACAGGGAAGCTAGCAGCAAA-3 and EcoRI-CCR5 reverse primer 5-TCACCACCCCAAAGG TGACCG-3. The PCR products were annealed after purification. Next, 2l of hybridized DNA were subjected to digestion with 0.5L T7EI (New England Biolabs) in NE Buffer 2 for 30min at 37C. Subsequently, the samples were loaded onto a 2% agarose gel electrophoresis with an equal amount of PCR product controls from non-edited samples.

R5-tropic HIV-1BaL virus and X4-tropic HIV-1NL4-3 virus were produced by transient transfection of pWT/BaL plasmid (NIH AIDS Reagent Program, cat#11414) or pNL4-3 plasmid (NIH AIDS Reagent Program, cat#114) into HEK293T cells. Monolayers of HEK293T cells (5106 cells per 10-cm dishes) were transfected with 10g of each plasmid using a calcium phosphate transfection method51,52,53. After 8h, the transfection mixture was withdrawn, replaced by 10ml of 10% FBS in DMEM medium. The transfected cells were incubated for 24h. HIV-1 viruses were harvested from the culture supernatants and filtered through sterile syringe filters with a 0.45-m pore size (Merck Millipore). HIV-1 samples were aliquoted and kept at80C. The virus titer was determined for the HIV viral load using the COBAS AMPLICOR HIV-1 Monitortest (version 1.5; Roche Molecular Systems, Branchburg, NJ).

The 1106 cells were incubated with HIV-1 at MOI of 1 and 10 for 16h. The cells were then washed three times with serum-free medium and resuspended in fresh growth medium. The infected cells were split into half at 3-day intervals, to maintain a cell density of approximately 106cells/ml. HIV-1 replication was monitored in culture supernatants, using HIV-1 p24 Simple Step ELISA kit (Abcam, cat#ab218268) and viral load assay, as described above. The cell pellets were kept determining cell viability by 7AAD staining (Biolegend, cat#420404) and flow cytometry.

Data was obtained from triplicate experiments (n=3). The data were analyzed, and standard deviations (SD) were calculated using the Prism 7 (GraphPad) statistical software program. Unpaired t-tests with Welchs correction were used to calculate p values, and p<0.05 was considered statistically significant.

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CRISPR/Cas9 genome editing of CCR5 combined with C46 HIV-1 fusion inhibitor for cellular resistant to R5 and X4 ... - Nature.com

Recommendation and review posted by Bethany Smith

Genome editing has been successfully used to treat liver disease in fetal monkeys while still in the womb.

A collaborative team led by Dr William Peranteau from the Children's Hospital of Philadelphia and Professor Kiran Musunuru from the University of Pennsylvania, Philadelphia, has successfully used base editing, a form of CRISPR/Cas9 genome editing whereby only a single DNA base is changed, to treat hereditary liver disease in fetal monkeys.

'Genetic diseases affecting the liver, including metabolic liver diseases, are some diseases that may benefit from in utero editing,' the researchers explained while presenting their work at the 27th Annual Meeting of the American Society of Gene and Cell Therapy in Baltimore, Maryland. 'This body of work presents evidence that a one-time injection is enough to fix a broken gene by editing it to correct a disease-causing mutation.'

In this study, the researchers used base editing to treat hereditary tyrosinemia type one (HT1), a condition that also affects humans. This liver disease is caused by a mutation in the Fah gene, leading to a buildup of toxic byproducts that cause severe damage to the liver.

Base editing avoids the risks associated with the double-strand breaks created in traditional CRISPR techniques, which can lead to unpredictable edits and higher levels of cellular toxicity (see BioNews 1217 and 1091). Currently, this disease is managed with nitisinone, a medication that blocks the HPD enzyme. This enzyme acts upstream of Fah and blocks the pathway that leads to the production of toxic byproducts, thereby preventing damage to the liver.

The researchers also targeted the HPD enzyme, by disabling the HPD enzyme-coding gene. Previously, the they had success using this approach in fetal mice while still in the womb (see BioNews 971). In the current study, the researchers have tested the feasibility of in utero base editing in crab-eating macaques, which provide a much closer genetic model to humans, allowing researchers to refine the approach for potential future therapies.

To test the efficacy of the approach in utero, the researchers also delivered the base-editing injection to three-year-old crab-eating macaques. They noted that the editing levels were approximately 2- to 4.5-fold lower compared to fetal monkeys, which highlights the potential advantages to editing during fetal development.

'We also find that delivering our injection earlier in life matters, and improves how well we can edit the disease gene. With this work, we hope to pave the way to one-day offering patients these types of one-time injections to [treat] diseases caused by genetic mutations,' the researchers said at the Meeting.

Although HT1 is a rare disease affecting roughly one in 100,000 individuals and treatable with nitisinone for most patients, this research acts as a proof-of-concept to treat a wide range of congenital diseases before birth using CRISPR base editing.

However, despite the promising results, significant hurdles remain before this technique can be applied safely and effectively in humans.

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CRISPR base editing treats liver disease in fetal monkeys | PET - BioNews

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Plans for Australias first cryonic storage facility have been set for southern New South Wales.

The Cryonics Centre would freeze bodies in the hope they will be revived years, decades or even centuries later.

A site has been scouted at Holbrook and is now waiting council approval.

Matt Fisher from Stasis Systems said freezing is a detailed process.

"We have cryoprotectants that essentially prevent the formation of ice crystals within the body during the freezing process."

If it goes ahead, the Cryonics Centre will be the first in the Southern Hemisphere.

Gavin Smith is one of many prospective customers already warming to the idea.

"The bits that make me me are here and they don't die immediately so if there's a way to hit pause, I'd love to do that, he told 7 News.

The process would cost around $80,000, possibly from your life insurance, to freeze and preserve your dead body.

But the method does have strong skeptics.

Bosch Professor of Histology Chris Murphy told 7 News cryonic preservation is pointless.

"When we use cryoprotectants we use them to preserve tissue for looking at in the microscope, not for bringing it back to life... the structure is still dead, he said.

At this point in time, scientists agree that cryogenics is not possible.

Supporters of the procedure, however, still have high hopes, holding faith in future technologies that will fill the gaps in our knowledge.

Mr Fisher said he does not doubt it will be a possibility one day.

"When you're talking about what happens in a thousand years or ten thousand yearsit's really difficult to put upper limits on what's possible, he said.

He hopes the centre will open next year.

News break March 3

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Cryonics Centre planned for southern NSW - Yahoo New Zealand News

Recommendation and review posted by Bethany Smith

The French new space in-orbit services company Infinite Orbits raised a 12 million round led by Newfund Capital along with the EIC Fund, IRDI Capital Investissement and Space Founders France. The main objective of this fundraising is enabling Infinite Orbits to accelerate the development of its main project Endurance, a Life Extension satellite for space assets.

Endurance will empower satellite operators to optimize their fleet management strategies while increasing their return on investment through adding years to the life of their satellites. Endurance is planned to demonstrate the first European Life Extension mission in 2026.

Last year, we already achieved a major step: we are the first startup in the world to have a commercial nanosatellite in geostationary orbit, we formed a key alliance with satellite operator Intelsat to develop services for its fleet. With four major clients, including Azercosmos, Intelsat, CNES and Hispasat, the Company has closed its second year of revenues and is on a strong growth path to cross the 60 million figure in 2027, explained Adel Haddoud, CEO of Infinite Orbits. And as a commercially driven company, the next logical step for us is to offer all GEO operators new reliable and competitive services such as Life Extension.

Founded in 2017, Infinite Orbits Endurance will be operated by Infinite Orbits RPO technology (Rendezvous and Proximity Operations), an autonomous vision based navigation technology that allows assets in space to approach and dock to one another. RPO technology will pave the way towards a sustainable utilization of space resources, allowing for a durable space environment. The technology is designed to be replicated for advanced in-orbit services, including close inspection, re-fueling, in-space manufacturing and many more applications yet to materialize following the spectacular growth of satellites launched in recent years.

The verification and validation roadmap of Infinite Orbits technology started in 2019 with key technology partnerships including ESA & CNES and with funding from the European Innovation Council (EIC), bpifrance, La Region Occitanie. This roadmap has led to the launch of Infinite Orbits Orbit Guard #1 to GEO orbit on May 1st 2023, carrying the first version of Infinite Orbits technology.

We are thrilled to partner with a company like Infinite Orbits, perfectly embodying our commitment to backing deeptech ventures with the potential to lead globally, said Salim Hassad, Investment Director at Newfund. Infinite Orbits stands out with its exceptional founding team, robust technological foundation, and promising commercial trajectory. We are confident that Infinite Orbits will not only excel in the in-orbit servicing sector but also emerge as a future international champion.

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Toulouse-based space tech Infinite Orbits bags 12 million to build first life extension satellite - EU-Startups

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Top OB/GYN Is on a Mission to Impact the Lives of Women

ATLANTA, GA / ACCESSWIRE / May 9, 2024 / May is Women's Health Month, including Women's Health Week from May 12-18. This annual observance is designed to focus attention on the health issues facing women. LaReesa Ferdinand, MD., shares some information about unique products that are available to promote better health outcomes. Dr. Ferdinand believes women should take time to focus more on their own personal health and needs. The U.S. Department of Health and Human Services points out that heart disease, breast cancer, osteoporosis, depression, and autoimmune diseases are the top five health issues facing women.

RECOMMEND SOME SUPPLEMENTS

For many patients, supplements can work wonders. One that's highly recommended is Life Extension's FLORASSIST Probiotic Women's Health, an oral probiotic supplement designed with women's vaginal, digestive and immune health in mind. Probiotics play an important role in women's health by providing healthy bacteria. FLORASSIST promotes healthy vaginal flora, pH levels and tissue health, while encouraging digestive comfort and improving immune response. Get it for under $25. For more information, visit http://www.lifeextension.com

HOW IMPORTANT IS REGULAR EXERCISE

Exercise and good self-care are key to maintaining mental and physical health, and this is especially true for women. For this, try Arnicare Gel. It is scientifically formulated with Arnica and provides relief from everyday muscle aches and pains, swelling from injuries and discoloration from bruises making it an essential part of a pain-free active lifestyle. The fragrance-free formula is quickly absorbed into the skin. Get Arnicare at Walmart and Walgreens. For more information, visit http://www.arnicare.com

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Women's Health Expert Dr. LaReesa Ferdinand Shares Tips to Kick Start Health and Wellness on TipsOnTv - AccessWire

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Although no firm decision has been taken yet by the Army, EDR On-Line understands that the Army should take a decision soon. These vehicles have been in service for over 20 years and definitely need refurbishment.

No modification were made to the powerpack, although both the engine, a Detroit Diesel 6V53T providing 300 hp, and the transmission, an Allison X200-4 with four forward and one reverse gears, were fully refurbished bringing them to zero hour. Some sensors were added, which allow the driver to see on the new Driver Vision Enhancement system numerous data that were not available in the past, such as rounds per minute, speed, fuel status and battery status. No modification were made on the protection, although the company does not exclude the adoption of a spall liner. This, together with the maintaining of the single-pin tracks, will allow the 14 tonnes tracked vehicle to maintain its swimming capability.

To improve crew safety and vehicle survivability a new generation fire suppression system that uses FM-200 gas in place of Freon; the system in the engine compartment provides a double shot, the same being for that in the cabin and troop compartment, personnel being also able to activate the fire suppression system manually. LED lighting replaced conventional one both externally and internally.

The firepower will be improved. Two options are proposed, the first retaining the FNSS one-man Sharpshooter stabilised turret, armed with a 25 mm cannon and a 7.62 mm coaxial machine gun, and upgrading it adding a new gunners sight, a thermal imager, both from Thales Defense & Security, a laser rangefinder, and a new fire control system. The second option is to replace the existing turret with the Saber unmanned turret, also from FNSS, which is already fitted with all those systems and can host a 12.7 machine gun or a 40 mm automatic grenade launcher as secondary armament.

The Adnan features four sets of four 76 mm grenade launchers at each corner of the vehicle, while 8 grenades are located on the turret, in two clusters of three plus one tubes each located at the rear sides of the turret. Deftech replaced the grenades fire control box with an upgraded one that allows to understand if the tubes are loaded or not, improving considerably survivability chances. The launchers are interfaced through the new Battle Management System (BMS) Light with the new Laser Warning Receiver, produced by Deftech, which allows smoke grenades to be launched automatically, semi-automatically or manually. The BMS also includes a Blue Force Tracking system. The whole vehicle wiring has been replaced, allowing among other the installation of a 360 situational awareness system, front and rear sensors being both daylight and thermal. The radio suite has also been replaced by new comms in the VHF and HF bands, all software defined.

These allow the ACV-300 Adnan to swap data with other Malaysian Army vehicles, such as the AV-8 Gempita 88 armoured wheeled combat vehicle, as well as with the SR-10 VTOL unmanned air system. Electrically powered, it has a 2.2 metres wingspan and in horizontal flight is propulsed by a two-blade propeller activated by a small 1.34 hp petrol engine; for vertical take-off and landing the lift is provided by four rotors activated by electric motors. The UAS has an endurance of 90 minutes, a range of 20 km, and a maximum speed of 40 knots. Its SR-10 take-off mass is 9 kg, and its payload is a gimballed optronic sensor installed in the nose. It will represent the forward eyes of the Adnan.

In August 2023 the ACV-300 demonstrator has been tested on and off road, logging over 700 km, and test fired both static and on the move. EDR On-Line understands the budget is allocated, the company awaiting the updated requirement document to finalise the proposal, that will then lead to the selection of one of the two contenders. While the programme aims initially at upgrading only the ACV-300 in the AIFV configuration, which can carry the driver, commander, gunner and eight dismounts, the programme might extend to the remaining versions of the ACV-300 in service with the forces.

Photos by P.Valpolini

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DSA 2024 - Deftech exhibits the proof of concet vehicle of the ACV-300 life extension programme proposal for the ... - EDR Magazine

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EBT-101, a CRISPR-based gene-editing therapy from Excision BioTherapeutics, was safe and well tolerated in a Phase I study, but it did not prevent viral rebound in the first three participants who stopped antiretroviral treatment, according to a presentation last week at the American Society of Gene & Cell Therapy (ASGCT) annual meeting.

Excision put a positive spin on the findings, noting that favorable safety data is a necessary step on the path to developing therapies for latent viral infections. The company also touted promising early results from studies of CRISPR-based therapies for herpes simplex virus and hepatitis B. But the HIV rebound news is disappointing, and it underscores the importance of remaining wary of exaggerated claims from industry and the mainstream press about the state of HIV cure research.

Initial data from the EBT-101-001 trial provides important clinical evidence thatagene editingtreatment modality can be safely delivered fortargetingthe HIV DNA reservoirs in human cells, study investigator Rachel Presti, MD, PhD, of Washington University St. Louis School of Medicine, said in a news release. This study provides researchers with invaluable insights for how CRISPR technology can be applied for addressing infectious disease and was an important first step towards additional programs designed to optimize this treatment modality for treating the millions of individuals who are impacted by HIV and other infectious disease.

Antiretroviral therapy can keep HIV replication suppressed indefinitely, but the virus inserts its genetic blueprints into the DNA of human cells and establishes a long-lasting reservoir that the drugs cant reach. This integrated HIV DNA lies dormant in resting T cells during treatment, but it can start churning out new virus when antiretrovirals are stopped, making a cure nearly impossible. The only way to tell whether an intervention leads to long-term remission is to discontinue antiretroviral therapy with careful monitoring, known as an analytic treatment interruption.

Kamel Khalili, PhD, of Temple University, and colleagues have been studying gene therapy to cure HIV for more than a decade. In 2014, they reported that a CRISPR-Cas9 tool could cut out a segment of integrated HIV DNA necessary for viral replication. A study published in 2019 showed that this approach could remove integrated HIV genes and clear latent viral reservoirs in mice.

This led to the development of EBT-101, a CRISPR-based therapy delivered by an adeno-associated virus that uses dual guide RNAs to target three sites on the integrated HIV genome. Making cuts at these locations prevents the production of intact virus. Last August, researchers reported that a single dose of a simian version of the therapy safely and effectively removed an HIV-like virus from viral reservoirs in monkeys on antiretroviral therapy, but this study did not include a treatment interruption.

The first human clinical trial of EBT-101 (NCT05144386) started in 2022, enrolling people on antiretroviral therapy with a stable undetectable viral load. The study protocol called for participants who maintained viral suppression at 12 weeks after receiving the gene therapy to undergo an analytic treatment interruption.

At the European Society for Gene & Cell Therapy annual meeting last October, Presti reported that EBT-101 was detectable in the blood of the first three treated participants after a single IV infusion at the initial dose level. EBT-101 was well tolerated with only mild temporary side effects. She did not present treatment interruption outcomes, but that didnt stop the Daily Mail from proclaiming that a cure for HIV could be months away.

Presti gave an update last week, and the news generally wasnt good. Of the five participants who have so far received the initial dose of EBT-101, three stopped antiretroviral therapy. Unfortunately, all three experienced viral rebound and had to restart their antiretrovirals. This likely occurred because the gene therapy did not reach all cells harboring latent HIV, and even a very small number of cells containing residual HIV DNA is enough to reignite viral replication.

But the news was not all bad. One EBT-101 recipient was able to maintain viral suppression for four months after treatment discontinuationconsiderably longer than it typically takes for the virus to rebound after stopping antiretrovirals. This suggests that EBT-101 or similar CRISPR therapies might play a role in a combination functional cure strategy.

We know that many people were hopeful that a first trial could provide evidence of a possible cure for HIV because the field has been waiting over 20 years for a cure, Excision senior vice president William Kennedy, MD, said in a news release. However, it was essential that this clinical trial establish safety for EBT-101 as a gene therapy product as well as safety related to the use of CRISPR for the field.

Excision is now testing a higher dose of EBT-101 in a second cohort and is exploring new CRISPR delivery methods that might be more efficient than the adeno-associated virus vector. One possibility is lipid nanoparticles like the ones used to deliver messenger RNA (mRNA) in COVID-19 vaccines.

The company is also exploring CRISPR-based approaches for other latent viral infections. Herpes simplex virus (HSV) persists in nerve cells, and it can reactivate to cause cold sores, genital herpes or eye inflammation (keratitis). Hepatitis B virus (HBV) establishes chronic infection in the liver, where it can potentially lead to cirrhosis and liver cancer. Unlike HIV and other retroviruses, however, HSV and HBV do not integrate their genetic blueprints into the chromosomes of human cells, so they may be easier to remove.

In other presentations at the ASGCT meeting, researchers reported preclinical results for another experimental CRISPR therapy dubbed EBT-104, showing that a single dose reduced HSV DNA by more than 99% in laboratory cell cultures. Whats more, it eliminated viral shedding in 11 of 12 rabbits with herpes keratitis, according to the news release.

In other preclinical research, a single dose of EBT-107a CRISPR compound delivered by lipid nanoparticlesreduced HBV DNA, hepatitis B surface antigen and hepatitis B e antigen by 98%, 97% and 92%, respectively, in a mouse model of hepatitis B. Unlike CRISPR delivered by viral vectors, EBT-107 in nanoparticles and could potentially be given as multiple doses to reach more latent virus.

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CRISPR HIV Gene Therapy Disappoints in Early Study - POZ

Recommendation and review posted by Bethany Smith

Pfizer has reported the death of a patient in the Phase II DAYLIGHT study of experimental gene therapy, fordadistrogene movaparvovec, to treat Duchenne muscular dystrophy (DMD).

The trial is designed to assess the safety and dystrophin expression in young boys with DMD.

In a community letter issued by the company, Pfizer said: We do not yet have complete information and are actively working with the trial site investigator to understand what happened.

The patient received the investigational gene therapy, fordadistrogene movaparvovec, in early 2023.

The company has also paused participant dosing associated with the crossover portion of its Phase III CIFFREO trial, while continuing other trial activities as scheduled.

Pfizer concluded the initial dosing of subjects in placebo-controlled, randomised crossover CIFFREO trial last year.

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Both DAYLIGHT and CIFFREO trials are part of Pfizers research into fordadistrogene movaparvovec for DMD treatment, targeting different age groups.

DAYLIGHT enrols boys aged two to less than four years, whereas CIFFREO involves boys aged four to less than eight years.

Despite the dosing pause in CIFFREO, the company clarified that this action does not extend to other ongoing trials within the fordadistrogene movaparvovec programme, as dosing in those studies has already been completed.

Pfizer is also collaborating with regulatory authorities and an independent external Data Monitoring Committee to investigate the cause of the patients death.

The company added: The safety and well-being of the patients in our clinical trials remains our top priority, and we are committed to sharing more information with the medical and patient community as soon as we can.

We are also aware that many in the patient community are hopeful about the potential benefit of fordadistrogene movaparvovec for the treatment of DMD, and we will continue to collect data from our trials to evaluate its ability to address this disease.

Earlier this month, the company reported net income of $3.11bn in the first quarter (Q1) of 2024, marking a decrease of 44% from $5.54bn posted in Q1 2023.

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Pfizer reports fatality in Phase II DMD gene therapy study - Clinical Trials Arena

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Pictured: Panelists on stage at ASGCT (L to R: NHLBIs Gary Gibbons, NCATS Joni Rutter, Amy Jenkins of Advanced Research Projects Agency for Health, NINDS Amir Tamiz and ASGCT President Jeffrey Chamberlain)/Photo by Greg Slabodkin

Advancing translational research of novel therapies is a priority for both the scientific community and the National Institutes of Health, according to stakeholders at the 2024 American Society of Gene & Cell Therapy (ASGCT) annual meeting.

In a Friday fireside chat with officials from the National Institute of Health (NIH) at ASGCT 2024, ASGCT President Jeffrey Chamberlain described translational research as the critical nexus between basic science and clinical investigation.

This is an area where were trying to bridge the gap between fundamental new discoveries and their applications to improve human health, Chamberlain said.

The sessions agenda highlighted some of the pivotal basic science and translational studies, as well as the researchers who are making breakthroughs when it comes to transformative genetic therapies.

Gary Gibbons, director of the National Heart, Lung, and Blood Institute (NHLBI) at NIH, touted his organizations launch of the Cure Sickle Cell Initiative in 2018 as a collaborative research effort to accelerate the development of gene therapies to cure sickle cell disease (SCD).

Gibbons said that new FDA-approved curative gene therapies for SCD Vertex Pharmaceuticals CRISPR-based Casgevy and bluebird bios gene therapy Lyfgeniaare a case study in the value of translational research for creating transformative treatments for patients.

We had folks from Vertex and bluebird bio at the table from the beginning, as well as our colleagues at FDA, Gibbons commented. Because we knew at that stage we were defining a regulatory pathway toward that end.

Chamberlain called Casgevy and Lyfgenia possibly the most exciting development ever in the field of gene therapies.

Joni Rutter, director of the National Center for Advancing Translational Sciences (NCATS) at NIH, said that one of her organizations primary missions is to re-engineer the translational research process so new treatments and cures for diseases can reach patients faster. Our mission is essentially to understand how to traverse that pipeline better, faster, more effectively and efficiently, Rutter said.

NCATS has taken credit for enabling a path to market for ReveraGen BioPharmas Duchenne muscular dystrophy therapy, which was approved by the FDA in late 2023. Another NCATS-led program Rutter pointed to is the Platform Vector Gene Therapy (PaVe-GT) pilot project. Launched in 2019, the PaVe-GT pilot aims to test the hypothesis of whether a platform vector approach will increase efficiency in preclinical testing and clinical trial start-up.

Rutter also pointed to the NCATS-led Bespoke Gene Therapy Consortium (BGTC), a public-private collaboration involving NIH, FDA, industry and patient groups intended to help accelerate the delivery of AAV-based gene therapies for rare diseases. With BGTC, were establishing how to optimize the biology and the studies behind developing the AAV vectors and then were also optimizing the manufacturing, Rutter said.

Greg Slabodkin is the news editor at BioSpace. You can reach him atgreg.slabodkin@biospace.com. Follow him onLinkedIn.

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ASGCT24: Translational Research Powering Potentially Transformative Therapies - BioSpace

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In posters presented at theISPORThe Professional Society for Health Economics and Outcomes Researchmeeting held in Atlanta, Georgia, researchers explored Duchenne muscular dystrophy (DMD) caregiver experiences through cross-sectional surveys,1,2 and an economic analysis examined gene therapys impact on work opportunities for caregivers.3

DMD is an inherited neuromuscular disorder that leads to progressive muscle degeneration and weakness.4 It primarily affects boys, and symptom onset is typically in early childhood. Life expectancy for those with DMD has increased in recent years, and survival into the early 30s is becoming more common.

As DMD progresses, affected individuals rely more heavily on their caregivers, the authors of one poster wrote.1 Their cross-sectional survey aimed to characterize the experiences of caregivers in the US, as contemporary data are lacking.

The analysis included 106 US-based caregivers who completed an online survey between March and May 2023. The survey included questions capturing the use of formal (paid) and informal (unpaid) care; informal burden on caregivers rated on a scale from 0 to 10, 10 being the most challenging; and how accessibility barriers impact family plans. The survey results were stratified by patient ambulatory status.

The mean caregiver age was 46 years, and the mean care recipient age was 14.5 years. Eighty-one percent of the caregivers were mothers, and 11% were caregivers for 2 individuals with DMD. Out of 118 total patients with DMD being cared for, 47% (n = 55) were nonambulatory. Caregivers of ambulatory patients reported spending a median of 4.5 hours per day caregiving, while caregivers of nonambulatory patients reported a median of 8 hours per day. The median amount of time spent giving care per day in the overall cohort was 6 hours per day.

Caregivers reported a median burden of 5 overall, with those caring for nonambulatory patients reporting a burden of 6 and caregivers of ambulatory recipients reporting a burden rating of 4. Overall, 75% of caregivers reported utilizing additional informal caregiving, and 26% used additional formal caregiving. Of those using formal caregiving, 13% were caregivers for ambulatory patients and 42% were caregivers for nonambulatory patients. Accessibility barriers led to frequent changes in everyday plans for 74% of families.

Overall, the findings show the significant amounts of time and energy required of caregivers for individuals with DMD, as well as the increasing demands associated with disease progression.

Muscular dystrophy | Image credit: Lemau Studio - stockadobe.com

Another poster highlighted the impacts of caregiving on paid work, including the extent of DMD caregivers paid work accommodations and lost productivity based on survey results.2 The investigators recruited survey participants through a US-based DMD advocacy group and included questions from the Work Productivity and Activity Impairment (WPAI) questionnaire for DMD caregivers, with higher scores indicating greater impairment.

Among 106 caregivers, 81% were mothers, and the mean age was 46, and 89% of caregivers reported caring for 1 individual with DMD. The annual household income was greater than $100,000 annually for 49% of households in the study.

Overall, 68% of respondents (n = 72) were employed, with 55% (n = 52) employed full-time. Based on WPAI scores, 40.7% of respondents had experienced overall activity impairment in the past week, and absenteeism among those employed was 8.4%. Presenteeism, or impairment at work, was reported by 30.5% of caregivers, and absenteeism plus presenteeism (work productivity loss) by 34.8%. Among the 11% of caregivers caring for 2 individuals with DMD, 67% (n = 8) were employed, and 62.5% (n = 5) reported work productivity was impacted by DMD.

Furthermore, 77% (n = 82) of caregivers reported work-related changes due to caregiving responsibilities. Twenty-five percent quit, 26% took lower paying jobs, 34% changed their job role or responsibilities, 29% reduced working hours, and 34% took time off from work.

The results demonstrate the considerable paid work impact associated with caregiving for one or two individuals with DMD, the authors concluded, noting that the findings are limited due to the low number of caregivers of 2 individuals included in the study.

In an economic analysis, investigators explored how delandistrogene moxeparvovec (Elevidys; Sarepta Therapeutics), a gene therapy approved for the treatment of ambulatory patients aged 4 to 5 years with DMD, could impact the work opportunity of those caring for patients with DMD.3 They developed a partitioned survival model including 5 statesearly ambulatory, late ambulatory, early nonambulatory, late nonambulatory, and deathand estimated outcomes and caregiver work opportunity with standard-of-care (SOC) treatment vs SOC plus gene therapy for a cohort of 4-year-old early ambulatory patients with DMD. The researchers applied 2.8% salary growth and 3% discount rates annually.

With SOC treatment alone, caregivers were estimated to lose 7194 (15.5%) hours and experience a lifetime income loss of $249,697 (16.1%)/undiscounted $431,911 (16.2%) compared with the general US population. Caregivers of patients treated with SOC plus gene therapy were estimated to lose 4564 (9.8%) hours, equating a lifetime income loss of $153,396 (9.9%)/undiscounted $318,375 (11.9%) compared with the general population.

DMD substantially impacts caregiver work opportunity and income, the authors concluded. Based on this model, delandistrogene moxeparvovec could potentially increase caregiver work opportunity.

Reference

1. Audhya IF, Dunne JS, Patel S, et al. Characterizing the experience of caregiving for those with Duchenne muscular dystrophy (DMD): results from a cross-sectional survey. Presented at: International Society for Pharmacoeconomics and Outcomes Research 2024; May 5-8, 2024; Atlanta, GA. Poster 6044.

2. Patel S, Dunne JS, Audhya IF, et al. The balancing act of paid work and caregiving in Duchenne muscular dystrophy (DMD): results from a cross-sectional survey. Presented at: International Society for Pharmacoeconomics and Outcomes Research 2024; May 5-8, 2024; Atlanta, GA. Poster 6018.

3. Innis B, Henry A, Nelson L, et al. Economic analysis of the impact of delandistrogene moxeparvovec gene therapy on work opportunity in caregivers of individuals with Duchenne muscular dystrophy. Presented at: International Society for Pharmacoeconomics and Outcomes Research 2024; May 5-8, 2024; Atlanta, GA. Poster 2050.

4. Duchenne muscular dystrophy (DMD). Muscular Dystrophy Association. Accessed May 9, 2024.https://www.mda.org/disease/duchenne-muscular-dystrophy

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Posters Characterize DMD Caregiver Experiences, Impact of Gene Therapy on Caregiving Demands - AJMC.com Managed Markets Network

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Published: 09 May 2024

A girl born deaf can now hear unaided after participating in a world-first trial.

TheCHORD study is for children born with hearing loss due to a genetic condition called auditory neuropathy.Auditory neuropathy is a condition where the ear can detect sound normally, but has a problem sending signals to the brain.

This can be caused by a fault in the OTOF gene. The gene makes a protein called otoferlin that allows cells in the ear to communicate with the hearing nerve. Changes in this gene can reduce sound transmission from the inner hair cells to the hearing nerves.

Gene therapy aims to deliver a working copy of the faulty OTOF gene. The treatment is given using a neutralised virus, injected into the inner ear under general anaesthetic.

The study started in 2023 using a treatment made by Regeneronand is supported by NIHR Cambridge Clinical Research FacilityandNIHR Cambridge Biomedical Research Centre.

Opal Sandy is the first patient treated in the global gene therapy trial. Opal was born completely deaf because of neuropathy. She was treated shortly before her first birthday.

During surgery, while Opal was given the gene therapy in right ear, a cochlear implant was fitted in her left ear.

Opals mother, Jo Sandy, said: When Opal could first hear us clapping unaided it was mind-blowing - we were so happy when the clinical team confirmed at 24 weeks that her hearing was also picking up softer sounds and speech. The phrase near normal hearing was used, and everyone was so excited that such amazing results had been achieved.

Professor Manohar Bance is the chief investigator for the trial. He is also an ear surgeon at Cambridge University Hospitals NHS Foundation Trust. He said: These results are spectacular and better than I expected. Gene therapy has been the future of otology and audiology for many years and Im so excited that it is now finally here. This is hopefully the start of a new era for gene therapies for the inner ear and many types of hearing loss.

Dr Richard Brown, Consultant Paediatrician at CUH, who is an investigator on the CHORD trial, said: The development of genomic medicine and alternative treatments is vital for patients worldwide, and increasingly offers hope to children with previously incurable disorders. It is likely that in the long run such treatments require less follow up so may prove to be an attractive option, including within the developing world. Follow up appointments have shown effective results so far with no adverse reactions and it is exciting to see the results to date.

Doctors in other countries, including China, are exploring very similar treatments for the OTOF gene mutation.Around 20,000 people across the US, UK, Germany, France, Spain and Italy are thought to have auditory neuropathy due to OTOF mutations, which shows the potential significance of a successful treatment. Patients are being enrolled in the study in the US, UK and Spain.

Addenbrookes Hospital in Cambridge is participating in the trial. Read more on the Cambridge University Hospitals website.

Photo of Opal Sandy taken byCambridge University Hospitals

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Baby born deaf can hear after breakthrough gene therapy - National Institute for Health Research

Recommendation and review posted by Bethany Smith

The companys presentation at ASGCT includes preliminary data results for a child who received the gene therapy.

Regeneron presented results from an ongoing Phase I/II clinical trial for its investigational gene therapy, DB-OTO, at the annual American Society of GeneandCell Therapy (ASGCT) meeting, being held in Baltimore, Md. from May 711. DB-OTO, a gene therapy for genetic deafness, improved hearing in one child, treated at 11 months old to normal levels within 24 weeks. A second child, treated at 4 years old, also showed hearing improvements at a six-week assessment, according to the company (1). Both children were born with genetic deafness due to variants of the otoferlin gene.

The results are from the ongoing Phase I/II CHORD (NCT# 05788536) first-in-human, multicenter, open-label trial. The trial evaluates the safety, tolerability, and preliminary efficacy of DB-OTO in infants, children, and adolescents with otoferlin (OTOF) variants in the United States, United Kingdom, and Spain. CHORD is a two-part study where participants will receive a single intracochlear injection of DB-OTO in one ear in Part A of the study. In Part B, participants receive simultaneous single intracochlear injections of DB-OTO in both ears at the selected dose from Part A.

Congenital deafness impacts approximately 1.7 out of every 1000 children born in the United States, according to Regeneron (1).Otoferlin-related hearing loss is especially rare and caused by variants in the otoferlin gene, which impairs the production of theOTOFprotein that is critical for the communication between the sensory cells of the inner ear and the auditory nerve.

Study data presented at ASGCT showsat the 24-week assessment the first participant, a 16-month-old child, had improvement of hearing to normal levelsacross key speech frequencies, with an average 84 dB improvement from baseline and one frequency measure reaching 10 dB in hearing level per PTA. Across all tested frequencies, an average 80 dB improvement from baseline was observed. There was also positive ABR responses, with best frequency reaching 45 dB.

The second participant, a four-year-old child, experienced consistent results to the first participant, including initial improvement of hearing with responses to loud sounds,which was observed across key speech frequencies, with an average 19 dB improvement from baseline and one frequency measure reaching 80 dB in hearing level per PTA. Across all tested frequencies, an average 16 dB improvement from baseline was observed. Positive ABR responses had best frequency reaching 75 dB.

The opportunity of providing the full complexity and spectrum of sound in children born with profound genetic deafness is a phenomenon I did not expect to see in my lifetime, saidLawrence R. Lustig, MD, chair of Columbia UniversitysDepartment of OtolaryngologyHead & Neck Surgery and a clinical trial investigator, in a press release. These impressive results showcase the revolutionary promise of DB-OTO as a potential treatment for otoferlin-related deafness, and we are excited to see how this translates into an individuals development, especially [because] early intervention is associated with better outcomes for speech development. With the DB-OTO CHORD trial now enrolling participants in sites across theUS andEurope, were part of the beginning of a new era of gene therapy research that looks to create treatment options that address the root cause of profound genetic deafness.

1. Regeneron. Latest DB-OTO Results Show Dramatically Improved Hearing to Normal Levels in a Child with Profound Genetic Deafness Within 24 Weeks and Initial Hearing Improvements in a Second Child at 6 Weeks. Press Release. May 8, 2024.

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Regeneron Presents Update on Gene Therapy for Genetic Deafness at ASGCT - Pharmaceutical Technology Magazine

Recommendation and review posted by Bethany Smith

A new study published in New England Journal of Medicine has described the effect of gene editing in a group of patients with Leber congenital amaurosis (LCA).

The clinical trial involved two children and 12 adults with the genetic eye condition. Participants received an experimental gene editing therapy called Edit-101.

Outcomes used to evaluate the clinical trial were: visual acuity; full-field test results; the ability of participants to navigate a research maze containing obstacles and varied lighting levels; and self-reported improvements in quality of life.

The researchers found that 11 of the participants (79%) showed improvements in at least one of the four outcome measures, while 6 participants (43%) showed improvement in two or more outcomes. Four patients (29%) experienced improvements in visual acuity.

The scientists reported that there were no serious adverse effects linked to the treatment.

Dr Mark Pennesi, of Oregon Health & Science University, shared how rewarding it was to hear about vision improvements among patients who took part in the trial.

One of our trial participants has shared several examples, including being able to find their phone after misplacing it and knowing that their coffee machine is working by seeing its small lights, he said.

While these types of tasks might seem trivial to those who are normally sighted, such improvements can have a huge impact on quality of life for those with low vision, he added.

More:
Gene therapy improves vision in Leber congenital amaurosis patients - Association of Optometrists (AOP)

Recommendation and review posted by Bethany Smith

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At four

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weeks, when we initially heard her

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turning to clapping, that was like

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I was mind-blown that had worked.

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Id sat behind Opal countless times

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when she's had, to us, ridiculously

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loud noises

00;00;18;17 - 00;00;20;09

blared through speakers in a sound booth

00;00;20;09 - 00;00;23;02

and she's never once turned, so to

00;00;23;02 - 00;00;28;23

see that happen is mind-blowing.

00;00;28;23 - 00;00;32;06

Good girl! Yippee!

00;00;32;09 - 00;00;34;02

Clever girl!

00;00;34;02 - 00;00;36;10

So this particular trial was for children

00;00;36;10 - 00;00;38;05

who have the OTOF gene variant

00;00;38;05 - 00;00;39;05

that does not produce

00;00;39;05 - 00;00;40;09

a particular protein

00;00;40;09 - 00;00;41;21

that they need for hearing.

00;00;41;21 - 00;00;43;06

And the surgery involves

00;00;43;06 - 00;00;43;29

approaching the ear

00;00;43;29 - 00;00;46;22

like a cochlear implant, infusing a virus

00;00;46;22 - 00;00;48;11

that makes the cells

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produce the protein that they're missing.

00;00;50;27 - 00;00;52;02

So one thing that we've been

00;00;52;02 - 00;00;53;11

really excited about here

00;00;53;11 - 00;00;54;22

is that we've been able to use

00;00;54;22 - 00;00;57;04

a really small dose of gene therapy

00;00;57;04 - 00;00;59;07

delivered directly to the cochlear,

00;00;59;07 - 00;01;00;06

and what that means is

00;01;00;06 - 00;01;01;12

that we're not delivering

00;01;01;12 - 00;01;02;25

a large dose of gene therapy

00;01;02;25 - 00;01;04;01

to the rest of the body.

00;01;04;01 - 00;01;06;11

So it means that we see

00;01;06;11 - 00;01;07;13

fewer side effects.

00;01;07;13 - 00;01;08;10

And for Opal,

00;01;08;10 - 00;01;09;20

this is a very big change,

00;01;09;20 - 00;01;11;23

because before the gene therapy,

00;01;11;23 - 00;01;13;06

she had profound hearing loss.

00;01;13;06 - 00;01;14;04

That meant she couldn't

00;01;14;04 - 00;01;16;02

hear any sounds in that ear at all,

00;01;16;02 - 00;01;17;13

even when they were very loud.

00;01;17;13 - 00;01;18;14

And now she can hear

00;01;18;14 - 00;01;20;11

sounds at a very soft level,

00;01;20;11 - 00;01;22;02

almost in the normal range for children

00;01;22;02 - 00;01;23;13

her age.

00;01;23;13 - 00;01;24;23

Can I have a kiss?

00;01;24;23 - 00;01;31;03

Aw, good girl, daddy kiss, mummy kiss.

00;01;31;05 - 00;01;35;11

Yeah, if she doesn't have her implant

00;01;35;11 - 00;01;38;07

on, she acts exactly the same

00;01;38;07 - 00;01;39;09

as if she did have it on,

00;01;39;09 - 00;01;42;05

because she can hear so well with this

Continued here:
Baby born deaf can hear after breakthrough gene therapy - Cambridge University Hospitals

Recommendation and review posted by Bethany Smith

Revolutionizing Treatment: ASGCTs Clinical Trials Spotlight on Immunotherapy, Cancer Vaccines, and Auditory Diseases

At this years ASGCTs 27th Annual Meeting in Baltimore, groundbreaking clinical trials took center stage, illuminating the latest advancements in gene therapy research. These trials presented innovative approaches ranging from targeted cell and gene therapies to advancements in AAV gene therapy and novel findings in cell therapy. Here we dive deeper into immunotherapy and cancer vaccines, alongside novel treatments for auditory diseases. With a focus on cutting-edge treatments and promising outcomes, these presentations underscore the transformative potential of gene and cell therapy in addressing diverse medical challenges.

Generating Antitumor T Cells: Personalized Neoantigen Vaccine and Pembrolizumab in Advanced Hepatocellular Carcinoma

Customized cancer vaccines targeting neoantigens from a patients tumor may boost the effectiveness of PD-1 inhibitors by stimulating tumor-specific immune responses. Findings from a single-arm Phase Ib/2a study investigate the use of a personalized therapeutic cancer vaccine (PTCV), designed to target specific mutations in an individuals tumor, in combination with pembrolizumab, a PD-1 inhibitor, for the treatment of advanced hepatocellular carcinoma (HCC). The results indicate that this combination therapy induces a robust immune response, involving both CD8+ and CD4+ T cells targeting tumor-specific neoantigens. These findings suggest a potential mechanism for the observed clinical activity and provide insight into improving treatment outcomes for patients with advanced HCC.

Long-Term Safety and Integration Site Analysis of T Cells Modified with Lentiviral or Gammaretroviral Gene Addition

No adverse events related to insertional mutagenesis were detected in a large patient cohort treated with CART therapy. Here, the long-term safety of CAR T cell therapy was assessed by analyzing data from 780 patients treated between 2001 and 2023. While 21 patients developed secondary malignancies, none were directly linked to the CAR T cell product. Integration site sequence analysis revealed insights into the behavior of transduced cell clones, with no evidence of pathogenic insertional mutagenesis. Overall, the study provides reassurance regarding the safety of CAR T cell therapy, despite recent concerns about secondary T cell malignancies.

CHORD Trial: DB-OTO Gene Therapy for Pediatric Hearing Loss

This trial highlights the significance of biallelic otoferlin gene mutations in causing severe-to-profound sensorineural hearing loss, emphasizing the potential for OTOF gene replacement therapy to restore physiological hearing. Subsequently, this investigation introduces DB-OTO, a novel adeno-associated virus vector designed for intracochlear delivery of the OTOF gene, and outlines the objectives of the CHORD trial (NCT05788536) evaluating its safety and efficacy in pediatric patients with profound hearing loss due to OTOF mutations. Initial results from the trial demonstrate promising outcomes, with no dose-limiting toxicities or DB-OTO-related adverse events reported. Significant improvements in hearing, as indicated by behavioral pure tone audiogram thresholds and auditory brainstem response, were observed in the DB-OTO-treated ear. Additionally, parental reports and auditory skills assessments further support the positive impact of DB-OTO on auditory function and development. These findings underscore the potential of DB-OTO gene therapy as a viable treatment option for patients with profound hearing loss caused by OTOF mutations, warranting further investigation in larger patient cohorts.

In conclusion, the presentations from the ASGCT annual conference in Baltimore showcase significant strides in immunotherapy, gene therapy, and treatments for ophthalmic and auditory diseases. The studies on personalized neoantigen vaccines, CAR T cell therapy safety, and DB-OTO gene therapy offer promising insights into improving outcomes for patients with advanced hepatocellular carcinoma, hematologic malignancies, and hearing loss due to genetic mutations. These findings underscore the potential of innovative cell and gene therapies in addressing unmet medical needs and enhance patient care. Moving forward, continued research and development in these areas will be instrumental in advancing precision medicine and improving the lives of individuals affected by these conditions. ASGCT 2024, gene therapy, gene therapy definition, cell and gene therapy, AAV gene therapy, Cell Reports impact factor, cell therapy, American, Baltimore, therapy, cell

Advancing Health: ASGCTs Clinical Trials Spotlight on Cell Therapy and Cell-Based Gene Therapy

2024-05-09

Profluent Achieves Human Genome Editing Milestone Using OpenCRISPR-1: The First AI-Generated, Open-Source Gene Editor

2024-05-08

The Gene & Cell Therapy Landscape: Recent Approvals and Upcoming Therapeutics of Interest

2024-05-06

See the article here:
Revolutionizing Treatment: ASGCT's Clinical Trials Spotlight on Immunotherapy, Cancer Vaccines, and Auditory ... - geneonline

Recommendation and review posted by Bethany Smith

Ace Therapeutics announced the unveiling of its gene therapy development services to advance glaucoma research. To advance glaucoma research, Ace Therapeutics, an integrated biotechnology company with a broad research scope and comprehensive services, has announced the unveiling of its gene therapy development services. The company's innovative gene therapy approach holds promise in providing a new, effective treatment option for individuals suffering from this eye disease that damages the optic nerve.

Glaucoma is a leading cause of irreversible blindness worldwide, and currently available treatments only offer temporary relief of symptoms. Studies have found the main risk factor for glaucoma is high IOP, primarily due to RGC injury and death. For this reason, Ace Therapeutics offers glaucoma gene therapy development [https://www.acetherapeutics.com/glaucoma/gene-therapy-development-for-glaucoma.html] services, targeting IOP and RGC-associated candidate genes, as well as many other related genes and pathways. By delivering therapeutic nucleic acids directly to the affected cells, the company's gene therapy has the potential to halt the progression of glaucoma and preserve vision for patients.

Considering the great potential of stem cells as a possible therapy for glaucoma, the expert team at Ace Therapeutics can provide specialized services in glaucoma drug development, offer the opportunity to develop drugs for glaucoma using stem cell technology and stem cell-based therapies to restore vision loss due to glaucoma. In addition, the company also utilizes siRNA-based gene silencing strategies to treat glaucoma, providing researchers with development services to study the composition and mechanisms of siRNA drugs, offering solutions to the fundamental challenges faced during development.

"We are excited to collaborate with worldwide researchers to develop a more effective treatment for glaucoma using our gene therapy expertise and technologies," said the senior scientist at Ace Therapeutics. "We can provide genomic analysis or directly select appropriate genes from candidate genes that are genetically linked to glaucoma to those involved in the relevant pathway. Also, we can develop gene delivery systems for glaucoma gene therapy to ensure the transfer of nucleic acid drugs to target cells and to advance the translational application of gene therapy."

About Ace Therapeutics

Ace Therapeutics Glaucoma [https://www.acetherapeutics.com/glaucoma/] is an innovative ophthalmic disease research company that offers a wide range of services in basic research, drug development and preclinical studies, delivering innovative and high-quality solutions to global clients. Ace Therapeutics' mission is to be a leader in the field of glaucoma research, providing unique drug development, and preclinical research solutions to advance researchers' project development.

Media Contact Company Name: Ace Therapeutics Contact Person: Daisy Mostert Email:Send Email [https://www.abnewswire.com/email_contact_us.php?pr=ace-therapeutics-unveils-gene-therapy-development-services-to-fuel-glaucoma-research] Country: United States Website: https://www.acetherapeutics.com/

This release was published on openPR.

See original here:
Ace Therapeutics Unveils Gene Therapy Development Services to Fuel Glaucoma Research - openPR

Recommendation and review posted by Bethany Smith

- Company announces clearance of IND application with FDA for anti-tau antibody VY-TAU01 for the treatment of Alzheimers disease; expect to begin single ascending dose trial in the coming weeks -

- Development candidates selected for Neurocrine-partnered GBA1 and Friedreichs Ataxia gene therapy programs; potential for three gene therapies, including SOD1-ALS, to enter the clinic in 2025 -

- Appointed neurology clinical development expert Toby Ferguson, M.D., Ph.D., as Chief Medical Officer -

- Strong cash position of approximately $400 million as of March 31, 2024; expected to provide runway through multiple clinical data readouts into 2027 -

LEXINGTON, Mass., May 13, 2024 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to advancing neurogenetic medicines, today reported first quarter 2024 financial and operating results.

We have obtained IND clearance for our anti-tau antibody VY-TAU01 for Alzheimers disease, and we expect to dose the first subject in our single ascending dose trial in healthy volunteers in the coming weeks, said Alfred W. Sandrock, Jr., M.D., Ph.D., Chief Executive Officer of Voyager. Our gene therapy pipeline is also advancing, with development candidates selected in the GBA1 and Friedreichs Ataxia programs partnered with Neurocrine, as well as in our wholly-owned SOD1-ALS program. We expect to achieve IND filings for all three of these gene therapy programs in 2025. We maintain a strong cash position of approximately $400 million at quarter-end, with runway into 2027, which we anticipate will enable us to reach multiple data readouts in 2025 and 2026.

First Quarter 2024 and Recent Highlights

Anticipated Upcoming Milestones

First Quarter 2024 Financial Results

Financial Guidance

Voyager is committed to maintaining a strong balance sheet that supports the advancement and growth of its platform and pipeline. Voyager continues to assess its planned cash needs both during the current period and in future periods. We expect our cash, cash equivalents, and marketable securities, along with amounts expected to be received as reimbursement for development costs under the Neurocrine and Novartis collaborations, certain near-term milestones, and interest income, to be sufficient to meet Voyagers planned operating expenses and capital expenditure requirements into 2027.

Conference Call

Voyager will host a conference call and webcast today at 4:30 p.m. ET to discuss first quarter 2024 financial and operating results. To participate via telephone and join the call live, please register in advance here: https://register.vevent.com/register/BI1f6af80e7a614ca7925cbad2f35a55c6. Upon registration, telephone participants will receive a confirmation email detailing how to join the conference call, including the dial-in number and a unique passcode. A live webcast of the call will also be available on the Investors section of the Voyager website at ir.voyagertherapeutics.com, and a replay of the call will be available at the same link approximately two hours after its completion. The replay will be available for at least 30 days following the conclusion of the call.

About the TRACER Capsid Discovery Platform

Voyagers TRACER (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) capsid discovery platform is a broadly applicable, RNA-based screening platform that enables rapid discovery of novel AAV capsids to enable gene therapy. Voyager has leveraged TRACER to create multiple families of novel capsids that, following intravenous delivery in preclinical studies, harness the extensive vasculature of the central nervous system (CNS) to cross the blood-brain barrier and transduce a broad range of CNS regions and cell types. In cross-species preclinical studies (rodents and multiple non-human primate species), intravenous delivery of TRACER-generated capsids resulted in widespread payload expression across the CNS at relatively low doses, enabling selection of multiple development candidates in Voyagers wholly-owned and partnered gene therapy programs for neurologic diseases.

About Voyager Therapeutics

Voyager Therapeutics, Inc. (Nasdaq: VYGR) is a biotechnology company dedicated to leveraging the power of human genetics to modify the course of and ultimately cure neurological diseases. Our pipeline includes programs for Alzheimers disease, amyotrophic lateral sclerosis (ALS), Parkinsons disease, and multiple other diseases of the central nervous system. Many of our programs are derived from our TRACER AAV capsid discovery platform, which we have used to generate novel capsids and identify associated receptors to potentially enable high brain penetration with genetic medicines following intravenous dosing. Some of our programs are wholly owned, and some are advancing with partners including Alexion, AstraZeneca Rare Disease; Novartis Pharma AG; Neurocrine Biosciences, Inc.; and Sangamo Therapeutics, Inc. For more information, visit http://www.voyagertherapeutics.com.

Voyager Therapeutics is a registered trademark, and TRACER is a trademark, of Voyager Therapeutics, Inc.

Forward-Looking Statements

This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as expect, will, believe, anticipate, potential, trigger or continue, and other similar expressions are intended to identify forward-looking statements.

For example, all statements Voyager makes regarding Voyagers ability to advance its AAV-based gene therapy programs and tau antibody program, including expectations for Voyagers achievement of preclinical and clinical development milestones for its potential development candidates such as IND filings, the initiation of clinical trials, and generation of clinical data and proof-of-concept; Voyagers ability to advance gene therapy product candidates under the Neurocrine and Novartis collaborations; Voyagers anticipated financial results, including the anticipated receipt by Voyager of revenues or reimbursement payments from collaboration partners; and Voyagers cash runway and ability to generate sufficient cash resources to enable it to continue its business and operations are forward looking.

All forward-looking statements are based on estimates and assumptions by Voyagers management that, although Voyager believes such forward-looking statements to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Voyager expected. Such risks and uncertainties include, among others, the expectations and decisions of regulatory authorities; the timing, initiation, conduct and outcomes of Voyagers preclinical and clinical studies; the availability of data from clinical trials; the availability or commercial potential of product candidates under collaborations; the willingness and ability of Voyager's collaboration partners to meet obligations under collaboration agreements with Voyager; the continued development of Voyagers technology platforms, including Voyagers TRACER platform and its antibody screening technology; Voyagers scientific approach and program development progress, and the restricted supply of critical research components; the development by third parties of capsid identification platforms that may be competitive to Voyagers TRACER capsid discovery platform; Voyagers ability to create and protect intellectual property rights associated with the TRACER capsid discovery platform, the capsids identified by the platform, and development candidates for Voyagers pipeline programs; the possibility or the timing of Voyagers receipt of program reimbursement, development or commercialization milestones, option exercise, and other payments under Voyagers existing licensing or collaboration agreements; the ability of Voyager to negotiate and complete licensing or collaboration agreements with other parties on terms acceptable to Voyager and the third parties; the success of programs controlled by third party collaboration partners in which Voyager retains a financial interest, and the success of Voyagers product candidates; the ability to attract and retain talented directors, employees, and contractors; and the sufficiency of cash resources to fund its operations and pursue its corporate objectives.

These statements are also subject to a number of material risks and uncertainties that are described in Voyagers most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission. All information in the press release is as of the date of this press release, and any forward-looking statement speaks only as of the date on which it was made. Voyager undertakes no obligation to publicly update or revise this information or any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

Contacts

Trista Morrison, NACD.DC, tmorrison@vygr.com Investors: Adam Bero, Ph.D., abero@kendallir.com Media: Brooke Shenkin, brooke@scientpr.com

GAAP vs. Non-GAAP Financial Measures Voyagers financial statements are prepared in accordance with generally accepted accounting principles in the United States, or GAAP, and represent revenue and expenses as reported to the Securities and Exchange Commission. Voyager has provided in this release certain financial information that has not been prepared in accordance with GAAP, including net collaboration revenue and net research and development expenses, which exclude the impact of reimbursement by Neurocrine Biosciences (Neurocrine) for expenses we incur in conducting preclinical development activities under our collaboration agreements. Management uses these non-GAAP measures to evaluate the Companys operating performance in a manner that allows for meaningful period-to-period comparison and analysis of trends in its business. Management believes that such non-GAAP measures are important in comparing current results with prior period results and are useful to investors and financial analysts in assessing the Companys operating performance. Non-GAAP financial measures are not required to be uniformly applied, are not audited and should not be considered in isolation. The non-GAAP measures give investors and financial analysts a better understanding of our net revenue and net research and development expenses without the pass-through impact of Neurocrine costs. The non-GAAP financial information presented here should be considered in conjunction with, and not as a substitute for, the financial information presented in accordance with GAAP. Investors are encouraged to review the reconciliation of these non-GAAP measures to their most directly comparable GAAP financial measures set forth below.

Note 1: Under the Company's existing collaboration agreements with Neurocrine and Novartis, Neurocrine and Novartis have agreed to be responsible for all costs the Company incurs in conducting preclinical development activities for certain collaboration programs, in accordance with joint steering committee agreed upon workplans and budgets. Reimbursable research and development services performed during the period are captured within collaboration revenue and research and development expenses in the Company's consolidated statements of operations. During the three months ended March 31, 2024, we incurred $3.2 million of reimbursable research and development services recorded within collaboration revenue and research and development expenses. During the three months ended March 31, 2023, we incurred $0.3 million of reimbursable research and development services recorded within collaboration revenue and research and development expenses.

Link:
Voyager Therapeutics Reports First Quarter 2024 Financial and Operating Results - GlobeNewswire

Recommendation and review posted by Bethany Smith

Mesenchymal Stem Cells Market

Major Highlights of TOC: Chapter 1: Overview of the Global Mesenchymal Stem Cells Market in 2024 1.1 GREEN HYDROGEN Industry Analysis 1.2 Key Companies and Product Profiles 1.3 Mesenchymal Stem Cells Market Segments 1.4 Industry Value Chain Analysis 1.5 Market Dynamics- Trends, Drivers, and Opportunities 1.6 Pricing Analysis 1.7 Porter's Five Forces Analysis 1.8 SWOT Profile 1.9 Macro-Economic and Demographic Impact Analysis 1.10 Scenario Analysis

Chapter 2: Global GREEN HYDROGEN Demand Forecasts 2.1 Overview of the Segment 2.2 Global Historic Mesenchymal Stem Cells Market Size (2018-2023) by Types, Applications, and Other Segments 2.3 Global Forecast Mesenchymal Stem Cells Market Size (2024-2030) by Types, Applications, and Other Segments

Chapter 3: Segment-wise Mesenchymal Stem Cells Market Forecasts 3.1 Key Market Segments 3.2 Premium Insights- Largest Types, Applications and Segments 3.3 Premium Insights- Most Lucrative Types, Applications, and Segments

Chapter 4: Mesenchymal Stem Cells Market Outlook by Country 4.1 Mesenchymal Stem Cells Market by Regions 4.2 Mesenchymal Stem Cells Market Revenue Share by Region 4.3 North America (US, Canada, Mexico) 4.4 Europe (Germany, UK, France, Spain, Italy, Russia, Others) 4.5 Asia Pacific (China, Japan, India, South Korea, Australia, South East Asia, Others) 4.6 Latin America (Brazil, Argentina, Chile, Others) 4.7 Middle East and Africa (Saudi Arabia, UAE, Qatar, South Africa, Nigeria, Egypt, Others)

Player Analysis in Chapter Five 5.1 Players' Market Share Analysis (2023) 5.2 Regional Market Concentration Rates 5.3 Business Profiles, SWOT Analysis, Financial Details, Product Portfolio of Companies ..........continued For a comprehensive competitive analysis, Buy this report now and gain access to a detailed table of contents @ https://www.usdanalytics.com/payment/report-9048 Why should you purchase this report? USD Analytics offers essential historical and analytical data on the global Mesenchymal Stem Cells Market. The report thoroughly evaluates future market trends and potential changes in market behavior. It provides various strategic business methodologies to support informed business decisions. Gain a competitive advantage in the market with this detailed research report, which covers competitive landscape analysis, growth drivers, applications, market dynamics, and other In conclusion, the Mesenchymal Stem Cells Market report is a genuine source for accessing the research data which is projected to exponentially grow your business. The report provides vital information including economic scenarios, benefits, limits, trends, market growth rates, and figures. Further, SWOT analysis and PESTLE analysis are also incorporated in the report. Review the Executive Report: @ https://www.usdanalytics.com/industry-reports/mesenchymal-stem-cells-market Thanks for reading this article; You can also get individual chapter-wise sections or region-wise report versions like North America, Middle East, Africa, Europe, MENA, LATAM, and Southeast Asia.

Contact Us: Harry (Business Consultant) USD Analytics Market Phone: +1 213-510-3499 sales@usdanalytics.com

About Author: USD Analytics is a leading information and analytics provider for customers across industries worldwide. Our high-quality research publications are connected market. Intelligence databases and consulting services support end-to-end support our customer research needs.

This release was published on openPR.

Continued here:
Mesenchymal Stem Cells Market to Witness an Outstanding Growth by 2030 - openPR

Recommendation and review posted by Bethany Smith

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Are any specific respiratory viruses more severe than others in recipients of allogeneic stem cell transplantation? A ... - Nature.com

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Roughly 20 years ago, a biologist named Caroline Gargett went in search of some remarkable cells in tissue that had been removed during hysterectomy surgeries. The cells come from the endometrium, which lines the inside of the uterus. When Gargett cultured the cells in a petri dish, they looked like round clumps surrounded by a clear, pink medium. But examining them with a microscope, she saw what she was looking for two kinds of cells, one flat and roundish, the other elongated and tapered, with whisker-like protrusions.

Gargett strongly suspected that the cells were adult stem cells rare, self-renewing cells, some of which can give rise to many different types of tissues. She and other researchers had long hypothesized that the endometrium contained stem cells, given its remarkable capacity to regrow itself each month. The tissue, which provides a site for an embryo to implant during pregnancy and is shed during menstruation, undergoes roughly 400 rounds of shedding and regrowth before a woman reaches menopause. But although scientists had isolated adult stem cells from many other regenerating tissues including bone marrow, the heart, and muscle no one had identified adult stem cells in the endometrium, Gargett says.

Such cells are highly valued for their potential to repair damaged tissue and treat diseases such as cancer and heart failure. But they exist in low numbers throughout the body and can be tricky to obtain, requiring surgical biopsy or extracting bone marrow with a needle. The prospect of a previously untapped source of adult stem cells was thrilling on its own, says Gargett. And it also raised the exciting possibility of a new approach to long-neglected womens health conditions such as endometriosis.

Before she could claim that the cells were truly stem cells, Gargett and her team at Monash University in Australia had to put them through a series of rigorous tests. First, they measured the cells ability to proliferate and self-renew and found that some of them could divide into about 100 cells within a week. They also showed that the cells could indeed differentiate into endometrial tissue and identified certain telltale proteins that are present in other types of stem cells.

Gargett, who is now also with Australias Hudson Institute of Medical Research, and her colleagues went on to characterize several types of self-renewing cells in the endometrium. But only the whiskered cells, called endometrial stromal mesenchymal stem cells, were truly multipotent, with the ability to be coaxed into becoming fat cells, bone cells, or even the smooth muscle cells found in organs such as the heart.

Around the same time, two independent research teams made another surprising discovery: Some endometrial stromal mesenchymal stem cells could be found in menstrual blood. Gargett was surprised that the body would so readily shed its precious stem cells. Since they are so important for the survival and function of organs, she didnt think the body would waste them by shedding them. But she immediately recognized the findings significance: Rather than relying on an invasive surgical biopsy to obtain the elusive stem cells shed identified in the endometrium, she could collect them via menstrual cup.

More detailed studies of the endometrium have since helped to explain how a subset of these precious endometrial stem cells dubbed menstrual stem cells end up in menstrual blood. The endometrium has a deeper basal layer that remains intact and an upper functional layer that sloughs off during menstruation. During a single menstrual cycle, the endometrium thickens as it prepares to nourish a fertilized egg, then shrinks as the upper layer sloughs away.

Gargetts team has shown that these special stem cells are present in both the lower and upper layers of the endometrium. The cells are typically wrapped around blood vessels in a crescent shape, where they are thought to help stimulate vessel formation and play a vital role in repairing and regenerating the upper layer of tissue that gets shed each month during menstruation. This layer is crucial to pregnancy, providing support and nourishment for a developing embryo. The layer and the endometrial stem cells that produce its growth also appear to play an important role in infertility: An embryo cant implant if the layer doesnt thicken enough.

Endometrial stem cells have also been linked to endometriosis, a painful condition that affects roughly 190 million women and girls worldwide. Although much about the condition isnt fully understood, researchers hypothesize that one contributor is the backflow of menstrual blood into a womans fallopian tubes, the ducts that carry the egg from the ovaries into the uterus. This backward flow takes the blood into the pelvic cavity, a funnel-shaped space between the bones of the pelvis. Endometrial stem cells that get deposited in these areas may cause endometrial-like tissue to grow outside of the uterus, leading to lesions that can cause excruciating pain, scarring, and, in many cases, infertility.

Researchers are still developing a reliable, noninvasive test to diagnose endometriosis, and patients wait an average of nearly seven years before receiving a diagnosis. However, studies have shown that stem cells collected from the menstrual blood of women with endometriosis have different shapes and patterns of gene expression than cells from healthy women. Several labs are working on ways to use these differences in menstrual stem cells to identify women at higher risk of the condition, which could lead to faster diagnosis and treatment. Menstrual stem cells may also have therapeutic applications. Some researchers working on mice, for example, have found that injecting menstrual stem cells into the rodents blood can repair the damaged endometrium and improve fertility.

Other research in lab animals suggests that menstrual stem cells could have therapeutic potential beyond gynecological diseases. In a couple of studies, for example, injecting menstrual stem cells into diabetic mice stimulated the regeneration of insulin-producing cells and improved blood sugar levels. In another, treating injuries with stem cells or their secretions helped heal wounds in mice.

A handful of small but promising clinical trials have found that menstrual stem cells can be transplanted into humans without adverse side effects. Gargetts team is also attempting to develop human therapies. She and her colleagues are using endometrial stem cells those taken directly from endometrial tissue rather than menstrual blood to engineer a mesh to treat pelvic organ prolapse, a common, painful condition in which the bladder, rectum, or uterus slips into the vagina due to weak or injured muscles.

The condition is often caused by childbirth. Existing treatments use synthetic meshes to reinforce and support weak pelvic tissues. However, adverse immune reactions to these materials have led these meshes to be withdrawn from the market. Gargetts research so far conducted only in animal models suggests that using a patients own endometrial stem cells to coat biodegradable meshes could yield better results.

Despite the relative convenience of collecting adult multipotent stem cells from menstrual blood, research exploring and utilizing the stem cells power and their potential role in disease still represents a tiny fraction of stem cell research, says Daniela Tonelli Manica, an anthropologist at Brazils State University of Campinas. As of 2020, she found, menstrual stem cell research accounted for only 0.25 percent of all mesenchymal cell research, while bone marrow stem cells represented 47.7 percent.

Manica attributes the slow adoption of menstrual stem cells in part to misogynistic ideas that uteruses are outside the norm and to reactions of disgust. Theres certainly something of an ick factor associated with menstrual blood, agrees Victoria Male, a reproductive immunologist at Imperial College London who coauthored an article about uterine immune cells in the 2023 Annual Review of Immunology.

Cultural taboos surrounding menstruation and a general lack of investment in womens health research can make it difficult to get funding, says Gargett. Immunologist Male has faced similar challenges it was easier to obtain funding when she used to study immune cells in liver transplantation than it is now that she works on immune cells in the uterus, she says.

If we want more research on menstrual fluid, we need more funding, says Male, noting that the logistics of collecting menstrual fluid over multiple days can be expensive. For that to happen, we have to tackle sex and gender bias in research funding. Through more equitable investments, she and others hope, menstruation will be recognized as an exciting new frontier in regenerative medicine not just a monthly inconvenience.

This article originally appeared in Knowable Magazine, an independent journalistic endeavor from Annual Reviews. Sign up for the newsletter.

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The Real Reason Studying Menstrual Blood Is So Important - Inverse

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At the 50th Annual EBMT Meeting, clinicians and researchers highlighted advancements in the blood and bone marrow transplant field, including significant advances centering on criteria for donor selection in allogeneic stem cell transplantation (ASCT) and ongoing efforts to refine outcomes and reduce complications in patients with graft-versus-host disease (GVHD), according to Mohamad Mohty, MD, PhD.

In an interview with OncLive, Mohty highlighted key takeaways from the meeting, emphasizing the discussions that revolved around donor selection criteria, as well as the impact that haploidentical donors and other donor sources may have on patients' needs.

The 2024 EBMT Meeting was a very exciting meeting; we celebrated the 50th anniversary of the European Society for Blood and Marrow Transplantation [EBMT], which was originally founded in 1974, Mohty said. This EBMT Annual Meeting proved to be very successful, and we [saw] the greatest advances in this field.

In the interview, Mohty discussed the implementation of post-transplant cyclophosphamide for GVHD prophylaxis, highlighted efforts to optimize cyclophosphamide dosing to minimize complications when treating this disease, and noted future directions for this research.

Mohty is a professor of hematology, as well as the head of the Hematology and Cellular Therapy Department at the Saint-Antoine Hospital and Sorbonne University in Paris, France. He is also a member and lead of the translational research team at the Saint-Antoine Research Center and is the chairman of the Acute Leukemia Working Party for the EBMT.

Mohty: What was striking for me were the criteria for choosing donors [and] the implementation and [expansion] of the use of posttransplant cyclophosphamide [PTCy] for GVHD prophylaxis. There are also novelties in the management of acute, especially steroid-refractory, but also ruxolitinib-refractory, GVHD. Weve seen some great advances when it comes to cGVHD, and overall, this was a memorable EBMT meeting where milestone data were presented.

When it comes to choosing a suitable stem cell donor [for] ASCT, there is a lot of excitement about the use of haploidentical donors. When combining matched sibling donors, fully matched unrelated donors, mismatched unrelated donors, and now haploidentical donors, youre able to find a suitable donor for approximately 95% of patients who need an ASCT. Although the use of cord blood cells has decreased over the past few years in the adult population, if you use cord blood cells [with] the new technologies and platforms for cord blood expansion, you would be able to cover approximately 100% of [patient] needs.

Interestingly, when it comes to living donors, the age of the donor remains an important parameter. Favoring a young donor, whether in the setting of haploidentical or unrelated donors, is likely to be an important parameter. Also, having a female donor to a male recipient remains an important risk factor for alloreactivity, especially in cGVHD. What remains controversial is how to choose between a young haploidentical donor, if available, and a young, fully matched, unrelated donor. Here we dont have a final response to this question.

There are teams who now rely almost exclusively on family donors, and this would include matched sibling donors and haploidentical donors. There are still teams who prefer to have fully matched 10 out of 10 unrelated donors. This is proving to be very exciting, and despite the debates, the good news is [there is a possibility of] finding a donor for almost everybody who needs an ASCT.

Age of the donor is important when selecting a given donoron the other hand, you must take into account the age of the recipient [too]. When it comes to the recipients age, it is very important to consider the comorbidities, which will play an important role in the final outcome. We know from daily clinical experience that a patient who is 60 years oldbut has comorbidities such as hypertension, diabetes, a heavy smoking history, and cardiovascular complications[will be affected by] these comorbidities. [They will] negatively impact the incidence of nonrelapse mortality.

However, a patient whos 70 years old but also fit and without any comorbidities [will] have a lower likelihood of developing toxicities. We can see an important separation between biological age vs physiological age, which is correlated with comorbidities. The development of comorbidity indexes and trying to use new electronic artificial intelligence tools to try to predict outcomes are going to be major [focuses] of future research.

The use of posttransplant high-dose cyclophosphamide has, in the past 15 years, proved to be a major breakthrough in ASCT because it has allowed safe haploidentical stem cell transplantation [to be performed for the first time, thus] allowing for engraftment while reducing the risk of severe aGVHD and cGVHD.

The use of PTCy has rapidly spread because it is a relatively cheap drug and physicians are quite familiar with the use of cyclophosphamide, as it is a very common drug in hematology and oncology. It has great efficacy in depleting the alloreactive T cells. Now there is an accumulating body of evidence explaining the mechanism of action that allows PTCy to effectively prevent GVHD.

[Although] the initial experience started in the haploidentical setting, use of PTCy is spreading beyond haploidentical transplant and is being used increasingly in the mismatched unrelated setting. Some teams are also using it in the unrelated matched sibling setting. These are the positives with use. We also know with some long-term follow-up and greater experience that high-dose PTCymay not be suitable or safe for everybody because there is a risk of cardiac complications, which were described especially in patients with cardiovascular risk factors. We also know [there is] the high risk of hemorrhagic cystitis related to the BK virus.

At the EBMT meeting, we saw very nice translational work showing that PTCy can impact the immune repertoire and immune anti-infectious controlthat can explain the higher incidence of infectious and opportunistic infections after PTCy. With this background, efforts are aiming to test lower doses of PTCy, and our team presented data on reducing the dose by 20% or even 30%.

For instance, [we used] 35 mg/kg per day for 2 days on day 3 and day 4 or day 3 and day 5. You can achieve similar GVHD prevention while reducing the risk of complications, especially cardiac [and] infectious complications, but also shortening the duration of neutropenia and thrombocytopenia. This needs to be validated in prospective future randomized trials, but the use of PTCywill continue to grow. This is something that will make the ASCT procedure safer and more feasible, especially in older and frail patients.

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Mohty Shares Key Takeaways From the 50th Annual EBMT Meeting, Highlighting Advances in GVHD - OncLive

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