Category Archives: Genetic Therapy

Easy access to (temporarily) free counseling.Consumer telehealth and mail-order prescription services Hims and Hers are dipping into the mental health wellness space. The brands kicked off new anonymized group therapy and guided meditationofferings yesterday, with the therapy sessions led by a licensed mental health practitioner.

The group sessions are currently being offered free of charge in light of COVID-19 stress. The brands have also opened up wait-lists for upcoming mental health-focused services, such as individual online therapy.

Digital therapeutics want a role in COVID-19 response.The Digital Therapeutics Alliances is rallying industry players for a new effort targeting publicly funded health programs.

Dubbed the Urgent Access campaign, the new effort specifically calls for the U.S. Health & Human Services, and potentially Congress, to use its emergency COVID-19 powers to provide digital therapeutics access through Medicare and Medicaid. In Europe, the organization is pushing for the European Ministries of Health to authorize the use of these products within each country's healthcare systems, or to expand use if pathways already exist in a particular country.

The impacts of COVID-19 on patients and clinicians continue to grow as quarantines are extended, as vulnerable populations are increasingly at risk of viral exposure, and healthcare resources are further restricted to those requiring hospital care, Dr. Megan Coder, executive director of the Digital Therapeutics Alliance, said in a statement. While digital therapeutics do not treat COVID-19, they directly impact critical consequences of this pandemic. Yet today, patients covered by publicly funded programs rarely have access to this category of medicine.

FDA makes increases digitaleye-assessment availability.Last night the FDA issued new guidance extending the use of remote ophthalmic assessment and monitoring devices during the COVID-19 pandemic. Applicable to devices such as visual acuity charts, visual field devices, general-use ophthalmic cameras and tonometers, the agency guidance will be allowing certain modifications to these devices, so long as they "do not create undue risk in light of the public health emergency."

The new policy outlines a sampling of the types of modifications the FDA has in mind: indication changes from healthcare to home use, the addition of portable or handheld device features, and hardware or software changes "to include virtual reality or mobile technology for remote assessment or monitoring capability."

"In developing this policy, FDAs intent is to foster the continued availability of safe and effective medical devices while being flexible regarding modifications made to devices for remote assessment and monitoring of ophthalmic parameters in response to the COVID-19 public health emergency," the agency wrote.

Could genetics impact COVID-19 severity?Age, concurrent illness and health histories are all clear influences on a COVID-19 infection's severity, but now 23andMe is interested in exploring whether or not an individual's genes may be playing a role as well. The consumer genomics-testing company announced Monday that it is hoping to enroll hundreds and thousands of its U.S. customers in a longitudinal genome-wide association study, with the end goal of identifying specific genetic variants that might be associated with disease severity.

Ultimately, we want to publish our research findings in order to help provide more insight on COVID-19 for the scientific community," Joyce Tung, VP of research at 23andMe, said in a blog post from the company.

Read the rest here:
Hims kicks off mental health offerings with free group therapy, campaign calls for public access to digital therapeutics during COVID-19 emergency and...

Many charts of shares on the Frankfurt Stock Exchange are currently showing new buy signals, including the price of Evotec shares. On April 6th, the share price of the TecDAX-listed biotech company from Hamburg at 21.74/21.80 was already able to overcome the first important hurdles in a two-part technical obstacle zone.

The share price rose to 22.10 at the beginning of the week and left XETRA trading at 22.07 (+5.15%). Thus the breakout above part 2 of the hurdle remained marginal at 22.02. However, this buy-signal became more stable this morning! Current indications for the Evotec share are trading at 22.53/22.59.

From a chartist point of view, a stable rise above the core hurdles at 21.80/22.02 would be an enormously important buy signal for the biotech share. A V shaped trend reversal would then be the predominant chart technical formation after the corona crash from 26.77 to 17.17. At 23.59/23.79, the core zone of the next strong resistance levels would be present in such a bullish scenario. However, the first hurdles would already be expected at 23.12/23.27 and above.

Meanwhile, the technical hurdles for Evotecs share price that have been overcome are becoming support levels. Between 21.74/21.80 and the gap that was torn open yesterday at 21.18/21.37, the first stronger support for the TecDAX-listed biotech share should be found. Further support will then be only slightly below this level.

Discover the worlds most interesting financial news with the mobile app Born2Invest. The app provides its readers access to the latest news in global business, stock market, finance news, and also trending topics like bitcoin, cryptocurrency, and biotech.

Evotec is expanding its activities in the field of gene therapy. Evotec Gene Therapy, a new team of gene therapy experts from Evotec, will be based at a site in Orth an der Donau, Austria, the Hamburg-based biotech company announced on Monday. Evotecs team of scientists has a lot of knowledge in vectorology and virology, as well as in hematology, muscle diseases, and hemophilia.

At the same time, the North Germans also announced the conclusion of a multi-year collaboration in the field of gene therapy between Evotec Gene Therapy and the Japanese Takeda group.

As part of the alliance, Evotec will support several programs focused on the development of compounds in Takedas four core therapeutic areas: oncology, rare diseases, neurology, and gastroenterology, reported the TecDAX-listed group. However, Evotec has not presented financial details of the collaboration.

Steven Hitchcock, global head of research at Takeda, said in a statement We are excited to be broadening and expanding our discovery efforts with the Evotec team. Gene therapy is a growing therapeutic approach in our portfolio and this alliance with Evotec will help us further accelerate our delivery of transformative therapies for patients, particularly those with rare diseases.

The Hamburg-based company is particularly counting on a new trend in the development of treatment methods: In recent years, more and more precision medical approaches based on cell and gene therapies have been developed, and this trend is expected to continue in the future. Gene therapy is a promising approach for the development of genetic agents, particularly for hereditary and rare diseases, said Evotec CEO Werner Lanthaler.

__

(Featured image by qimono via Pixabay)

DISCLAIMER: This article was written by a third party contributor and does not reflect the opinion of Born2Invest, its management, staff or its associates. Please review our disclaimer for more information.

This article may include forward-looking statements. These forward-looking statements generally are identified by the words believe, project, estimate, become, plan, will, and similar expressions. These forward-looking statements involve known and unknown risks as well as uncertainties, including those discussed in the following cautionary statements and elsewhere in this article and on this site. Although the Company may believe that its expectations are based on reasonable assumptions, the actual results that the Company may achieve may differ materially from any forward-looking statements, which reflect the opinions of the management of the Company only as of the date hereof. Additionally, please make sure to read these important disclosures.

First published in 4investors, a third-party contributor translated and adapted the article from the original. In case of discrepancy, the original will prevail.

Although we made reasonable efforts to provide accurate translations, some parts may be incorrect. Born2Invest assumes no responsibility for errors, omissions or ambiguities in the translations provided on this website. Any person or entity relying on translated content does so at their own risk. Born2Invest is not responsible for losses caused by such reliance on the accuracy or reliability of translated information. If you wish to report an error or inaccuracy in the translation, we encourage you to contact us.

Read more from the original source:
Biotech company Evotec is showing strong performance on the stock market - Born2Invest

According to results of a study comparing CD38-positive tumor cells ofpatients with newly diagnosed multiple myeloma, a high-risk status determinedby either a validated gene-expression profiling assay or the presence of specificchromosomal abnormalities was independently predictive of poor outcome. Thesefindings were published in Leukemia.1

Because survival rates of patients with newly diagnosed multiplemyeloma can vary widely, it is important to identify those with high-riskdisease characterized by a more aggressive clinical course who may require a newtherapeutic approach.

Chromosomal aberrations known to be associated with more aggressivedisease include translocations t(4;16), t(14;16), and t(14;20), as well as thepresence of 4 or more copies of the 1q chromosome, as well as deletion of bothcopies of chromosome 17p. In particular, previous studies of patients with multiplemyeloma have shown low survival rates in those with disease characterized bythe presence of 2 or more of these genetic features (ie, a double hit). Inaddition, other methods that have been validated for the prediction of outcomein patients with multiple myeloma include gene-expression assays, such as the SKY92MMproflier (SKY92), and the International Staging System(ISS), as well as the combination of these 2 methods.2

In this study, the presence or absence of chromosomal aberrations, aswell as SKY92 expression profiles of 92 genes, were evaluated in CD38-postivetumor cells from a cohort of 329 patients with newly diagnosed multiple myelomareceiving intensive therapy in the NCRI Myeloma XI trial.

A comparison ofthe 81 patients in this cohort with disease characterized by a high-riskgene-expression profile showed median overall survival (OS) was significantlyshorter (36.7 months) compared with the subgroup with disease that lacked this gene-expressionsignature (median OS not reached; hazard ratio HR, 3.9; 95% CI, 2.7-5.7; P =2.5 x 10-13).

On multivariable analyses, a high-risk SKY92 profile (HR, 2.7; 95% CI, 1.84.2; P =4.4 106), high-risk chromosomal translocations (HR 1.8; 95% CI, 1.22.9; P =.007), and deletion of the 17p chromosome (HR; 2.5, 95% CI, 1.54.1; P =.0007) were all found to be independently associated with worse OS compared with patients without these markers of high-risk disease.

Furthermore,high-risk SKY92 and double-hit were independently prognostic by multivariableanalysis, with HRs for OS of 2.9 (95% CI, 1.94.2;P=2.6 107)and 2.3

(95%CI, 1.53.6; P=.0002) compared with subgroups withoutthese markers.

Percentagesof the 3 subgroups defined by a high-risk SKY92 profile and/or high-riskchromosomal marker(s), and the hazard ratios for OS for these subgroupscompared with the subgroup without these disease markers, were as follows:

Ofnote, these findings were validated in a separate cohort of 116 patientsenrolled in the transplant arm of MRC Myeloma IX study of newly diagnosed patientswith multiple myeloma.

The study authors stated that these results highlight the molecular diversity of [multiple myeloma] and demonstrate that single time point combined GEP and chromosomal profiling at diagnosis can predict clinical outcome with significant precision

References

Read the original here:
Gene-Expression Profiling Assay Predicts Survival in Newly Diagnosed Multiple Myeloma - Cancer Therapy Advisor

While the world is working to slow the COVID-19 pandemic by social isolation and quarantine, numerous companies globally are working to develop a vaccine against the virus. The most likely timelinesat bestfor a viable vaccine against SARS-CoV-2, the coronavirus causing COVID-19, are 12 to 18 months. And thats if everything goes right.

As a recent article in Bloomberg Businessweek notes, For that to happen in the next year or so, an almost equally implausible set of circumstances has to occur: flawless scientific execution, breakneck trials and a military-style manufacturing mobilization unlike any the pharmaceutical industry has put in place before.

Typically, it takes 10 to 15 years of deliberate and careful work to develop a commercial vaccineand as both the common cold and HIV have demonstrated, sometimes its not possible even then. The infrastructure for the seasonal flu vaccines that come out every year has been in place for decades.

One of the companies that is gaining a lot of attention is Cambridge, Massachusetts-based Moderna. They have a head-start and are using a novel technology to develop their COVID-19 vaccine, mRNA-1273, which is already in clinical trials. mRNA-1273 is a mRNA vaccine that encodes for a prefusion stabilized form of the Spike (S) protein. Whats different about the vaccine is that it is almost like a form of gene therapyit codes for the genetic sequence for the spike protein, and when injected into the body, causes the patients own cells to produce the protein (not the virus), which triggers an immune reaction that will prep itself to battle the virus.

The technology is interesting, but unproven. There are no approved RNA therapies. And Moderna has never brought a product to the market before.

One thing thats of note is how early a start Moderna got on the vaccine. On January 11, 2020, Chinese researchers posted the genetic sequence of the coronavirus, well before most of the world was paying any attention. Moderna scientists had been working on a novel vaccine for a different coronavirus disease and jumped on this sequence and began working on a vaccine against it.

Bloomberg writes, By late February, when President Trump was still downplaying the risk of coronavirus, Modernas scientists had already delivered the first batch of candidate vaccines to researchers at the U.S. National institutes of Health. When the coronavirus was starting to explode in the U.S. in mid-March, the first healthy patient received a dose in a small, government-sponsored safety trial.

Moderna may be the leader, but they are not the only company employing this technology. Pfizer and a few others are as well. Pfizer is working with a German company, BioNTech, hoping to get their experimental RNA vaccine into human trials sometime this month.

Johnson & Johnson is using technology it employed for its experimental Zika and Ebola vaccine. Paris-based Sanofi is modifying technology it uses for its flu shots. There are more than two dozen companies working on coronavirus vaccines that are already in early-stage testing.

There is no precedent for the speed at which we are moving, said Clement Lewin, an associate vice president at Sanofi. I cant think of a parallel, in his thirty years of vaccine work.

The RNA vaccine technology, despite the apparent speed with which Moderna, Pfizer and BioNTech are moving, has some risks. Not as much is known about its efficacy. Early-stage data suggests it should be safe, but there are concerns about unwanted immune responses. And all vaccines run a risk of what is called disease enhancement, where the immune system responds in an unusual way, which can cause the illness to be worse.

It's possible that any vaccines just wont be effective, or the side effects will be such that they wont be considered safe for use.

Scale-up is also a major roadblock. As of April 7, there are 1,362,936 confirmed cases of COVID-19 around the globe. But there are another 8 billion possible people who would possibly benefit from a vaccine.

Under a scenario where Modernas vaccine turns out to be effective and safe, no one has experience in commercial production of RNA vaccines, let alone in the type of scale necessary. The company indicates it has the manufacturing capability to produce millions of doses of the vaccine per month at a factory that was already scaling up for a different vaccine. They are apparently also in discussions with possible partners. But millions is a very long way from producing vaccines for billions of peopleor even a few hundred million people.

If a vaccine were to become available in the next year, an optimistic timeline, the first candidates for the drug would be first-line respondersdoctors, nurses, paramedics, as well as infants, toddlers and pregnant women. Thats a figure that exceeds 26 million people in the U.S. alone.

The next round would be essential personnel and children with preexisting conditions, higher-risk patients and people over the age of 65. Once you add all those, just in the U.S., youre talking more than 100 million who would be considered high-priority for a vaccine.

Mark Feinberg, a former Merck & Co. executive in vaccines who now runs the International AIDS Vaccine Initiative, told Bloomberg that the real question wasnt just efficacy, but how quickly can you ramp up manufacturing to meet global need.

There arent a lot of drugs in the industry that are filled at these scales, period, said Stephen Hoge, president of Moderna. Even large pharma companies dont usually operate on this kind of scale. No one entity or one company will be able to do it by themselves.

With any luck, social distancing and quarantine will slow the spread of the virus. Perhaps the coming summer months will also decrease its spread, although whether there is a seasonality component to the disease is still unknown. This would give biopharma companies a little more room to develop and distribute vaccines or to determine which drugs are best-suited to preventing or treating the disease.

Meanwhile, biopharma companies worldwide are working desperately to develop solutions.

Go here to see the original:
The Hopes and Challenges of a COVID-19 Vaccine - BioSpace

Apr 11, 2020 09:54 PM EDT

Israeli scientists are on the verge of developing the first vaccine against COVID-19, while Israeli's COVID-19 Pluristem's placenta-based cell-therapy showed a 100% survival rate.

Six critically ill coronavirus patientswith high risk for mortality were given Pluristem's placenta-based cell therapy product and survived, according to the data showed by Puristem.

In a report fromJerusalem Post, the patients were treated at three different Israel medical centers for one week under Israel's compassionate use program. The patients were suffering from acute respiratory failure and inflammatory complications due to COVID-19. Four of the patients had a failure of organ systems, including cardiovascular and kidney failure.

According to Pluristem, all patients survived. Four of the patients showed improvement in respiratory function, and three are about to be weaned from ventilators. Two patients with preexisting medical conditions showed recovery in a respiratory problem.

The Pluristem's PLX cellsare "allogeneic mesenchymal-like cells that have immunomodulatory properties". It prompts the immune system's T cells and M2 macrophages, causing the reversal of dangerous over activation of the immune system. It reduces the fatal symptoms of pneumonia and pneumonitis.

Notable therapeutic effects of pulmonary hypertension, lung fibrosis, acute kidney injury, and gastrointestinal injury were also noted in previous pre-clinical animal studies.

The company is in the process of applying for the initiation of a multinational clinical trial for the treatment of complications related to COVID-19. With this development, the company will no longer report on its compassionate use trials but the status and progress of the expected clinical trial.

Pluristem Therapeutics Inc. is a clinical-stage biotherapy company. It maximizes "placental cells and unique, proprietary, three-dimensional (3D) technology platform to develop cell therapies" for illnesses such as inflammation, ischemia, muscle injuries, hematological disorders, and exposure to radiation."

The Galilee Research Institute (MIGAL) is at the edge of developing thefirst vaccines against the novel coronavirus. The vaccine might be ready in three weeks, and available in 90 days should everything go as planned, Science and Technology Minister Ofir Akunis announced last week.

MIGAL has been developing a vaccine against infectious bronchitis virus (IBV) to treat a bronchial disease affecting the poultry industry. The vaccine has been proven effective has been in clinical trials carried out by Veterinary Institute.

The IBV vaccinewas developed after four years of research. It was discovered to have high genetic similarity to the human coronavirus. The company has since genetically modified the vaccine to treat COVID-19. The pre-clinical trials revealed that taking the oral vaccine prompts the release of a high level of specific anti-IBV antibodies, Dr. Chen Katz, MIGAL's Biotechnology group leader said.

For now, Katz said they are adjusting the new sequence and are hopeful that they will have the vaccine available in a few weeks.

MIGAL will go through a regulatory process that includes clinical trial and large scale production. MIGAL CEO Davis Zigdon said that they are doing their best to accelerate development, and the vaccine could "achieve safety approval in 90 days".

The vaccine will be available in oral form.

2018 NatureWorldNews.com All rights reserved. Do not reproduce without permission.

Go here to see the original:
COVID-19: Therapy, Vaccine Show Promising Breakthrough in Israel - Nature World News

WALTHAM, Mass., March 31, 2020 (GLOBE NEWSWIRE) -- Affinia Therapeutics, an innovative gene therapy company with a platform for rationally designed adeno-associated virus (AAV) vectors and gene therapies and a mission to develop transformative medicines for devastating diseases, today announced it has closed an oversubscribed $60 million Series A financing. Seed venture investors F-Prime Capital and New Enterprise Associates (NEA) co-led the round alongside new investor Atlas Venture, with participation from seed investors Alexandria Venture Investments, Lonza and Partners Innovation Fund.

The proceeds will be used to advance the companys platform and develop transformative gene therapies for people affected by muscle and central nervous system (CNS) diseases with significant unmet need.

The company is led by recently appointed Chief Executive Officer Rick Modi, who has a proven track record of building value at companies including AveXis, InterMune, MedImmune and Centocor. Joining the board of directors as part of the financing are Dave Grayzel, M.D., Partner, Atlas Venture; Ed Mathers, General Partner, NEA; and Robert Weisskoff, Ph.D., Partner, F-Prime Capital. Industry veteran and gene therapy leader Sean Nolan will chair the board.

At Affinia Therapeutics, were setting a new standard in gene therapies by leveraging our proprietary platform to methodically engineer novel AAV vectors and gene therapies that have remarkable targeting properties, said Modi, CEO of Affinia. We are pleased to partner with such a distinguished syndicate to advance our platform and investigational product candidates toward the clinic for patients in need.

The companys technology was licensed from Lonza and Massachusetts Eye and Ear. It was developed at the Grousbeck Gene Therapy Center and further advanced under a sponsored research agreement with Lonza led by Luk Vandenberghe, Ph.D., Associate Professor at Mass. Eye and Ear and Harvard Medical School and a co-inventor of AAV9.

In addition to Vandenberghe, joining Affinia Therapeutics as scientific co-founders are Botond Roska, M.D., Ph.D., Director, Institute of Molecular and Clinical Ophthalmology Basel; Aaron Tward, M.D., Ph.D., Assistant Professor, University of California, San Francisco; and Eric Zinn, Ph.D. student, Mass. Eye and Ear and Harvard University. Together, these scientists have authored more than 200 papers and filed more than 20 patents in the field of gene therapy.

Affinia Therapeutics is bringing together complementary expertise allowing us to realize a rational design future for AAV vectors, promoters and other components of gene therapies. By leveraging synthetic and systems biology combined with high-throughput screening and tissue and single-cell resolution, we are aspiring to achieve much-needed improved pharmacological control of this novel modality in medicine, said Vandenberghe, Director, Grousbeck Gene Therapy Center at Mass. Eye and Ear.

The Series A financing comes after Vandenberghe and his team successfully developed AAVSmartLibraries comprising thousands of functional novel AAV vectors. Each vector is uniquely identified, and the libraries can be screened across species for parameters of high interest, including tissue tropism, manufacturing yield and pre-existing immunity. Observations arising from each library screen provide insights into the vectors structure-function, enabling the rational design of novel vectors and gene therapies with remarkably improved properties. Affinia Therapeutics has potentially the worlds largest library of patented functional AAV vectors.

Affinia Therapeutics methodical process for designing and evaluating vectors is a differentiated approach to gene therapy, and the highly experienced leadership team will help carry these discoveries to the development, manufacturing and commercialization of transformative medicines, said Mathers, General Partner at NEA. We are pleased to accelerate the impact of this exciting field.

About Affinia Therapeutics

At Affinia Therapeutics, our purpose is to develop gene therapies that can have a transformative impact on people affected by devastating genetic diseases. Our proprietary platform enables us to methodically engineer novel AAV vectors and gene therapies that have remarkable tissue targeting and other properties. We are building world-class capabilities to discover, develop, manufacture and commercialize gene therapy products with an initial focus on muscle and central nervous system (CNS) diseases with significant unmet need.www.affiniatx.com.

Story continues

Media contact - media@affiniatx.com

Read the rest here:
Affinia Therapeutics Raises $60M in Series A Financing to Advance Rational Design AAV Platform and Transformative Gene Therapies - Yahoo Finance