Dive Brief:

The fields of synthetic lethality (a combination of DNA defects that work together to destroy a cancer cell) and DNA repair, fueled by gene editing technologies such as CRISPR/Cas9, have only been around a couple of decades but they are creating a solid foothold in basic research and are now moving into active development.

Repare Therapeutics is using its high-throughput, genomic synthetic lethal screening platform, which includes CRISPR/Cas9 genome editing, to find and exploit DNA damage repair (DDR) defects found across virtually all cancers.

"Versants commitment to and confidence in Repares distinct science has enabled the company to build the team, operations and initial programs away from the spotlight,"said Repare CEO Lloyd M. Segal. "With the added leadership of MPM and this syndicate, we are financed to achieve our goal of testing our multiple new, precision oncology therapeutics in a clinical setting."

CRISPR/Cas9 is becoming a busy field, with a number of companies and groups edging towards the clinic. The first in human study of CRISPR/Cas9 was approved by a Federal panel of the National Institutes of Health in mid-2016. The University of Pennsylvania study will target myeloma, melanoma and sarcoma. This was beaten to the clinic by Chinese scientists, who gave a patient with aggressive lung cancer cells edited using CRISPR/Cas9 in October 2016.

Editas Medicine expected to begin clinical trials by the end of 2017 for Leber congenital amaurosis type 10, a rare eye disorder, but these have now been delayed until mid-2018 because of issues with a third-party manufacturer. This is in development in collaboration with Allergan.

CRISPR Therapeutics expects to file a European clinical trial authorization for beta-thalassemia by the end of 2017, using CRISPR techniques to create variants that artificially induce hereditary persistence of fetal hemoglobin (HPFH), an asymptomatic and naturally-occurring condition that has been linked with better outcomes in people with beta-thalassemia and sickle cell disease.

Intellia Therapeutics, CRISPR Therapeutics and Editas Medicine all floated in 2016, though share prices for Intellia and Editas slumped towards the end of the year.

Continued here:

Celgene-backed CRISPR company emerges with $68M Series A - BioPharma Dive

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We've seen how CRISPR/Cas9 can be used to tackle HIV and cancer, and now the revolutionary gene editing technique has Huntington's disease in its sights, as scientists have used it to reverse signs of the condition in mice.

That suggests CRISPR/Cas9 might one day be able to do the same in humans, so we can push Huntington's higher up the list of priorities for future research.

Huntington's disease is a fatal, inherited condition where brain cells die off due to a toxic protein released by a mutant version of the Huntingtin gene (mHTT). Symptom onset is typically in early middle age, making it a devastating illness at a time when victims are often parents of young children.

It's that mutant mHTT gene that CRISPR/Cas9 could fix, according to the researchers from Emory University.

The new approach could "efficiently and permanently eliminate" the poisoning of the brain that leads to Huntington's, the researchers write.

If you're completely new to CRISPR, or clustered regularly interspaced short palindromic repeats, it enables scientists to "cut and paste" DNA data with great accuracy. Cas9 refers to one particular way of using CRISPR that's currently being explored. Other recent research usesCas3 to attack antibiotic-resistant superbugs.

Potentially, bad genetic code responsible for diseases could be cut out, and healthy genetic code could be pasted in instead. We haven't got that far yet in humans, but it's a goal researchers are working towards.

With mice engineered to have the same mutant Huntington's-causing gene as humans, the scientists used CRISPR/Cas9 to snip out the gene and remove the flow of the toxic protein that eventually leads to problems with motor control and mood swings.

After three weeks, almost all traces of the damaging protein had disappeared.

What's more, the treated mice showed "significant improvements" in their motor control, balance, and grip strength, though they didn't get back up to the same levels of mobility and dexterity shown by the control mice used in the experiments.

That suggests the nerve cells were able to heal themselves to some extent after the troublesome gene had been snipped out by CRISPR/Cas9.

The team says the technique would not have to be customised to each patient's genome, making it easier to apply, although they do stress that a lot more testing and research is required before we'll be sure this is healthy for humans to try.

Clinical trials are already underway for gene-silencing drugs that would block off the protein that causes Huntington's, but the advantage of CRISPR/Cas9 is that it could provide a one-time fix for the disease, with no further treatment required.

"Given that CRISPR/Cas9 can permanently eliminate the expression of targeted genes, using CRISPR/Cas9 should more efficiently deplete the expression of mHTT than has been possible with previous therapeutic approaches, which require continuous administration," the team saysin its paper.

Neurodegenerative diseases in humans have not yet been tackled by CRISPR/Cas9, as the complexity and delicacy of the brain means any kind of tinkering could have catastrophic consequences.

Scientists want to be sure they're on the right track first, which is where this latest research can help, showing one approach that might be effective.

The research has been published in the Journal of Clinical Investigation.

Continue reading here:

CRISPR could point to a cure for Huntington's disease, suggests ... - ScienceAlert

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Sweden aims to establish a new Center for Advanced Medical Products (CAMP) as part of a SEK 320-million ($36.6-million), 8-year Swedish government initiative to position the country as a leading biologics developer.

Swedish regenerative medicine firm Xintela has been appointed a partner in the 6-year project to establish the CAMP cell and gene therapy research center, with SEK 48 million ($5.5 million) in funding from the countrys innovation agency and research council, Vinnova and Vetenskapsrdet. Xintel said that as one of the CAMP initiative founders, it will work with Swedens universities, research institutes, and with firms including AstraZeneca, GE Healthcare and Pfizer. Xintela will initially act as an advisor for development of the center, but in the longer term expects to benefit from emerging R&D.

It is gratifying that the Swedish government, Vinnova and Vetenskapsrdet acknowledge the huge potential of cell and gene therapy and the strong position that Sweden has in this research field,commented Xintela CEO Evy Lundgren-kerlund. Xintela is one of the companies in Sweden with large development potential in cell therapy, which makes us a natural partner for this project.

In the short term CAMP aims to establish itself as an internationally recognised center for R&D, innovation and clinical practice, and to promote industrial growth and SMEs. Longer-term goals include attracting investment from the global pharmaceutical and biotech sectors.

Xintela is exploiting its XINMARK protein marker technology and XACT (Xintela assay for cell therapy) assay platform to develop an allogeneic mesenchymal stem cell-based therapy for repairing cartilage damage in osteoarthritis, and to progress a tumor-targeting antibody treatment for glioblastoma.

Last month the firm established a collaboration with Germany-based CO.DON, which develops autologous cell therapies for cartilage repair. The firms will work together on the development of Xintelas markers both for a next generation CO.DN cell therapy program and for Xintelas cartilage repair cell therapy product.

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Sweden Launches Initiative to Establish Center for Cell and Gene Therapy Research - Genetic Engineering & Biotechnology News

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bluebird bio today disclosed its first data, including some promising results, from a pair of clinical studies assessing its LentiGlobin gene therapy candidateincluding a Phase III study that followed a change in its manufacturing process.

In the Phase III Northstar-2 (HGB-207) study of LentiGlobin in patients with transfusion-dependent -thalassemia (TDT) and non-0/0 genotypes, early interim data showed that the first 3 of 15 patients treated to date achieved higher drug product vector copy number (DP VCN) and lentiviral vector positive (LVV+) cell production than in the earlier Northstar (HGB-204) study, bluebird said.

Those results are correlated with higher production of hemoglobin A (HbA)T87Q and ultimately may address known patient-to-patient variability, bluebird bio CMO David Davidson, M.D., said in a statement.

The first patient treated in this study exemplifies the promise of gene therapy: discontinuing blood transfusions approximately a month after treatment and achieving a normal level of total hemoglobin production at six months post-treatment, Dr. Davidson stated.

According to bluebird, the first patient achieved normal total hemoglobin (13.3 g/dL) after discontinuing transfusions; producing 9.5 g/dl of HbAT87Q at last follow-up.

bluebird cautioned that the data was early, and that not all three patients enjoyed results as positive as the first patient. The second patient showed a lower percentage of LVV+ cells (53%) than either patients 1 (77%) or 3 (77% and 82%). However, neither patients 2 or 3 had sufficient follow-up to record clinically relevant data for total hemoglobin or days since last transfusion.

Northstar-2 is an ongoing, open-label, single-dose, international, multicenter study designed to evaluate the safety and efficacy of LentiGlobin drug product for the treatment of patients with TDT and non-0/0 genotypes. As of June 2, the drug product had been manufactured for six patients. The median DP VCN for these patients was 3.0 (range: 2.44.0), compared to a median DP VCN of 0.7 (range: 0.31.5) in Northstar.

Although early, these data add to the growing body of clinical evidence that indicate that LentiGlobin may offer a transformative benefit for patients with TDT, added Alexis Thompson, MD, of Ann & Robert H. Lurie Childrens Hospital of Chicago, a primary investigator on the study.

The Phase III study followed what the company said was an improvement in the manufacturing process by which the patients cells are transduced with the LentiGlobin viral vector, a change aimed at increasing vector copy number and the percentage of cells successfully transduced.

bluebird bio also announced results from its Phase I/II HGB-205 study, designed to assess LentiGlobin in patients with TDT and severe sickle cell disease (SCD). Those results, according to the company, showed the potential for durable treatment effect of LentiGlobin, with stable HbAT87Q production through 3.5 years of follow-up and sustained clinical benefit.

The company cited data from four TDT patients, all of whom remained free of transfusions since shortly after receiving LentiGlobin. At the last study visit, one patient had been free of transfusions for 41.9 months, compared with 38.7 months, 20.3 months, and 20.4 months for the other three patientsthe last of which was also homozygous for the severe + mutation IVS1-110.

In the three patients with SCD, the patient with the best results was producing 50% HbAT87Q , well above the approximately 30% anti-sickling hemoglobin level predicted to have potential clinical impact on the disease, at last follow-up 31.7 months following treatment.

The other two SCD patients showed levels of 20% and 15% at last follow-up (6.1 months and 3.4 months after treatment, respectively.)

HGB-205 is an ongoing, open-label, single-center study in which four patients with TDT, and three with severe SCD, have undergone infusion with LentiGlobin drug product as of June 2.

We are beginning to see evidence of the long-term durability of benefit from treatment with LentiGlobin, with some TDT patients even transitioning off of chelation therapy, said the HGB-205 studys primary investigator, Marina Cavazzana, M.D., Ph.D., a professor of medicine at Paris Descartes University, research director at the Centre for Clinical Research in Biotherapy, Necker Hospital, and at the Imagine Institute of Genetic Diseases in Paris.

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Early Data for bluebird bio Gene-Therapy Candidate Show Some Promise - Genetic Engineering & Biotechnology News

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Photo: Keelin Daly / For Hearst Connecticut Media

Swimmers dive in for the Annual Swim Across America Greenwich-Stamford version in 2015. The funds raised by the event go to cancer gene therapy research. This years swim is set for Saturday.

Swimmers dive in for the Annual Swim Across America Greenwich-Stamford version in 2015. The funds raised by the event go to cancer gene therapy research. This years swim is set for Saturday.

The Annual Swim Across America Greenwich-Stamford in the Sound along the border of Greenwich and Stamford is set this year for Saturday. The funds raised by the event go to cancer gene therapy research.

The Annual Swim Across America Greenwich-Stamford in the Sound along the border of Greenwich and Stamford is set this year for Saturday. The funds raised by the event go to cancer gene therapy research.

Swim Across America Greenwich-Stamford set for Saturday

GREENWICH On Saturday, more than 200 swimmers are expected to take part in the 11th annual Swim Across America Greenwich-Stamford Swim, part of a national effort to raise money and awareness in the fight against cancer.

Event organizers say 100 percent of the proceeds go to the Alliance for Cancer Gene Therapy, which supports cancer cell and gene therapy research.

The local swim is chaired by town residents Michele Graham and Lorrie Lorenz, both of whom have had a child who was diagnosed with cancer. Nicole Graham is celebrating five years cancer free; Brooke Lorenz is six years cancer free.

Out of all the fundraisers we go to, this is the most fun, the most family oriented and the most beautiful, Michele Graham said. Its at a beautiful waterfront setting and people are so happy and joyful to be a part of it. Our vibe is a fun one. We want people to have fun and have it be an event that lifts people up.

Special guest speaker at the event will be Alec Fraser, father of Greenwich High School graduate Julian Fraser, who died of cancer earlier in the year.

Julian Fraser had been captain of GHS swim and water polo teams. He will be remembered at Saturdays swim by Team Julian, led by GHS swim team coaches Terry Lowe and Lorrie Hokayem.

This event attracts a lot of young people who might not otherwise be so directly involved in the fight against cancer, Alec Fraser said. Many of Julian's swim team and water polo teammates participate, both in Stamford and California. Although everyone knows someone who has had cancer, many of the members of Team Julian were his teammates, and so very personally affected by his loss.

Alec Fraser is also swimming in his sons honor.

Although I am not at all a swimmer, it is meaningful to me to be in the water with so many of his friends and former teammates to honor his memory in a sport and venue that was so important to him, the senior Fraser said.

Swimmers will dive into Long Island Sound starting from 96 Cummings Point Road early Saturday right at the border between Greenwich and Stamford. At 7 a.m. the three-mile swimmers will start.

At 8:30 a.m., the mile-and-a-half swimmers the largest group will dive into the water; the half-mile swimmers will jump in at 8:45. Swimmers start to return to shore about 8:50 a.m.; the award ceremony is slated to start at 10 a.m.

Since it started locally, more than $3.1 million has been raised for ACGT by close to 2,000 swimmers totaling 2,500 miles in the water.

Among the participants will be Greenwich resident Andy Alisberg, who is swimming with the team of Peters Defeaters.

Cancer has continued to intrude on the universe of people I know and about whom I care, Alisberg said. Vietor Evans, a childhood of his daughters, his graduate school suite mate Paul Stuka and a friend, Robin Zothian, were among people who have lost their lives to cancer or are fighting the disease.

The memory of those wonderful friends whom we have lost to cancer in recent years burns bright, Alisberg said.

Nationally, the Swim Across America effort has brought in $65 million since it was founded in 1965.

According to the organizations fact sheet, nearly 1.7 million new cases of cancer will be diagnosed and 600,920 people in the United States will die from the diseases in 2017 alone.

More information is online at http://www.SwimAcross

America.org/Greenwich.

kborsuk@greenwich

time.com

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Swim Across America Greenwich-Stamford set for Saturday ... - Greenwich Time

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Off Target Effects

But almost as soon as the technology was introduced, scientists raised concerns about off target effects. Said Xiao-Hui Zhanget al.of the College of Veterinary Medicine, South China Agricultural University and coauthors at MIT in a 2015Molecular TherapyNucleic Acidsarticle, The high frequency of off-target activity (50%)RGEN (RNA-guided endonuclease)-induced mutations at sites other than the intended on-target site is one major concern, especially for CRISPR technology therapeutic and clinical applications.

The growth of any new technology, the authors note, including CRISPR/Cas9, demands progressive enhancement. And while research in CRISPR/Cas9 has made huge strides in the evolution of gene editing, it and other RGENs, which include ZFNs and TALENs, have more severe off-target effects than other nucleases due to their inherent structure and mechanism.

At an American Society of Hematology workshop on genome editing, CRISPR pioneer J. Keith Joung, M.D., Ph.D., of Massachusetts General Hospitalsaid, In the early days of this field, algorithms were generated to predict off-target effects and [made] available on the web miss a fair number of off-target effects. He added, These tools are used in a lot of papers, but they really arent very good at predicting where there will be off-target effects, according toSTAT.

Observations in the recent literature have raised more alarms among CRISPR cognoscenti, all of whom would agree than technical improvements are needed. In one of the most blogged-about papers on CRISPR, Unexpected mutations after CRISPRCas9 editingin vivo, Kellie A. Schaefer and colleagues at Stanford concluded that More work may be needed to increase the fidelity of CRISPR/Cas9 with regard to off-target mutation generation before the CRISPR platform can be used without risk, especially in the clinical setting.

The authors had, they reported in a 2016 study by W.H. Wuet al.inMolecular Therapy,used CRISPR/Cas9 for sight restoration in blindrd1mice by correcting a mutation in thePde6bgene. Mice homozygous for the rd mutation have hereditary retinal degeneration and have been considered a model for human retinitis pigmentosa.

Citing persistent concerns about secondary mutations in regions not targeted by a single guide RNA (sgRNA)concerns also expressed by a number of other scientistsKellie A. Schaefer, Ph.D., at Stanford University and colleagues at Howard Hughes and Massachusetts General Hospital performed whole genome sequencing (WGS) on DNA isolated from two CRISPR-repaired mice (F03 and F05) and one uncorrected control.

CRISPR/Cas9-treated mice were sequenced at an average depth of 50, and the control to 30 to identify all off target mutations. The sequencing the authors said identified an unexpectedly high number of single nucleotide variations (SNVs), contrary to the widely accepted assumption that CRISPR causes mutations mostly at regions homologous to the sgRNA.

CRISPRs penchant for promiscuous behavior has spawned an entirely new research field focused on fixing it. Patents have already been filed on the fixes and the developers believe that these advances will incrementally enable more reliable CRISPER performance. Most efforts have concentrated on modifying CRISPR nuclease Cas9 using structure-design based changes in the enzyme, chemical modifications, and amino acid substitutions at critical sights to better predict and control its function.

Writing inNaturein 2015,Benjamin P. Kleinstiver, Ph.D., of the Molecular Pathology Unit and Center for Cancer Research at the Massachusetts General Hospital and colleagues noted that although CRISPR/Cas9 nucleases are widely used for genome editing, the range of sequences that Cas9 can recognize is constrained by the need for a specific PAM.

The investigators reported that they could modify Cas9 to recognize alternative PAM sequences using structural information, bacterial selection-based directed evolution, and combinatorial design. The altered PAM specificity variants, they said, could edit endogenous gene sites in zebrafish and human cells that are not targetable by wild-type SpCas9. Further, they said, the variants genome-wide specificities are comparable to wild-type SpCas9 as judged by GUIDE-seq analysis and establish the feasibility of engineering a wide range of Cas9s with altered and improved PAM specificities.

Kleinstiver and other investigators working in Joungs also developed the unique endonuclease SpCas9-HF1, which they describe as a high-fidelity enzyme variant with alterations designed to reduce non-specific DNA contacts. The scientists hypothesized that reducing iCas9 and the target DNA interactions might help eliminate off-target effects while still retaining the desired on-target interaction.

Since certain portions of the Cas9 nuclease can itself interact with the backbone of the target DNA molecule, the team altered four of these Cas9-mediated contacts by replacing the long amino acid side-chains that bind to the DNA backbone with shorter ones that could not bind.

SpCas9-HF1 retained on-target activities comparable to wild-type SpCas9 with >85% of single-guide RNAs (sgRNAs) tested in human cells. Notably, with sgRNAs targeted to standard nonrepetitive sequences, SpCas9-HF1 rendered all or nearly all off-target events undetectable by genome-wide break capture and targeted sequencing methods.

Jiang and Doudna pointed out in a 2017 piece inAnnual Review of Biophysicshow Cas9 locates specific 20-base-pair (bp) target sequences within the genomes that are millions to billions of base pairs long and, subsequently, how it induces sequence-specific double-stranded DNA (dsDNA) cleavage remain critical questions, not just in CRISPR biology, but in the efforts to develop more precise and efficient Cas9-based tools.

Molecular insights from biochemical and structural studies such as those describe above will provide a framework for rational engineering aimed at altering catalytic function, guide RNA specificity and PAM requirements, and reducing off-target activity for the development of Cas9-based therapies against genetic diseases.

See the original post here:

Fixing CRISPR - Genetic Engineering & Biotechnology News (blog)

Recommendation and review posted by simmons

The potential of genome-editing techniques, such as CRISPR/Cas9, to alleviate disease burden has ignited the imagination for thousands of researchers looking for new therapeutic strategies.

Now, a group of investigators led by scientists at Emory University is hoping to open up new avenues of neurodegenerative research and rapidly move toward human trials after the release of their new findings. The research team showed that the CRISPR/Cas9 system could snip part of a gene that produces toxic protein aggregates in the brains of 9-month-old mice used as a model for Huntingtons disease. Moreover, the scientists noted that when they looked at the brain region where the vector was applied, some weeks later, the aggregated proteins had almost disappeared. Amazingly, the motor abilities of the mice had improved, although not to the level of control mice.

The study revealed the capacity of brain cells to heal themselves if the genetic source of the toxic proteins is removed. Moreover, in comparison with control Huntingtons mice, CRISPR/Cas9-injected mice showed significant improvements on tests of motor control, balance, and grip strength, although they did not recover to the point where they performed as well as control mice.

[Read the full study here]

The GLP aggregated and excerpted this blog/article to reflect the diversity of news, opinion, and analysis. Read full, original post:CRISPR Reverses Huntingtons Disease in Mice

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Reversing Huntington's? Brain shown to heal itself after disease source edited out in mice - Genetic Literacy Project

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Posted 21 June 2017 By Zachary Brennan

Although the US market is likely more than a year or two away from seeing any commercialized medical products that rely on CRISPR-Cas9 technology, the rapidly developing field has already grabbed the attention of the US Food and Drug Administration (FDA) and other drug regulators.

According to the Broad Institute, CRISPR (pronounced "crisper") stands for Clustered Regularly Interspaced Short Palindromic Repeats, which form the basis for a genome editing technology known as CRISPR-Cas9, which can be programmed to target specific segments of genetic code and edit DNA precisely.

Unlike other genome editing technologies that have entered the clinic, like what Sangamo Therapeutics has been developing, experts say CRISPR-Cas9 is easier.

The potential for such technology to help treat or even cure genetic or other diseases has spurred the creation of a number of different companies, though none of the developing products have begun clinical trials in the US yet.

FDA senior policy adviser Ritu Nalubola explained to attendees at the DIA annual conference in Chicago on Wednesday that the agency is in the early stages of building capacity to regulate treatments that use CRISPR-Cas9 technology, and though they are collaborating with other regulators and working with the National Academies of Science, the cross-border regulation of such treatments seems unlikely.

"Harmonization is not always possible because of the statute," Nalubola said.

And although there is a gene therapy working group within the International Pharmaceutical Regulators Forum that addresses transnational issues, Nalubola said that there's a "lot of research that we cant oversee," and that there's "a role for further public engagement and addressing best practices."

Kurt von Emster, managing partner of the investment firm Abingworth, which made early investments in the Switzerland-based company CRISPR Therapeutics that he said is now worth about $650 million, noted European regulators have so far been more receptive than FDA.

The European Medicines Agency's Committee for Advanced Therapies has discussed the prospects for CRISPR-Cas9 products. And FDA's Office of Pharmaceutical Quality has an Emerging Technology Team that Nalubola mentioned could help companies with early discussions.

And though the initial hope was that CRISPR-based treatments "would cure one or two diseases, I think that's moved up quite a bit," von Emster said, noting that CRISPR Therapeutics has invested more than $80 million in pre-clinical models and still has its sights on entering clinical trials in the US for the first time before the end of 2017. Massachusetts-based Editas Medicine, which was co-founded by the Broad Institute's Feng Zhang, recently delayed the filing of its IND for its lead CRISPR program.

"This is a technology, not a product," von Emster added, noting that CRISPR Therapeutics decided early to partner with companies like Vertex Pharmaceuticals and Bayer on some projects, though "we decided to keep oncology to ourselves."

He also said the next six months will clarify a lot in the CRISPR space, especially as intellectual property (IP) issues get further ironed out. The US Patent and Trademark Office recently upheld a series of patents granted to the Broad Institute for theCRISPR-Cas9technology, which will likely be a win for Editas and the Broad Institute.

But Editas, CRISPR Therapeutics and Intellia Therapeutics, among other companies in the CRISPR-Cas9 space, are moving in different directions, von Emster said.

He also told DIA attendees that "no one should think were pushing Editas aside," as it's "in our common interests to work together to overcome the IP situation to get to the task of curing disease."

But in terms of how to approach regulators with what many believe could be breakthrough treatments, companies are just beginning to test the waters.

"We haven't spent a lot of time approaching FDA because we don't know what our product is yet," von Emster said.

Eva Essig, VP of regulatory affairs at Intellia, noted the importance of early technology platform discussions with FDA as such discussions can help with determining which indications are more plausible to bring forward in development.

And both he and Essig said they had their doubts about a small study in mice that made headlines recently on off-target issues with CRISPR, though they think it's important that different approaches are being undertaken.

For now, a lot of the discussion on which treatments will win approval is still hypothetical.

When asked what news headlines will look like a year from now on CRISPR and gene editing, Essig pointed to human trials conducted in China that will probably start seeing early results.

Read more:

Regulating CRISPR: FDA and Industry Offer Perspective | RAPS - Regulatory Focus

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Versant Venture and MPM Capital co-led a $68-million Series A fundraising round to establish Repare Therapeutics, a Canada-based cancer drug company exploiting a high-throughput, CRISPR-based screening platform to identify synthetic lethality cancer targets and develop precision anticancer therapies.

For the last 18 months, Repare has been working in stealth mode under Versant incubation to develop its synthetic lethality platform and identify initial oncology targets. The firm says a number of programs have now been moved into preclinical development, and clinical trials with an initial candidate could start in 2019.

Additional investors in Repares Series A fundraising included Fonds de solidarit FTQ, Celgene Switzerland, and BDC Capitals Healthcare Venture Fund.

Versants commitment to and confidence in Repares distinct science has enabled the company to build the team, operations and initial programs away from the spotlight, said Repare CEO Lloyd M. Segal. With the added leadership of MPM and this syndicate, we are financed to achieve our goal of testing our multiple new, precision oncology therapeutics in a clinical setting.

Synthetic lethality occurs when the presence of mutations in two specific genes cause cell death, whereas the presence of one of the mutations alone does not. The concept of using synthetic lethality to target cancers is already being exploited through the development of PARP inhibitors for treating cancers with BRCA1 or BRCA1 mutations.

Repares discovery engine exploits CRISPR/Cas9 gene editing, in combination with protein crystallography, computational biology, and clinical informatics, in a high-throughput screening platform that is designed to identify new drug targets that, when blocked, induce synthetic lethality in cancer cells exhibiting commonly found tumor-related mutations.

Repares first disclosed program targets PolQ, which codes for enzyme DNA-directed DNA polymerase theta, a polymerase enzyme that plays a key role in repairing double stranded DNA breaks. Polymerase theta is highly expressed in ovarian, breast and other cancers. The PolQ program is based on drug discovery work carried out by Agnel Sfeir, Ph.D., at NYU School of Medicine, to which Repare has an exclusive license.

Based on Quebec, Canada, and in Boston, MA, Repare is headed by Lloyd M. Segal, who is a Versant entrepreneur-in-residence, who has previously headed Caprion Pharmaceuticals, Advanced Bioconcept, and Thallion Pharmaceuticals. The Repare management team also includes Executive Vice President and Head of R&D Michael Zinda, Ph.D., who previously led the oncology bioscience operation at AstraZeneca in Boston. Repares vice president of discovery, Cameron Black, Ph.D., led Merck Frossts medicinal chemistry efforts for 18 years.

Jerel Davis, Ph.D., managing director at Versant, and Todd Foley, managing director at MPM Capital, will join Repares board of directors. We are impressed by the speed and precision with which Repare, in collaboration with its founders and scientific advisors, generated impressive insights and multiple novel targets, Dr. Davis commented. We evaluated nearly every opportunity in the synthetic lethality space and have complete conviction that Repare, its founders and its SAB members represent the leaders in the field, added Foley.

Read more here:

Repare Raises $68M in Series A to Develop CRISPR-Based Synthetic Lethality Platform - Genetic Engineering & Biotechnology News (blog)

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At 11am on a Sunday morning, I slip into a row of seats in front of a podium with flower bouquets on each side. Im here to listen to an aging white man talk about the afterlife. A woman in a fancy hat arranges a potluck lunch on a back table. Other attendees, mostly gray-haired, pass around a wicker basket and toss in $20 bills and personal checks.

We arent in church. This is godless Silicon Valley.

The Humanist Society has welcomed Ralph Merkle, a Livermore native, to explain cryonicsthe process of freezing ones own body in liquid goo like Austin Powersto the weekly Sunday Forum. We all want to know about being re-awoken, or reborn, in the future.

Merkle, who has a Ph.D. in electrical engineering from Stanford and invented whats called public key cryptology in the 70s, makes his pitch to the audience: hand over $80,000 plus yearly dues to Alcor, and the Scottsdale, Arizona-based company will freeze your brain, encased in its skull, so that you and your memories can wait out the years until medical nanotechnology is advanced enough to both bring you back from a frozen state as well as fix the ills that brought you to death in the first place.

You get to make a decision if you want to join the experimental group or the control group, Merkle says. The outcome for the control group is known.

Alcor gained infamy in 2002, when the body of baseball legend Ted Williams was flown to the companys Arizona headquarters, where his head was then severed, frozen and, according to some reports, mistreated.

The Humanist Society is an ideal audience for Merkles presentation, as its congregants arent held back by the tricky business of believing in a soul. Debbie Allen, the perfectly coiffed executive director and secretary of the national board of the American Humanist Association, considers cryonics as a practical tool. Religion has directed the conversation for thousands of years, she says. Allen prefers to focus on ethics, and whether cryonics advances the well-being of the individual or the community.

Science fiction, someone whispers behind me, as Merkle talks about nanorobots of the future. He also notes how respirocytes and microbivores can be programmed to run around inside a cell and do medically useful things like make you healthy.

As one might expect in a room full of humanists, skepticism runs high during the Q&A portion of the meeting. People are wondering exactly what kind of animals the scientists have used to test the cryonics process (the answer: nematodes), when Alcor freezes bodies (after ones heart stops if a DNR, or Do Not Resuscitate, order is requested), whether a frozen brain is any good if the rest of the body deteriorates (Toss it, Merkle says. Replacement of everything will be feasible.) and what happens if Alcor goes bankrupt.

We take that very seriously, the doctor says.

Lunch is served.

Why would he want to preserve somebody like Adolf Trump? asks Bob Wallace, 93, who ate salad and cubed cheese with his partner, Marge Ottenberg, 91, whom he met at a Humanist Society event.

Obviously, the worst possible people are most likely to want to live forever, says Arthur Jackson, 86, a retired junior high school teacher.

Ottenberg seems more open to the idea of coming back from the dead than her golden-year counterparts. Whatever works, she says.

Silicon Valley is the sort of place where people dream about nanorobots fixing our medical disorders. Its the sort of place where hundreds of millions of dollars are spent chasing that dream.

The last five years have seen an investment boom in whats called life extension research. Some of it is straight-up science, such as the Stanford lab researching blood transfusions in mice to cure Alzheimers. Scientists are in a race against time to help as many people as possible, as fast as possible. Theyre battling a disease that saw an 89 percent increase in diagnoses between 2000 and 2014, and Alzheimer's or other dementia is currently the sixth leading cause of death. There are also nontraditional sources of cash flowing into biotech, which was once considered a risky investment.

But death, itself, is the biggest social ill Silicon Valley is trying to solve.

We can build apps to keep track of diabetics' blood glucose levels, to measure how soundly we're sleeping and access medical records in an instant, but none of this stops the body from wearing out. Alongside the scientists laying the medical foundation to get us to the nanorobots envisioned by Merkle, techie utopians are looking at other ways to cheat death. A cluster of tech companies are attracting far more funding from Silicon Valley than academia, shifting the research landscape with infusions of cash.

Bryan Johnson, an entrepreneur who sold his online payment company to PayPal for $800 million, was the first investor in Craig Venters Human Longevity, Inc., which aims to create a database of a million human genome sequences, including people who are over 100 years old, by 2020. Oracle founder Larry Ellison, who once said death makes me very angry, and is one of the oldest of the life-extension investors at 70, has also invested in Human Longevity. Johnson infused even more cash into the biotech field, investing another $100 million of his own money into the OS Fund in 2014 to support inventors and scientists who aim to benefit humanity by rewriting the operating systems of life.

Such projects are examples of Silicon Valleys extreme confidence in its own ability to improve the world. In an email, Johnson describes his work in grandly optimistic terms.

Humanity's greatest masterpieces have happened when anchored in hope and aspiration, not drowning in fear, he says.

It takes some serious chutzpah to say youll extend the human lifespan, and for Johnson, he and his colleagues are venturing where no one has gone before.

Building good technology is an act of exploration, and that it is very difficult for us to imagine the good that might come from any new technology, Johnson says. We proceed, as explorers, nonetheless.

Online payment entrepreneur Bryan Johnson was one of the first people to invest in Human Longevity, Inc., which aims to extend life by rolling back the aging process. (HCM Media, via Wikimedia Commons)

Johnsons lofty goals are similar in scale to other giant anti-aging investments in Silicon Valley. In 2013, Google created an anti-aging lab called Calico (for California Life Company), hiring top scientist Cynthia Kenyon, known for altering DNA in worms to make them live twice as long as they usually do. Calico is not your local university research lab; it has $1.5 billion in the bank and has remained close-lipped about its progress, like a Manhattan Project for life extension.

For Google co-founder Sergey Brin, 43, Calico may be another way to attack a more personal health concern: Brin carries a gene that increases his likelihood of contracting Parkinsons disease and has already invested $50 million in genetic Parkinsons research, conducted by his ex-wifes company, 23andMe. Brin said in 2009 that he hoped that medicine could catch up to cure Parkinsons before hes old enough to develop it.

That hope is a common thread among health-obsessed tech investors like PayPal founder Peter Thiel, 49. A libertarian and Trump adviser, Thiel is trying to avoid both death and taxes. His foundation hired a medical director, Jason Camm, whose professional goals include increasing his clients prospects for Optimal Health and significant Lifespan Extension. Like Brin, who swims and drinks green tea to prevent Parkinsons, Thiel has changed his daily habits to live longer. Hes aiming for 120, so he avoids refined sugar, follows the Paleo diet, drinks red wine, and takes human growth hormone, which he believes will keep bones strong and prevent arthritis.

Thiel has also expressed personal interest in a company called Ambrosia in Monterey, where Dr. Jesse Karmazin is conducting medical trials for a procedure called parabiosis, which gives older people blood plasma transfusions from people between 16 and 25. Karmazin has enrolled more than 70 participants so far, each of whom pays $8,000 for the treatment. Much has been made of Thiel harvesting and receiving injections of young people's blood, though Karmazin recently denied that Thiel was a client of his.

Karmazin doesnt call himself utopian, but he does note that his work requires some faith. Theres always uncertainty about whether its going to stand the test of time, whether itll work at all, he says. Thats especially true in technology, and you have to believe in it.

At the same time, the dystopians of Silicon Valley are preparing for the apocalypse. Reid Hoffman, CEO of LinkedIn, told the New Yorker that he guesses up to 50 percent of tech executives have property in New Zealand, the hot new hub for the end of the world. Steve Huffman, CEO of Reddit, bought multiple motorcycles so he can weave through highway traffic if theres a natural disaster and he needs to escape. He also got laser eye surgery so he wouldnt have to rely on glasses or contacts in a survival scenario.

Among the dystopians is Elon Musk, whose brand-new Neuralink company is investigating what Musk calls neural lace, a digital layer on top of the brains cortex that connects us to computers. Such inventions could eventually lead us to what Google Director of Engineering Ray Kurzweil calls technological singularity, or the time when ever-more-powerful artificial intelligence will surpass human intelligence, around 2045. Musk is nervous about that day, and part of the reason he wants to colonize Mars through his SpaceX plan is because humans need an escape route in case computers take overor, perhaps, in case of environmental apocalypse. Musk recently quit two of President Donald Trumps business advisory councils over Trumps decision to leave the Paris climate accords, tweeting Climate change is real.

Tech companies as a bloc urged Trump not to leave the Paris agreements; Tim Cook of Apple called him after the announcement to try to get him to change his mind, and Mark Zuckerberg wrote on his Facebook page that leaving Paris would put our childrens future at risk.

Zuckerberg has been trying for years to knock down four houses to build a residential compound in Palo Alto, including a basement structure that sounds like a bunker, with dark steel doors and windows and a dark grey standing seam metal roof, perfect for hiding the whole family if the world ends.

Whether climate change destroys California or regular old death arrives before investors have funded a cure, Musk, Zuckerberg and their elite peers have the resources to plan an escape. The question is whether theyre interested in planning anyone elses.

Tony Wyss-Coray, director of the Stanford Alzheimers Research Center, which is on the forefront of anti-aging research, has seen that conflict up close.

I have been approached by billionaires from LA and Texas, and they already have their clinics in the Bahamas or wherever, where they inject themselves with stem cells, he says.

But those billionaires werent interested in funding his lab or curing disease for anyone else.

Theyre interested in living, Wyss-Coray says. They realize quickly they cant buy this directly from Stanford University.

The line between science and someones obsession with mortality is blurry, especially with this much cash flowing.

Its hard to completely disassociate the influence of wealthy, rich people from what we do, Wyss-Coray says. Until the recent influx of funding and attention, the anti-aging scientists he knew were just a bunch of academic geeks studying worms. Hes interested not in extending life as much as figuring out why certain people can live past 100 years old.

The average person at 60 or 65 starts to suffer from a multitude of age-related diseasesarthritis, heart disease, cognitive declinethat for some reason the centenarians seem to be able to escape from, and thats what drives many of us in the field.

Peter Thiels interest in extending his life has led to wild speculation on just how far he would go. (Photo by Dan Taylor of Heisenberg Media, via Wikimedia Commons)

But when Thiel is reading ones research, things get more complicated. Wyss-Corays studies on the benefits of parabiosis in mice, for example, form the basis of the Monterey trial that so fascinates Thiel. Wyss-Coray is quick to distance himself from Karmazin. He cites all our work on his website, Wyss-Coray says.

The first two studies in the Science section of the Ambrosia website are from Stanfords labs, and the first study Karmazin lists about plasma transfusions in mice is Wyss-Corays.

Many scientists consider clinical trials like Karmazins unethical and scientifically unsound, since they require participant payment for unproven treatments, and you cant charge someone $8,000 for a placebo, so theres no simultaneous control group. The Ambrosia trial passed an ethical review, but Karmazin acknowledges the criticism.

Some people are opposed to it for ethical reasons, he says. Thats understandable, but I still think its worth doing, so Im trying to treat people.

Wyss-Coray is ambivalent about his research being exploited for profit. You contribute a small piece to knowledge that frequently can be abused by somebody, he says. I feel somewhat guilty, but I hope at the same time, we can contribute to maybe having an impact on some diseases, and that will be offset.

Back under the fluorescent lights at the Humanist Society, Merkle explains that in addition to freezing themselves, people can use Alcor as a bank, putting money aside so that they dont wake up poor in 100 years. Future poverty is a common enough concern that Merkle includes it in his presentation. Why would anyone want to live forever if it meant working three jobs to survive?

Indeed, people who are struggling to pay rent right now wont be able to afford to freeze themselves, so anyone waking up from cryogenic sleep will be wealthy, and most of them will be white, just like the bros pioneering biotech startups and building underground bunkers. Indeed, about 75 percent of Alcors frozen customers are male, and Max More, its CEO, is a libertarian like Thiel. The men who have everything want to keep it all, indefinitely.

Income inequality makes life extension the ultimate oligarchical fantasy. A month before Gawker shut down last year, bankrupted by Thiels campaign against it, reporter J.K. Trotter mused, Its not hard to imagine a Thielist future in which members of the overclass literally purchase the blood of the young poor in order to lead longer, healthier lives than their lesser counterparts can afford.

In Thiels libertarian universe, the luckiest people could live forever, feeding on the blood of Silicon Valleys youthful underclasshey there, San Jose renters!and living on extra-governmental barges like the seasteads Thiel dreams about, without paying taxes to help anyone else. Floating cities might be helpful if flooding and erosion destroy the California coastline, as CalMatters Julie Cart reported could happen 70 years from now.

Taking the scenario a little further, birth would be unnecessary, since no death would mean no one would need to be replaced. That might make people with wombs a little less than necessary, as well, especially if those barges are populated with the new crop of alt-right dudes who sleep with men because they worship masculinity.

Baseball legend Ted Williams, right, had his dead body turned over to Alcor so the company could freeze his head and, presumably, bring him back to life in the future. (Photo by NASA, via Wikimedia Commons)

Thiel, who is gay, would probably find it preferable to get by without women; he considers date rape as belated regret and once blamed womens voting rights for the eventual demise of democracy. His worldview is the warped conservative version of feminist theorist Donna Haraways Cyborg Manifesto, in which she imagined the freedom in a world without genesis, but maybe also a world without end.

Back in 1984, the author predicted a future where we merged with machines, but warned against letting racist, male-dominant capitalism control technology, since hippie progressives are not cheerleading the convergence of humans and machines.

It might all sound far-fetched, but Thiel shares an anarcho-capitalist worldview with White House senior adviser Steve Bannon, among the most powerful people in America right now. And the House passed a health care law that saves money on insurance by letting poor people die faster, moralizing that poor people dont want to be healthy.

Californians may not agree with that law outright, but Silicon Valleys bootstrappy cult of health is based on the nerds association between fitness and brainpower. Theyre taking up kiteboarding, tracking steps on Fitbits and eating ketogenic diets during stressful times at startups. Its not a big jump to life extension for the rich, who deserve to live longer after all that effort.

Are the ethics of life-extension technology any different from historical questions of who gets access to medicine? Maybe not.

Karmazin hadnt yet considered the topic before our phone call. I havent had this kind of conversation with anyone yet, he says. But Karmazin compares his trial to the introduction of antibiotics. Someone who didnt have access to antibiotics when they were invented? Man, theyd probably be really upset. Thats reasonable. He foresees similar problems with blood plasma as a cure for aging: I think its going to be unevenly distributed.

Wyss-Coray has serious concerns about that distribution.

We have enough problems in the world already, and I definitely do not want a select group of people to live longer just because they can afford it, he says.

In this country, the richest 1 percent in the U.S. live 15 years longer than the poorest 1 percent, meaning Wyss-Corays fear is already our reality. The question is how much worse things can get, and whether a medically assisted longer life will be inaccessible to almost all of us.

Thats assuming, of course, that we even want a longer life, or to wake up after a cryogenic sleep. We may value our time on Earth, but not everyone thinks its worth it to stick around indefinitely.

If your Silicon Valley brain sees the world as a place of obstacles that can always be overcome, where every system can be disrupted for the better and your brain is the one that will unlock a better future, you might be more inclined to stay. That might also be true if you think the universe is a place to conquer, whether via spaceship to Mars a la Musk or through politics like Thiel.

But what might the future look like, for those who want to (and can afford to) stay?

Googles Kurzweil envisions three medical stages before singularity, starting with our current push to slow aging. Stage two: building on genomic research, including personalized fixes for diseases like cancer. Kurzweil believes well get to the medical nanotechnology that Merkle envisions by the 2030s, which would lead us to the last phasenanorobots connecting us to the cloud in 2045. At that point, avatars of our brains could be loaded into another body. Then wed live forever.

Bodily ailments would be curable and wed access consciousness from the cloud, but wed still lose our memories when our physical brains stopped working. A better (and still terrifying) option might be freezing our brains via cryonics and then bringing them back with nanorobots.

Kurzweil has signed himself up to be frozen, in case the 90 supplements he takes daily dont keep him alive.

Wyss-Coray, of Stanford, has chosen not to go into the meat locker. I cant think of any way to connect that to what were doing, he says. I havent signed up for that myself.

Neither have most other people. Cryonics remains unproven, cost prohibitive and unusually creepy to the general population, an option for the rich and famous who would need several lifetimes to see their savings run dry. At this rate theyll likely outlive us, so we might as well enjoy some refined sugar, pay our taxes and stop fearing the reaper.

The rest is here:
Brotopia: How the Valley's Tech Elite Plan to Outlive the Rest of Us - San Jose Inside (blog)

Recommendation and review posted by sam

Sergio Canavero, the Italian surgeon who audaciously plans to perform the worlds first human head transplant within the next 10 months (pending the availability of a donor body) is now preparing to reawaken cryogenically frozen brains and transplant them into someone elses skull.

In an interview with a German-language magazine, Canavero says he will attempt to bring the first brainsfrozen in liquid nitrogen at an Arizona-based cryogenics bank back to life not in 100 years, but three years at the latest.

Transplanting a brain only and not an entire head gets around formidable rejection issues, Canavero said, sincethere will be no need to reconnect and stitch up severed vessels, nerves, tendons and muscles as there is when a new head is fused onto abrain-dead donor body.

Canavero allows that one problematic issue with brain transplants, however, would be that no aspect of your original external body remains the same.

Your head is no longer there, your brain is transplanted into an entirely different skull, he told OOOM magazine, published by the same company that handles the Italian brain surgeonspublic relations.

The flamboyant neuroscientist who some ethicists have decried as nuts rattled the transplant world when he first outlined his plans for a human head transplant two years ago in the journal, Surgical Neurology International.

Bioethicist Arthur Caplan called Canaveros latest proposal to merge head transplants with resurrecting the frozen dead beyond ridiculous. People have their own doubts about whether anything can be salvaged from these frozen heads or bodies because of the damage freezing does, said Caplan, head of ethics at NYU Langone Medical Centre in New York City.

Then saying that he has some technique for making this happen, that has never been demonstrated in frozen animals, is absurd.

Caplan accused the maverick surgeon of playing to peoples fantasies, that somehow you can come back from death, fantasies that you can live forever if you just keep moving your head around and to fears science is out of control. Thats why I pay attention to him.

According to Canavero, the greatest technical hurdle to a head transplant is fusing the donor and recipients severed spinal cords, something never before achieved in humans, and restoring function, without causing massive, irreversible brain damage or death.

In an exclusive interview with the National Post last year, Canavero said what makeshisbrazen, and critics say ethically reckless, protocolpossible isa special fusogen, a waxy, glue-like substance developed by a young B.C.-born chemist that will be used to reconnect the severed spinal cord stumps and coax axons and neurons to regrow across the gap.

Canavero said the first head transplant will be performed in Harbin, China, and the surgical team led by Xiaoping Ren, a Chinese orthopedic surgeon who participated in the first hand transplant in the U.S. in 1999. Ren has been performing hundreds of head transplants in mice in preparation.

The first patient will be an unidentified Chinese citizen, and not, as originally planned, Valery Spiridonov, a 31-year-old Russian man who suffers from a rare and devastating form of spinal muscular dystrophy.

Canavero called Ren a close friend of mine and an extraordinarily capable surgeon.

At the moment, I can only disclose that there has been massive progress in medical experiments that would have seemed impossible even as recently as a few months ago, Canavero told OOOM. The milestones that have been reached will undoubtedly revolutionize medicine.

He declined to offer up exactly what those milestones are, saying that results of the most recent animal experimentshave been submitted for publication in renowned scientific medical journals.

Last September, the team reported they had succeeded in restoring functionality and mobility in mice with severed spinal cords using the special fusogen, dubbed Texas-PEG. Canavero claims the mice were able to run again.

Your head is no longer there, your brain is transplanted into an entirely different skull

He said numerous experiments have been conducted since then on an array of different animals in South Korea and China and the results are unambiguous: the spinal cord and with it the ability to move can be entirely restored, he told OOOM.

Canavero envisions the head (or, perhaps more accurately, body) grafting venture as a cure for people living with horrible medical conditions. The plan is to cut off the head of two people one, the recipient, the other, the donor whose brain is dead but whose body is otherwise healthy, an accident victim for example. Surgeons will then shift the recipients head onto the donor body using a custom-made swivel crane. They will have less than an hour to re-establish blood supply before risking irreversible brain damage.

In a few months we will sever a body from a head in an unprecedented medical procedure, Canavero said. At the moment of decapitation, the patient will be clinically dead. If we bring this person back to life, we will receive the first real account of what actually happens after death, he told the magazine, meaning, he said, whether there is an afterlife, a heaven, a hereafter or whatever you may want to call it or whether death is simply a flicking off of the light switch and thats it.

Canavero said a brain transplant has several advantages over a head-swap, including that there is barely any immune reaction, which means the problem of rejection does not exist. The brain is, in a manner of speaking, a neutral organ, he said.

Others are hugely skeptical of the prospect of reawakening brains, or bodies, frozen after death. In an interview with the Posts Joe OConnor two years ago, Eike-Henner Kluge, a bio-ethicist at the University of Victoria, refers to cryonics patients as corpsesicles.

Unless it is technically possible, and it is not, to replace all the water left in a bodys cells with glycol, unfreezing a frozen corpse will rupture the cell walls ensuring that you are mush a corpsesicle.

However, two years ago researchers with 21st Century Medicine, a California cryobiology research company, reported they had succeeded in freezing a rabbits brain using a flash-freezing technique to protect and stabilize the tissue. After the vitrified brains were rewarmed, electron microscope imaging from across the rabbit brains showed neurons and synapses were crisp and intact.

Canavero hopesto get his first brains from Alcor Life Extension Foundation in Scottsdale, Ariz. Alcors most famous patient is Red Sox baseball legend Ted Williams, the greatest hitter in baseball history, whose head was detached from his body and cryopreserved after his death at 83 in 2002.

Email: skirkey@nationalpost.com | Twitter:

Read the rest here:
The plan to 'reawaken' cryogenically frozen brains and transplant them into someone else's skull - National Post

Recommendation and review posted by simmons

NobleGnthon has teamed up with ignominious Sarepta to develop a gene therapy for the Duchenne variety of the muscle wasting disease.

Dedicated to rare diseases since 1990 and more recently to gene therapy, Gnthon is one of veryfew not-for-profit companies in European biotech. Though it has not yet brought a drug to market, it is well established as a nonprofit, credit for which is due toits creator, the French Muscular Dystrophy Association, AFM Tlthon. The company has nowteamed up with the (in)famous American company, Sarepta, to work on a gene therapy for Duchenne Muscular Dystrophy (DMD).

DMD first affects the shoulder and upper arm muscles and the muscles of the hips and thighs. (Source: mda.org)

When I last spoke to Gnthons CEO,Frdric Revah, he told me that while the majority of the companys financial support comes from Tlthon, an increasing amount comes fromsuch partnerships.We get more and more support from industrial partnerships as we outlicense more of our drugs, explains Revah. However, Tlthon will always remain the main source of our funding; the funding from out-licensing is a complement.

But is Sarepta the best partner? While the American biotech can boast about its FDA-approved drug for DMD, Exondys 51, (which is just sold to Gilead for $125M,) this achievement isdubious:not only was the key clinical trial tiny, the FDAadmittedthat patients did not receive aclear benefit fromthe drug in the study. These circumstances prompted arenowned journalist to suefor thedocuments pertaining to the decision, an attack thatSareptas stock into a downward spiral in May.

Nevertheless, under the terms of the partnership agreement,Gnthon will trust Sarepta as a potential co-developer ofits DMD candidates, all of which are preclinical. The French biotechhas been developing amicro-dystrophin gene therapythat has proven itself applicable to the disease. As it countswith Europes largest cGMP vector manufactory, YposKesi, andone of the worlds largest research and clinical groups developing rare disease therapies, Gnthon is an attractive partner for any companies prospecting in the field.

Sareptas pricing practices may also offendthe sensibilities of a nonprofit, since theyraised the hackles of the drug pricing patrol with a plan to charge $300k per year for Exondys 51. According to STAT News, the companysCEO, Edward Kaye, saidthis figure isin the middle of the range for rare disease drugs,and given the sensitivity to pricing, we tried to be reasonable when looking at all the costs.

Though financial details of the agreement have not been disclosed, Gnthon may have enough influence to sway Sareptaaway from gouging.Our main goal is to ensure that patients have access to drugs and that they are affordable. Price should not be an obstacle,Revah told me.

Whatever we do here, we hope to apply the same tech to diseases that affect more people, like sickle cell anemia and cancer,he continued. With a crowded arena of companies like CRISPR Therapeutics, AMO Pharma, andDebiopharmall battling to bring the next DMD drug to market, having a back up plan via a platform seemssensible.

Images via Alila Medical Media, Anatomy Insider / shutterstock.com

Original post:
French Nonprofit Partners with Big American Name to Advance a Gene Therapy for Muscular Dystrophy - Labiotech.eu (blog)

Recommendation and review posted by sam

Two research reports from the 2017 American Urological Associations annual meeting, May 12-16 in Boston, demonstrated that treatment with the novel 4.5% nasal testosterone gel, (marketed as NatestoR), restored total serum testosterone to normal levels while preserving normal concentrations of pituitary gonadotropins in men with hypogonadism1, and led to clinical improvements in both erectile function and mood. 2

In an open-label, dose-ranging study, reported by William Conners, MD, from Harvard Medical School and Mens Health Boston, hypogonadal subjects were randomized to self-administer NatestoR either twice daily (BID) (n=122) or 3 times daily (TID) (n=151), for a total daily dose of 22.0 mg or 33.0 mg, respectively, over 90 days. Titration was based on mens blood levels, aiming to achieve the eugonadal range of 300-1050 ng/dL. Serum samples were obtained from subjects at baseline and after 90 days. 1

The treatment resulted in an increase in total serum testosterone from a mean Cavg 200.8 ng/dL at baseline to a mean Cmax 818.49 ng/dL at about 40 minutes.

After 90 days, 90% of men in the TID group, and 71% of men in the BID group reached normal testosterone levels, and a mean total testosterone Cavg 421 ng/dL and 375 ng/dL, respectively.1

Baseline follicle-stimulating hormone (FSH) in the BID group was 8.49 IU/L: the mean at day 90 FSH was 5.99 IU/L. Baseline FSH in the TID daily group was 6.42 IU/L. The mean at day 90 was 3.12 IU/L. Baseline luteinizing hormone (LH) in the BID group was 5.42 IU/L. The mean at day 90 was 3.56 IL/L. The baseline TID group was 5.25 IU/L. The mean at day 90 was 2.20 IU/L. 1

The authors concluded that treatment with the 4.5% nasal testosterone gel, NatestoR, restored serum total testosterone to normal levels. Although FSH and LH levels were reduced, they noted that mean levels remained well within the normal range, in a finding that contrasts with other therapies for hypogonadism, injections in particular. 1

In another evaluation of the efficacy and safety of the 4.5% nasal gel, NatestoR, authors led by Dr. Larry Lipshultz, from Baylor University College of Medicine, reported the extent of clinical improvements in erectile function and mood, in a 90-day, randomized, open-label, dose-ranging study in hypogonadal men, with sequential safety extensions to one year. 2

The investigators evaluated erectile function and mood at baseline (day 0), and at 30-day intervals throughout the 90-day treatment period using the International Index of Erectile Function (IIEF) and the Positive and Negative Affect Schedule (PANAS).

Results demonstrated that treatment with NatestoR resulted in statistically significant improvements in each of the 5 domains of erectile function (F(3,8,13) =83.96, p<.001). Most of the benefit was evident by day 30 (t= -9.8714, df=288, p- value <.001), with smaller increases until completion of the study (Figure 1)

NatestoR also produced clinically and statistically significant improvements in mood, as assessed by PANAS, by day 30. Continued improvements in mood were seen through the studys completion (Figure 2).

Gerwin Westfield, PhD, Medical Affairs Manager with Aytu BioScience, spoke with UroToday about these results, which are unique when compared with effects of other forms of testosterone therapy, especially by a notable lack of suppression of pituitary gonadotropins LH and FSH.

Men who were treated with NatestoR nasal testosterone gel had significant improvements in mood as early as 30 days, and continued improvement all the way out to Day 90representing both significant improvements in the positive mood attributes and significant improvement in negative mood attributes. This finding was quantified by the self-reporting instrument PANAS, (Positive and Negative Affect Schedule).

Moreover, said Dr. Westfield, treatment with NatestoR showed statistically significant improvement in all five domains of erectile function as early as Day 30, and the improvement continued to Day 90.

References: 1. Conners W, Morgentaler A, Guidry M, Westfield G, Bryson N, Goldstein I. Preservation of normal concentrations of pituitary gonadotropins despite achievement of normal serum testosterone levels in hypogonadal men treated with a 4.5% nasal testosterone gel. American Urological Association. May 12-16, 2017. Poster MP89-06. 2. Lipshultz LI, Westfield G, Guidry M, et al. Clinical improvements in erectile function and mood in hypogonadal men treated with 4.5% nasal testosterone gel. American Urological Association. May 12-16, 2017. Poster PD69-06.

More here:
Nasal Testosterone Gel Shown to Normalize Androgen Levels, Improve Erectile Function & Mood in Hypogonadal Men - UroToday

Recommendation and review posted by sam

Lipocine Inc (NASDAQ:LPCN) is considerably higher in mid-afternoon trading today, currently up 27 cents at $4.29 a boost of nearly seven percent. After the market close yesterday, the company released positive data from a dosing validation as well as a dosing flexibility study evaluating its testosterone replacement therapy (TRT).

The news has also sent Lipocines volume up today, as more than 7.4 million shares of the biotech have changed hands already. On a typical day, only about 164,000 shares of Lipocine end up getting traded.

The companys TRT, called LPCN 1021, was developed for adult males suffering from symptoms associated with a deficiency of endogenous testosterone a conditioned referred to as hypogonadism. According to Lipocines press release yesterday, the dosing validation and flexibility trials confirmed a fixed dose approach moving forward. Additionally, researchers will not need to do dose titration to orally administer the drug.

LPCN 1021 managed to successfully meet the FDAs regulations in the dosing validation study, as 81 percent of the enrolled patients hit average testosterone levels that fall within the normal range. In the dosing flexibility study, 70 percent of the trials subjects had average testosterone levels restored confirming a twice-per-day dosing regime for resubmission moving forward.

We are pleased with the confirmation of LPCN 1021 efficacy, especially with a more practical patient and physician preferred no titration dosing regimen, said the companys Chairman, President, and Chief Executive Officer, Dr. Mahesh Patel. We believe the results should address the label-related deficiency cited by the FDA in our NDA submission.

Dr. Patel told shareholders that he hopes that LPCN 1021 can be a differentiated TRT option for hypogonadism patients that will both improve patient compliance and reduce risk of testosterone interference. The company plans on resubmitting their New Drug Application (NDA) in the third quarter of the current year.

The author of this article holds no position in any of the companies mentioned above.

Here is the original post:
Lipocine (LPCN) Spikes On Testosterone Dosing Data - Economic Calendar

Recommendation and review posted by simmons

Here we have compiled a list of several clinics offering stem cell treatments. Please note that the "conditions treated" refers to the conditions that THEY claim to treat. Most, if not all, stem cell treatments (except hematopoietic stem cell transplantation) aren't FDA approved, meaning that they haven't been clincally tested for safety or efficacy. Please be aware that receiving an unapproved medical treatment isrisky and may cause serious complications and possibly death.

It was only a few years ago when Europe's most popular stem cell clinic (XCell-center) was forced to close after one of the treatments caused the death of a boy. In the past, we have also covered the case of a woman that had serious adverse effects following an unapproved cosmetic stem cell treatment(facelift).

We have not included clinics offering hematopoietic stem cell transplantation, as this treatment is medically approved and offered virtually in any country that has an above the average hospital.

The stem cell clinics are categorised by alphabetical order. We are not paid by any of them and we have listed them for your ease. We have probably missed a few ones, feel free to leave a comment and we will add them asap.

Stem cell clinics list

Beijing Puhua International Hospital

Conditions Treated:Diabetes, Epilepsy, Stroke, Ataxia, Spinal Cord Injuries, Parkinson's Disease, Brain Injury, Multiple Sclerosis, Batten's Disease

Interview of a patient treated in Beijing Puhua International Hospital. The video is from the hospital's official youtube channel, so it may be biased

Elises International

Conditions Treated: No info available at their website

Advertisement video ofElises International

EmCell

Conditions Treated:ALS, Alzheimer's,Anemia, Cancer, Eye Diseases, Diabetes, Liver Diseases, Multiple Sclerosis Parkinson, and other

Location:Ukraine

EmCell Advertisement

Global Stem Cells

Conditions Treated:Type 2 Diabetes, Hepatitis C, Osteoarthritis, joint pain, hair regrowth, cosmetic anti-aging, ulcerative colitis, heart disease

Location:Bangkok Thailand

MD Stem Cells

New Zealand Stem Cell Clinic

Stem Cell Institute

Video of a patient treated in theStem Cell Institute. The video is taken from the clinic's official youtube channell,so it may be biased.

Okyanos Heart Institute

Conditions Treated:Cardiac conditions

Okyanos Promotinal Video

Stemedix, Inc

Conditions Treated:Multiple sclerosis, COPD, ALS, Alzheimers Disease, Parkinsons, Diabetes, Rheumatoid Arthritis and other

Location:Florida, United States

StemGenex

Conditions Treated: Multiple sclerosis, Alzheimer, Parkinson, Diabetes, Rheumatoid Arthritis and other

Location:San Diego, California.

Stem Cells Thailand

Conditions Treated:Alzheimer, Autism, Diabetes, Erectile Dysfunction, Face lift, Multiple Sclerosis, Arthritis and other

Regennex

Conditions Treated: Regennex mainly offers treatments for bone and cartilage regeneration in all major joints like knee, ankle, hip, back, shoulder etc

Dr. Centeno, founder of the clinic, talking about Regenexx

More here:
Stem Cell Clinics List | Stem Cells Freak

Recommendation and review posted by Bethany Smith

Founded in 2004, LifeCcell has technological collaboration with the US-based Cryo-Cell Internationalthe worlds first private stem cell bank with over 25 years of experience. (PTI)

Chennai-based LifeCell, the provider of preventive healthcare services for family wellness, is the worlds second-largest provider of umbilical cord stem cells. Founded in 2004, the company has technological collaboration with the US-based Cryo-Cell Internationalthe worlds first private stem cell bank with over 25 years of experience. As many as 2 lakh Indian parents have chosen to trust their newborns umbilical cords to LifeCell through its umbilical cord banking service BabyCord. The company has a 60% share in the Indian market.Stem cells are mother cells that have the potential to become any type of cell in the body. One of the main characteristics of stem cells is their ability to self-renew or multiply, while maintaining the potential to develop into other types of cells. These cells can repair and rebuild damaged tissue. The uses of stem cells are still being researched. In fact, stem cell tissues have proved effective in cancer treatment too. The applications have been steadily increasing in the last few years. They have been used for treating wound healing, including diabetic foot ulcers. In a country where concepts like bone marrow donations and stem cell banking are still not widely known, Mayur Abhaya, the CEO and managing director of the company, is betting on these treatments of the future.

The company is the most accredited stem cell bank in the country, with certifications from national and international organisations for standards. It is also the only player in the industry providing comprehensive stem cell solutions, including menstrual stem cell banking, R&D and point-of-care stem cell therapy for orthopaedic and vascular specialities.Mayur has been heading LifeCell since 2008. He comes from the family that set up Shasun Group of companiesthe provider of contract pharmaceutical manufacturing services for global companies. Mayur studied biotechnology in India and the US, and then worked in the US for a year. Before moving to LifeCell, he worked for many years at Shasun Pharmaceuticals, where he led their new product development, intellectual property and licensing initiatives. In 2013, LifeCell International got an investment of Rs35 crore from Helion Venture Partners, an India-focused venture fund, to support its plans of increasing market penetration of stem cell banking in India and enabling the development of novel cell-based therapies.

Also Watch: Mayur says LifeCell currently operates in 150 cities, employing more than 1,500 people. We have given an opportunity to our sales people to become their own bosses. They remain on company rolls and get to enjoy all the company benefit plans, such as insurance and welfare schemes. They grow with the company and also have the opportunity to explore and add non-conflicting products or services to their distribution network and enhance their earnings. These internal franchisees bring 50% of our revenue and it is growing. More than 50 such entrepreneurs have been created.LifeCell recently bought over the stake held by Helion Ventures with borrowings from family-owned firms. Three months ago, it changed its business model. We are introducing an on-demand model for sharing cord blood cells, Mayur says. Parents can let the company know if their babies cord blood cells can be used for other needy patients. Cancer patients cannot be treated with their own stem cells. Patients usually do not have much time. Cord cells can be used even if all the six parameters that are required to transplant tissues do not match. By letting their stem cells be used by others, parents and their children get access to cord blood cellsof the entire cord blood cell bankwhen they are in need. So far, stem cells were banked only for the baby from whom these were removed.

Our inventory will come to the aid of people who do not have babies. We will refund the amount paid for having their babys stem cell stored. The processing fee is Rs17,000 and the storage fee each year is Rs4,000. Mayur says that the worlds largest birthing country has a long way to go to create a viable stem cell bank. We are going to follow the blood bank model and hope to bank 2,50,000 cords, which is the critical amount, he adds. We hope to contribute significantly to the ever-developing scope of transplant medicine. Currently, India is importing cord cells, which are prohibitively expensive. With scale, prices will come down in the country. Parents in India will have higher future access to stem cells than even those enjoyed by patients in advanced countries such as the US. We will have a linkage with global inventory. Earlier this month, LifeCell was invited by AABB (formerly American Association of Blood Banks) to present the concept of Community Stem Cell Banking at the 15th International Cord Blood Symposium held in San Diego, US. In 15 years, it is the only second stem cell bank to present its innovation at such a prestigious global platform.

With a turnover of Rs126 crore, LifeCell is operationally profitable. It has enough cash to run its business, but is yet to make net profits. However, Mayur believes very soon LifeCell will turn profitable, and that this year the number of stem cells brought into the labs will be higher by at least 30%. Mayur has extended LifeCells services to introduce and popularise the concept of essential preventive diagnostics for mothers and babies. BabyShield has been introduced to bring down infant mortality ratio. Addressing gaps in marketplace and with innovative business models, it has established market leadership in newborn screening. It has also acquired a prenatal screening service provider. In India, only 2% babies go through prenatal and newborn screening. Nobody has focused on this. We will also be providing diagnostic medication. Doing this can prevent so many false positives. We are building all this together as a package and are offering it at an affordable price, he says.

Read more:
How LifeCell became the most accredited stem cell bank in India - Financial Express

Recommendation and review posted by sam

Elizabeth Nega knows that time is not on her side.

The Vancouver mom was diagnosed with acute lymphoblastic leukemia in February and she needs a bone marrow transplant.

Finding a suitable donor has been difficult.

Im very lucky if I get that person who [is a] match to me, she said. My brother and my sister didnt match. If somebody matches, thats a miracle.

She has been undergoing regular chemotherapy treatments since her diagnosis.

The first time they told me if I get 90 per cent remission, they will treat it for two years with chemotherapy, then I will be fine, Nega said. Unfortunately, it didnt show 90 per cent remission.

Nega, who goes by the name Elsa, and her family set up a website and social media channels to help in the search for a donor.

READ MORE: I need a man: Ethnic donors desperately needed for bone marrow registry

Nega, who was born in Ethiopia, needs a match from someone of African descent, which could prove to be a challenge.

Ethnic diversity in the stem cell donor bank is woefully limited, according to Trudi Goels of Canadian Blood Services.

Our registry is about 69 per cent Caucasian, which is huge and it doesnt really reflect the diversity in Canada accurately, she said.

So what we really want to encourage is people who are of African descent to come forward and register to support Elsa.

The ideal candidate is between 17 and 35 years of age. Men are preferred, but women are just as welcome.

Nega hopes a candidate can be found quickly. Her doctor said she can only do about two more chemotherapy treatments before theyll have to stop.

If I get bone marrow transplant, that means another chance to survive, she said. A second chance in my life. If I get that I will survive. Thats my thinking, thats my hope.

2017Global News, a division of Corus Entertainment Inc.

See the original post here:
BC woman in need of bone marrow transplant pleas for more donors of African descent - Globalnews.ca

Recommendation and review posted by simmons

If DNA were an essay and genes individual words, CRISPR would be the keyboard: adding, deleting and editing to create, for instance, drought-resistant crops.

PANAMA CITY Imagine a world where scientists could manipulate an organisms DNA with enough precision to reliably edit a single gene, creating drought-resistant crops or eliminating mosquitoes that carry malaria.

You actually dont have to imagine that hard. The technology already exists and this fall, it will be taught in lab courses at Gulf Coast State College (GCSC).

Biology professors and science teachers from Bay District Schools spent their day Tuesday learning about a cutting-edge gene manipulation called CRISPR clustered regularly interspaced short palindromic repeats which essentially uses a particular bacterias immune system to edit DNA on the singular gene level. If DNA were an essay and genes individual words, CRISPR would be the keyboard: adding, deleting and editing.

Understandably, it has everyone in the biotechnology field talking.

CRISPR itself is causing a revolution in molecular biology, said Ben Stephenson, a former Arnold High graduate who, after graduating from GCSC and the University of Florida, returned to help teach his former professors this new method. There are medicines and all types of technologies with different applications that are coming about just because the system is so amenable to innovation.

In fact, the possibilities are so mind-boggling that even scientists like Stephenson havent considered them all. Almost any time Stephenson talks to someone about CRISPR, they inevitably come up with an idea or application he hasnt thought of.

Its a technology that gets people excited, he said. It will get kids excited.

Carrie Fioramonti, a natural sciences teacher at Gulf Coast who was participating in the workshop, said the faculty at the college already is brainstorming how they can incorporate CRISPR into their classes. Similar to when smartphones became more accessible and people became more interested in learning how to code, CRISPR, which is in a sense biological coding, has the potential to draw in people who arent trained in biology but are interested in the application. Do-it-yourself CRISPR starter kits already are available on Amazon for about $165, and Stephenson said bio-hacker clubs have sprung up in several major cities.

This is one of the hottest, fastest-growing fields of technology, Fioramonti said.

But as with many scientific fields that have exploded suddenly, regulations havent quite kept up. Gene editing comes with serious ethical implications, and while their samples were in the centrifuge, several of the participants in Tuesdays workshop chatted about experiments on embryos in Sweden and China using CRISPR to edit human genes. The experimentsdidnt go well, and CRISPR isnt quite understood well enough to use on humans yet, but the ethical implications are there.

Beyond the human question, there also is the question of whether such a precise gene-editing tool should be so widely available. According to the 2015 NY Magazine article The Crispr Quandary, the methods co-inventor, Jennifer Doudna, has questioned whether the technology should be available to students, as a minor mistake could have major implications, or a mutation might be accidentally introduced into the wild and affect a whole ecosystem.

While that concern is valid, Stephenson said the CRISPR kits, and anything being taught in a lab, would be fairly self-contained and largely harmless. The kits usually use an innocuous bacteria to test on, and on Tuesday, participants experimented with sea anemone DNA, adding a gene for fluorescence.

Many of the teachers at the workshop said they were amazed at how far biotechnology had come since they were in school, remembering when gene manipulation was a cumbersome, imprecise process. Even Stephenson, who just finished grad school, has to work to keep up, and he said most students now are starting college with a better understanding of CRISPR than he had after he graduated.

This is what our students are going to be going to school for, so we need to give them what they need, Fioramonti said of future labs using CRISPR.

Original post:

Gulf Coast State College looks to incorporate CRISPR into labs - The News Herald

Recommendation and review posted by sam

Two major biotech companies have criticised a recent study that claimed CRISPR may cause hundreds of unwanted mutations.

Scientists from the CRISPR-based firms Intellia TherapeuticsandEditas Medicinehave written open lettersto the journal Nature Methods, stating that the conclusions of the study are 'unsubstantiated'.

'Our opinion is that the authors failed to sufficiently control the reported study in such a way that one could conclude that CRISPR induces the observed mutations,' said Editas'letter, co-signedby 13 of the company's scientists. 'In our view, the genetic differences seen in this comparative analysis were likely present prior to editing with CRISPR.'

DrNessan Bermingham, CEO of Intellia, has called for the paper to be retractedon grounds offlawed design and interpretation. Both firms' share prices have been negatively impacted by the study, andsome companies saw afall of up to 15 percent.

The study reported the discovery of over 100 large deletionsorinsertions in the genome of two mice which had previously undergone CRISPR genome editing. These had not been anticipated by other widely used methods to detect the accuracy of the technique, the authors claimed (see BioNews 903).

The studyhas gathered a lot of attention since its publication last month. A number of scientists have highlighted flaws within the research, including the small number of animals and misidentification of off-targets effects. Many have offered alternative explanations for the findings.

Talking to The Scientist, Dr Gatan Burgio,a geneticistfrom the Australian National University, Canberra, points out that the use of published sequence data rather than non-edited control animals kept in the same laboratory conditions may mean that the study was unable to 'tease out which variants are due to the natural genetic variation and which ones are CRISPR-related.'

In response to the criticism, a spokesperson at Springer Nature, which publishesNature Methods, told MIT Technology Review, 'We are carefully considering all concerns that have been raised with us and are discussing them with the authors.'

Scientists are aware that using CRISPR will sometimes result in unintended genomic changes. 'Getting people to talk about the need for controls is a good outcome of this whole thing. I think the data is perfectly fine. It's just the interpretation of it that to me seems odd,' saidDr Matthew Taliaferro,who studies genome editing at Massachusetts Institute of Technology.

Co-author of the study in question,Dr Vinit MahajanofStanford University, told The Scientistheremains 'very excited' about the potential of CRISPR, though admits that they cannot be completely certain that it caused all of the observedmutations. However, 'if you make an observation that's important enough to share with your community, you're obligated to do that right away,'hetold WIRED.

Link:

Biotech companies criticise CRISPR mutation study - BioNews

Recommendation and review posted by sam

SAN FRANCISCO, Calif. Mayo Clinic Ventures has partnered with a California-based company to make cancer-fighting gene therapies available to the public.

Vineti, a pioneering cell and gene therapy software and analytics company, announced Tuesday that it had completed its initial round of funding raising $13.75 million aimed at delivering "the first cloud-based software solution to improve patient access, accelerate life-saving treatment delivery, and promote safety and regulatory compliance for individualized cell therapies."

The funding was provided by Mayo Clinic Ventures, GE Ventures, DFJ and LifeForce Capital. It's just the 15th company that Mayo Clinic Ventures has backed since it was formed, according to Andy Danielson, vice chairman of Mayo Clinic Ventures.

"One thing with Vineti that we liked is that we have a commitment to cell and gene therapies at Mayo," Danielson told TechCrunch.com. "Vineti will make the gene and cell therapy production process more efficient and as a result, less costly. It's all part of the equation of making these therapies more affordable and opening them up to a greater number of people."

The targeted cancer therapy under development by Vineti is part of a thriving field that conducted more than 800 clinical trials in 2016 while investing nearly $6 billion. It's all aimed at positively impacting the oncology field, the largest market in medicine that's expected to grow to $165 billion by 2021.

The first two cell therapies are expected to hit the market later this year.

Vineti touts its plans as one that "integrates logistics, manufacturing and clinical data to improve product performance overall and enable faster, broader access for patients."

"Physicians, medical researchers and pharmaceutical companies are working together to develop successful therapies, transitioning from a one-size-fits-all model to individualized treatments for each patient," said Amy DuRoss, CEO at Vineti. "Now, the process for creating and delivering these treatments can be as innovative as the therapies themselves. We are developing the Vineti platform to help these treatments reach the patients who need them the most, and are confident the partnership between our advances technologies and leading medical research will deliver better outcomes across the globe."

Continue reading here:
Mayo Clinic Ventures funds new cancer-fighting cell, gene therapy - Post-Bulletin

Recommendation and review posted by sam

At least three hormonal replacement therapy clinics have opened in Cy-Fair since March. Jason Starr, owner of Mens T Clinic, said the trend could be credited to a growing awareness of benefits.

Starr left an 18-year corporate career with Mattress Firm to open Mens T Clinic in Houston in November 2015 and another in Cypress this March after seeing positive results from testosterone replacement.

Mens testosterone levels decrease as they age, and thats not new, he said. What is newer is this notion that men no longer have to accept it.

Starr, 42, said men and women are having children later in life: He has a 2-year-old and a 4-year-old. In the last five years, pharmaceutical giants have taken notice and invested in hormone replacement gels and injections, he said.

Hormone clinics have been opening up because clients are looking for convenience and specialization, Starr said. Men can have blood work done in the on-site lab and see their results in about half an hour.

Our medical staff is equipped to deal with a wide array of issues, he said. Weve discovered tumors in people in the short time weve been open. Were not just about injecting someone with one medication.

Treatment plans can be personalized and are monitored regularly to ensure safety and efficiency, Starr said. Most clients range in age from 35 to 65 and come every week or two for hormone injections.

Starr said the stigma around his industry is treatment is about sexual dysfunction. Although this can be a factor for some men, other outcomes include feeling younger, having more energy and being able to burn more fat and build more muscle mass.

We hear about how peoples weekends used to be about catching up on rest, staying on the couch, he said. Now theyre about getting out and living their life, playing with their children, being more involved in organizations. Weve also seen people come in six months later having lost 25 pounds.

According to the Mayo Clinic, risks of testosterone therapy include sleep disorders, skin reactions, prostate growth, enlarged breasts, limited sperm production, blood clots and heart disease. Risk levels can depend on age, medical history and family history, so the clinic advises both men and women to speak with a doctor before engaging in hormone therapy.

If someone has a pre-existing condition that testosterone could accelerate or is treating himself without monitoring his hormone levels, the therapy can become a risk, Starr said.

Youre substituting a naturally made hormone in your body with a synthetic version of it, he said. Were continually monitoring to make sure were treating people safely and effectively.

Karilyn Barnett opened BeBalanceda hormone therapy practice focused on womenon Hwy. 290 in April. Similar to Starr, Barnett opened the clinic after finding success in the program herself.

I saw my family physician right before I found [BeBalanced], and I had hit a plateau for several weeks, Barnett said. She looked at me and said, Sometimes you just have to accept the fact that youre getting older. She didnt have any answers.

Barnett said she had heavy menstrual cycles as a young girl, and she was diagnosed with endometriosis in her 20s. This led to infertility, and she had a hysterectomy to address the issues, which led to other problems, including a 60-pound weight gain.

Many women come into the clinic at a loss because exercising and dieting does not always deliver weight loss results, Barnett said.

I had worked from home for the last 15 years, which allowed me to hide and harbor my weight, she said. It got to the point where I didnt want to do anything socially or leave the house, and I had anxiety and depression.

About a year and a half ago, Barnett started researching career opportunities outside the home and stumbled upon franchising for BeBalanced online. Skeptical at first, she went through treatment to learn more and quickly began to feel better about herself after using homeopathic creams that support glandular systems and balance progesterone levels.

Within the first month, Barnett said she lost 18 pounds, no longer experienced anxiety or depression, stopped having hot flashes and did not crave sugar anymore. Results were not only immediate, but they were also long-lasting, she said.

Our centers are designed to be a place where women can share some of the things theyve been experiencing, she said. We validate that youre not crazy, but these things are very real. To me, the biggest thing that I got was hope that there was something out there that could help me.

Go here to read the rest:
Cy-Fair opens new hormone replacement therapy clinics as interest builds - Community Impact Newspaper

Recommendation and review posted by Bethany Smith

UNDATED (AP) - Tiger Woods' agent says the golfer has checked into a clinic to get help for dealing with pain medication. Mark Steinberg of Excel Sports Management wouldn't disclose the location of the in-patient treatment Woods is receiving or say how long he might be there. Steinberg says Woods' May 29 arrest in Jupiter, Florida, on a DUI charge shook him up. Woods says his arrest stemmed from a reaction to prescription drugs.

UNDATED (AP) - Phil Mickelson and his caddie have decided to part ways after 25 years of one of the most famous player-caddie relationships on the PGA Tour. Mickelson and Jim "Bones" Mackay say the decision to split was mutual and not based on an incident. Mackay is the only full-time caddie Mickelson has had in a career that has brought him 45 victories worldwide, five majors and a spot in the World Golf Hall of Fame. Mickelson says his brother, Tim Mickelson, will caddie for him the rest of the year.

UNDATED (AP) - The PGA Tour is beefing up its anti-doping policy by adding blood testing next season. Blood testing will allow the tour to detect any use of human growth hormone, which is on the list of banned substances but can't be detected through urine. The tour also is bringing its list of banned substances in line with the World Anti-Doping Association. The revised policy takes effect in October at the start of next season.

CROMWELL, Conn. (AP) - For a second year, a spot near the final hole of the Travelers Championship has been designed to accommodate people who have Lou Gehrig's disease. More than 100 ALS patients will watch some of the world's best golfers from air-conditioned viewing spots with wide travel spaces for easy navigation. They'll get rides to the tent from the parking lot and be served easy-to-eat meals. The late chief executive of the Travelers, Jay Fishman, had the motor neuron disease. He died two weeks after last year's tournament.

BOSTON (AP) - The Boston Red Sox have placed third baseman Pablo Sandoval on the disabled list with a left ear infection. The team also optioned right-hander Austin Maddox to Triple-A Pawtucket before Tuesday night's game in Kansas City. Infielder Deven Marrero and first baseman Sam Travis were recalled from Pawtucket.

See the article here:
Tiger Woods checks into clinic...Mickelson and his caddie part ways...PGA Tour to start blood testin - WMDT

Recommendation and review posted by Bethany Smith

Sinclair Broadcast Group donates pallets of food to Maryland Food Bank

BALTIMORE (WBFF) -- Employees of Sinclair Broadcast Group, which owns Fox45, delivered two pallets of food to the Maryland Food Bank on Monday as part of the companys Sinclair Cares initiative.

As part of the initiative, Sinclair and its stations help with organizations that are dedicated to improving the communities we live in.

We have a vision, our vision is connecting people with content everywhere, but we also feel we need to connect with our communities, said Sinclair Broadcast Group's Vice President Joe Koff. I think being a partner with the Maryland Food Bank, it helps us in their mission, which is together we can improve the lives of Marylanders.

Almost a quarter-ton of food was donated.

To donate to the Maryland Food Bank, click here. For more on Sinclair Cares, click here.

More here:
Sinclair Broadcast Group donates pallets of food to Maryland Food Bank - Fox Baltimore

Recommendation and review posted by Bethany Smith

Vietnam has built an LGBT-friendly medical centre so the community can have safe access to health care, sexual health information and counselling.

The centre is working towards improving access to health care to trans people especially, as it is currently illegal for people to access hormone of surgery in the country.

Although the country has put laws in place to decriminalise this, they will not take full force until 2019 at the earliest.

As well as decriminalising hormone and surgery treatment, the legislation has introduced more protections for trans people.

The centre will be providing free STI testing as well as providing this extra level of care to trans people,

It will also provide care for those diagnosed with HIV by working with other facilities who may be better equipped.

Based in Ho Chi Minh City, it has been set up by the Mens Health Centre and G -Link, who promote health care within the LGBT community.

Doctors at the clinic have said that it will greatly improve the care of LGBT people, especially trans people who may be self-prescribing hormones.

Dr Tr Anh Duy is a doctor at Bnh Dn Hospital in the same region as the new clinic explained that the clinic was crucial as some trans people can overdose or use substandard hormones, he also fears that these hormones might not be administered in a hygienic way.

One 24-year-old trans woman said that the community was often scared of seeking medical help in the country because they feared being judged.

She explained that she and friends she knew had bought hormones from Thailand but had bad side effects.

She said: I could easily buy hormones from people who had visited Thailand for sex-change surgery. Hormones and that kind of surgery are still not available in Vit Nam.

My friends, who had gone through gender transition, had problems from overdoses because they did not go to a doctor, but just listened to advice from friends.

They had vomiting and spinal pain, and at times felt dizzy. Others even had bleeding after returning to Vietnam from Thailand where they had sex-change surgery.

See the article here:
Vietnam has built an LGBT friendly medical centre PinkNews - PinkNews

Recommendation and review posted by Bethany Smith

Damn, son.

A man in India recently got the side effect of the year when doctors began treating him for hypogonadism. He was injected with hormones for nine months, and when that treatment was done, they discovered that his penis had doubled in length. Amazingly, it went from1.85 inches (flaccid, duh) to 3.7 inches (...still flaccid).

Related:Want To Know His Penis Size? Look At His Fingers!

In case you didn't know (totally possible, because most folks do not while away their free time learning all there is to know about men, their penises,and the vast array of medical conditions that can affect them and/or their sex lives at any given time), hypogonadismis a condition wherein the male body produces little to no testosterone.

As you might imagine, this can create all sorts of different problems for the men suffering from this condition. Symptoms like hot flashes, muscle weakness, fatigue, and depression are common among men suffering from male hypogonadism.

Another symptom of the disorder? The effects it can have on the size of the penis. Male hypogonadismaffects the development of the penis itself. Most sufferers have penises as adults that are no larger than a boy's pre-pubescent penis. It's not surprising thatthis one symptom,penis size,in particular(along with the accompanyingside effects of low sex drive and erectile dysfunction), is the one that most commonly sends men running to consulttheir doctors.

There's no shame in that game.

I'm a woman and I've avoided treatingdepression but have been ON MY SHIT when I notice even the slightest waning of my sex drive. We sex having folks must indeed prioritize things, after all. If a man has a small penis and thinks a doctor can find a way to get a bigger penis for him, then why the hell not?

All that said, it is interesting that the reports coming out about this treatment focus so closely on his penis size and fail to really address whether the other symptoms he sought treatment for (lack of pubic hair, fewer erections, etc) were ever alleviated. Hypogonadism can be a serious condition andpenis size is usually the least of most sufferers' worries. It speaks a lot to the importance that men are taught to place on their penis size over everything elseeven theirown health.

What good is a bigger penis if it can't stand at attention when the hour comes for it to do just that?

Related:So Wait ... Does Size REALLY Matter??

I think it's wonderful that men who are insecure about the size of their penis now potentially have an option to tackle this problem in a doctor's office and not at home with some device they bought off the late night shopping channel. That said, it kinda sucks that we have taught men that they need to prioritize their penis size over their mental, physical, and emotional health.

View post:
Hormone Treatments Helps Man's Penis DOUBLE In Size - YourTango

Recommendation and review posted by sam