Category Archives: Genetic Therapy

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COVID-19 has ravaged the economy, and it was expected to quash the IPO market, too. But the biotech sector is defying the pandemic with crossover financings and freshly minted public companies. On Friday, three firms added their names to the list of life science companies preparing to join the public markets.

Gene therapy company Generation Bio, vaccines developer Vaxcyte, and cancer diagnostics maker Burning Rock Biotech each filed IPO paperwork just ahead of the Memorial Day weekend. The filings come as an index of the largest and most liquid IPOs of the past two years reached an all-time high, according to Renaissance Capital. The IPO research firm says the indexs rise was led by Moderna (NASDAQ: MRNA), the Cambridge, MA-based biotech that this week released preliminary Phase 1 data for its experimental COVID-19 vaccine.

Investors are betting that new technologies and services are best suited for the post-pandemic world, Renaissance says.

Heres a look at the three new additions to the biotech IPO queue.

GENERATION BIO EYES NEXT-GEN GENE THERAPIES

Generation Bio aims to improve upon gene therapy with an alternative to the engineered viruses currently used to ferry these therapies into cells. Viral delivery has limitations that include safety risks and a relatively small genetic payload capacity, the Cambridge-based company says in its filing. Furthermore, if patients dont already have antibodies to the viruses, they develop them after their first dose, which means patients cant receive additional doses if the initial one doesnt work as expected or stops working over time. Gene therapies that employ viral delivery are also expensive to manufacture.

Instead of a virus as its delivery vehicle, Generation Bio uses a lipid nanoparticle. This approach permits an individualized approach to treatment as a patient can be redosed until reaching the level needed for effective treatment, the company says. The technology also has a greater payload capacity and its less expensive to manufacture at scale compared to viral gene therapies. Those differences will enable delivery of gene therapies to more types of tissue, which in turn will allow for the treatment of a broader range of diseases spanning more patients, Generation Bio says.

Generation Bios initial focus is developing gene therapies targeting the liver and the eye. The most advanced liver programs are for for phenylketonuria (PKU), an inherited metabolic disorder, and the bleeding disorder hemophilia type A, the most common form of the bleeding disorder. For the eye, Generation Bio is developing a gene therapy for an inherited form of vision loss called LCA10 and for Stargardt disease, which is a form of macular degeneration.

Generation Bio has raised more than $227 million, most recently a $110 million Series C financing in January. That funding round added crossover investors, whose involvement is viewed as an indication a company is preparing for an IPO. CEO Geoff McDonough acknowledged as much at the time, telling Xconomy he expected to take the company public in advance of beginning clinical trials.

In its filing, Generation Bio set a preliminary $125 million target for its IPO. The company has applied for a Nasdaq listing under the stock symbol GBIO. At the end of the first quarter of this year, Generation Bio reported having $104.5 million in cash. The company says it plans to use the IPO proceeds to continue R&D, including the preclinical work to support an application to start clinical testing of one of its liver disease gene therapies.

Generation Bios largest shareholders are Jason Rhodes, the companys chairman and founding CEO, and Atlas Venture. Each holds a 37 percent pre-IPO stake, according to the filing. Fidelity Investment owns 14.9 percent of the company, followed by funds advised by T. Rowe Price, which hold 8.9 percent.

VAXCYTE SETS SIGHTS ON TOPPING A PFIZER VACCINE

Vaxcyte is the new name for SutroVax, which changed its moniker this week. The Foster City, CA-based company spun out of Sutro Biopharma, and it develops vaccines using technology licensed from its former parent. The company says in its IPO filing that its cell-free protein synthesis technology enables it to design protein carriers and antigensa vaccines key componentsthat are better than what can be produced using conventional vaccine technologies.

Pneumococcal bacteria, which can cause pneumonia and meningitis, are Vaxcytes first target. The top pneumococcal vaccine, a Pfizer (NYSE: PFE) product called Prevnar, is a blockbuster seller that protects against 13 of the more than 90 pneumococcal strains. Vaxcytes preclinical vaccine candidate, VAX-24, is being developed to address 24 strains.

The IPO filing comes two months after Vaxyte closed a $110 million Series D round that added crossover investors. The company says in the filing that it has raised about $282 million cumulatively. As of March 31, Vaxcytes cash holdings totaled $154.7 million. The companys largest shareholders include Abingworth Bioventures, Longitude Capial Management, and Roche Finance, though the percentages of those stakes were not disclosed.

Vaxcyte says it plans to apply for a Nasdaq listing under the stock symbol PCVX. The vaccine developer set a preliminary $100 million goal; proceeds will be used to complete preclinical development and advance VAX-24 into human testing. The cash will also finance manufacturing, as well as continued development of other vaccine candidates.

BURNING ROCK BIOTECH BLAZES A PATH TO NASDAQ

Burning Rock Biotech is based in China, where it sells next-generation sequencing products that help physicians select cancer treatments for their patients. Now its seeking a Nasdaq listing that will give US investors a chance to grab a stake.

The company says in its filing that it offers 13 tests spanning solid tumors including cancers of the lung, prostate, and breast, as well as blood cancers. In addition helping physicians treat cancer patients, Burning Rock says its products support clinical trials conducted by large pharmaceutical companies, including AstraZeneca (NYSE: AZN), Bayer, and Johnson & Johnson (NYSE: JNJ). The companys central laboratory processes biopsy samples from hospital patients as well as from its pharmaceutical partners. The central lab business is the companys largest business segment.

Burning Rock reported $53.9 million in 2019 revenue. For the first quarter of 2020, revenue was $9.5 million. The company set a preliminary $100 million goal for its IPO, and says it plans to apply for a Nasdaq listing under the stock symbol BNR. According to the filing, Burning Rock expects to use the IPO cash for research and development of early cancer detection technologies, as well as for seeking approvals in China for additional cancer therapy selection products.

Image: iStock/peterschreiber.media

Frank Vinluan is an Xconomy editor based in Research Triangle Park. You can reach him at fvinluan@xconomy.com.

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Generation Bio Leads a Trio of Biotech Companies Aiming for the Nasdaq - Xconomy

COVID-19 impact will also be included and considered for forecast.

Global Gene Therapy Market research report provides detail information about Market Introduction, Market Summary, Global market Revenue (Revenue USD), Market Drivers, Market Restraints, Market Opportunities, Competitive Analysis, Regional and Country Level.

Gene Therapy Market Size Covers Global Industry Analysis, Size, Share, CAGR, Trends, Forecast And Business Opportunity.

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The detailed market intelligence report on the Global Gene Therapy Market applies the most effective of each primary and secondary analysis to weighs upon the competitive landscape and also the outstanding market players expected to dominate Global Gene Therapy Market place for the forecast 2019 2025.Scope Of The Report:Report evaluates the growth rate and the Market value based on Market dynamics, growth inducing factors. The complete knowledge is based on latest industry news, opportunities and trends. The report contains a comprehensive Market analysis and vendor landscape in addition to a SWOT analysis of the key vendors.Geographically, this report split global into several key Regions, revenue (Million USD) The geography (North America, Europe, Asia-Pacific, Latin America and Middle East & Africa) focusing on key countries in each region. It also covers market drivers, restraints, opportunities, challenges, and key issues in Global Gene Therapy Market. Key Benefits for Gene Therapy Market Reports Global market report covers in-depth historical and forecast analysis. Global market research report provides detail information about Market Introduction, Market Summary, Global market Revenue (Revenue USD), Market Drivers, Market Restraints, Market Opportunities, Competitive Analysis, Regional and Country Level. Global market report helps to identify opportunities in market place. Global market report covers extensive analysis of emerging trends and competitive landscape.Gene Therapy Market Segmentation:By Disease Indication Cancer Genetic disorders Cardiovascular diseases Ophthalmology Neurological conditions Others

By Type of Vectors Viral vectors Non-viral vectors

By Type of Cells Somatic cells Germline cellsBy Region North Americao U.S.o Canadao Mexico Europeo UKo Franceo Germanyo Russiao Rest of Europe Asia-Pacifico Chinao South Koreao Indiao Japano Rest of Asia-Pacific LAMEAo Latin Americao Middle Easto AfricaGene Therapy Market Key Players: Pfizer Inc. Novartis AG Bayer AG Sanofi GlaxoSmithKline plc. Amgen Inc. Boehringer Ingelheim International GmbH uniQure N.V. bluebird bio, Inc. Celgene Corporation OthersThis comprehensive report will provide: Enhance your strategic decision making Assist with your research, presentations and business plans Show which emerging market opportunities to focus on Increase your industry knowledge Keep you up-to-date with crucial market developments Allow you to develop informed growth strategies Build your technical insight Illustrate trends to exploit Strengthen your analysis of competitors Provide risk analysis, helping you avoid the pitfalls other companies could make Ultimately, help you to maximize profitability for your company.Our Market Research Solution Provides You Answer to Below Mentioned Question: Which are the driving factors responsible for the growth of market? Which are the roadblock factors of this market? What are the new opportunities, by which market will grow in coming years? What are the trends of this market? Which are main factors responsible for new product launch? How big is the global & regional market in terms of revenue, sales and production? How far will the market grow in forecast period in terms of revenue, sales and production? Which region is dominating the global market and what are the market shares of each region in the overall market in 2017? How will each segment grow over the forecast period and how much revenue will these segment account for in 2025? Which region has more opportunities?

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Our Market Research Solution Provides You Answer to Below Mentioned Question:

Which are the driving factors responsible for the growth of market?

Which are the roadblock factors of this market?

What are the new opportunities, by which market will grow in coming years?

What are the trends of this market?

Which are main factors responsible for new product launch?

How big is the global & regional market in terms of revenue, sales and production?

How far will the market grow in forecast period in terms of revenue, sales and production?

Which region is dominating the global market and what are the market shares of each region in the overall market in 2017?

How will each segment grow over the forecast period and how much revenue will these segment account for in 2025?

Which region has more opportunities?

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US Gene Therapy Market: 2020 Opportunities, Key Players, Competitive and Regional Analysis by Forecast 2025 - Jewish Life News

Surveys consistently report that people fear total blindness more than any other disability, and currently the major cause of untreatable blindness is retinal disease. The retina, a part of the brain that extends into the eye during development, initiates vision by first detecting light with the rod and cone photoreceptors. Four classes of retinal neurons then begin the analysis of visual images. Defects in the optical media that transmit and focus light rays onto the retina (lens and cornea) can usually be dealt with surgically, although such treatments are not available in some parts of the world, resulting in as many as 20 to 30 million legally blind individuals worldwide. Untreatable retinal disease potentially causes legal or total blindness in more than 11 million people in the United States alone, but progress in treatments raises the possibility of restoring vision in several types of retinal blindness (1).

Retinal neurons comprise bipolar and horizontal cells, which are second-order neurons that receive signals from the photoreceptors in the outer retina. Third-order amacrine and retinal ganglion cells are activated in the inner retina by bipolar cells. Axons from the ganglion cells form the optic nerve and carry the visual message to the rest of the brain (see the figure). The cells most susceptible to blinding retinal disease are the photoreceptors and ganglion cells. Whereas progress has been made in combating blindness caused by photoreceptor degeneration, little can be done currently to address ganglion cell loss, such as occurs in glaucoma.

The approach that has been most successful in restoring photoreceptor loss that results in complete blindness is the use of retinal prosthetic devices, with two now approved for clinical use (2). These devices electrically stimulate either bipolar or ganglion cells. They require goggles that have a camera that converts visual stimuli into electrical stimuli that activate the device, which in turn stimulates the retinal cells. Several hundred of these devices have been implanted in blind or virtually blind individuals, 70 to 80% of whom report improvement in quality of life. For those who are completely blind, the ability to experience again at least some visual function is viewed as a miracle.

There are substantial limitations to the devices, however. The best visual acuity attained so far is poor (20/500) and visual field size is limited, but many improvements, mainly technical, are being developed and tested, including the potential use of electronic low-vision devices to increase visual field size and acuity (3). Retinal prostheses are not useful for patients who are blind because of loss of ganglion cells and/or the optic nerve, but prostheses that bypass the retina and stimulate more central visual structures, including the lateral geniculate nucleus (the intermediary between retina and cortex) and visual cortex, are being developed and tested in humans (4). There remain considerable technical issues, but preliminary data indicate that such devices are feasible.

A second approach to treat photoreceptor degeneration and potential blindness, now in the clinic, is gene therapy (5). This involves injecting a viral construct into the eye that contains a normal gene to replace an abnormal one. Success so far has been limited to the treatment of Leber congenital amaurosis (LCA) type 2, a rare form of retinitis pigmentosa in which the gene whose product is required to form the correct isomer of vitamin A aldehyde, the chromophore of the visual pigments, is mutated. Little of the correct isomer is made in LCA patients, resulting in substantial loss of photoreceptor light sensitivity. This is reversed when viral constructs encoding the normal gene are injected deep into the eye between the photoreceptors and pigment epithelium.

Two factors make this approach feasible in LCA: The genetic defect is monogenic, and many of the photoreceptors in the patients remain alive, although compromised. Thus, how broadly feasible gene therapy will be for treating the enormous range of inherited retinal diseases now known to exist (300) remains to be seen. But at least a dozen other gene therapy trials on monogenic inherited eye diseases similar to LCA are under way (6). Other methods to manipulate genes are now available, including CRISPR-mediated editing of retinal genes. So far, the experiments have been mainly on isolated cells or retinas, but these powerful techniques are likely to have eventual clinical applications.

A variation on the use of gene therapy techniques is optogenetics, in which light-sensitive molecules are introduced into non-photosensitive retinal cells. This approach holds much promise for restoring vision to totally blind individuals whose photoreceptors have been lost. Using viruses to insert genes encoding light-sensitive molecules into bipolar and ganglion cells, as well as surviving photoreceptor cells that are no longer photosensitive, has been accomplished in animals and shown to restore some vision (7). Again, technical issues remain: The cells made light-sensitive require bright light stimuli, and the light-sensitive cells do not adapt. That is, whereas photoreceptors normally allow vision over as much as 10 log units of light intensity, the cells made light-sensitive respond only to a range of 2 to 3 log units. Various methods to overcome these limitations are now being developed, and at least one clinical trial is under way. Experiments to make cortical neurons sensitive to light or other stimuli that better penetrate the skullmagnetic fields or ultrasound, for exampleare also being developed and tested in animals.

Other promising approaches to restore vision are being explored. In cold-blooded vertebrates, retinal cells (in fish) and even the entire retina (in amphibians) can regenerate endogenously after damage. Regeneration of retinal cells in zebrafish is now quite well understood (8). The regenerated neurons come from the major glial cell in the retina, the Mller cell. After retinal damage, Mller cells reenter the cell cycle and divide asymmetrically to self-renew and produce a progenitor cell that proliferates to produce a pool of cells capable of differentiating into new retinal cells that repair the retina.

A number of transcription factors and other factors identified as being involved in retinal regeneration in zebrafish have been shown to stimulate some Mller cell proliferation and neuronal regeneration in mice. Regenerated bipolar and amacrine cells, as well as rod photoreceptors, have so far been identified in mouse retinas, and these cells are responsive to light stimuli (9, 10). Further, cells postsynaptic to the regenerated neurons are activated by light stimuli, indicating that the regenerated neurons have been incorporated into the retinal neural circuitry. So far, the regenerative capacity of mammalian Mller cells is limited, but directed differentiation of specific types of neurons with a mix of factors appears to be a possibility. Regrowth of ganglion cell axons after the optic nerve is disrupted is also under active investigation, and although the number of axons regrowing is low (10%), those that do regrow establish synaptic connections with their correct targets (11). Therefore, endogenous regeneration is still far from clinical testing, but substantial progress has occurred.

The retina lines the back of the eye and consists of rod and cone photoreceptors, as well as four types of neuron: second-order bipolar and horizontal cells and third-order retinal ganglion cells (RGCs) and amacrine cells. Mller glial cells fill the spaces between the neurons. The pigment epithelium, critical for photoreceptor function, underlies the retina. Photoreceptors and RGCs are most susceptible to blinding retinal disease. Progress in combating photoreceptor degeneration has been made, but there are few strategies to address RGC loss.

A long-studied area of research is transplantation of retinal cells, particularly photoreceptors, into diseased retinas. In experiments with mice, transplanted postmitotic rod photoreceptor precursor cells derived from embryonic retinas or from stem cells appeared to integrate into diseased retinas in reasonable numbers and to be functional. A surprising and unexpected complication in the interpretation of these experiments was recently discovered. Rather than integrating into diseased retinas, the donor cells appear to pass material (RNA or protein) into remaining host photoreceptor cells, rejuvenating them, and these appear to be most of the functional cells (12). The current evidence suggests that only a small proportion of the donor cells integrate, but progress in overcoming this setback is being made.

More success has been reported with stem cells induced to become pigment epithelial (PE) cells, which provide essential support for photoreceptors. A number of blinding retinal diseases relate to the degeneration of the PE cells, and replacement using such cellsin a suspension or on a scaffoldis being actively pursued. PE cells do not need to integrate synaptically with retinal cells; they simply need to contact the photoreceptor cells. This is achieved when PE cells are placed between the retina and the back of the eye. Early clinical trials suggest that the transplants are safe, but retinal detachment, a serious complication, can occur and efficacy has yet to be shown (13).

The finding that donor photoreceptor cells can help diseased host retinal cells to recover function suggests that certain substances can provide neuroprotection. Indeed, a substantial number of such neuroprotective molecules have been shown to affect retinal disease progression, especially degeneration of photoreceptor cells. No one factor has been shown to be effective generally, but two have received much attention. One, ciliary neurotrophic factor (CNTF), promotes photoreceptor survival in light-induced photoreceptor degeneration and in several other models of retinal degeneration (14). Some evidence suggests that CNTF acts primarily on Mller cells, but how it works, and on what cells, is still unclear. The other factor, rod-derived cone viability (RDCV) factor, has received less research attention, but with recent industrial support, it is now being advanced to the clinic. Current evidence indicates that RCDV factor protects cones after rod degeneration.

Two of the most common retinal diseases in developed countriesage-related macular degeneration (AMD), the leading cause of legal blindness (visual acuity of less than 20/200), and glaucoma, the leading cause of total blindnessare not monogenic diseases, and so genetic treatments for them are not obvious. Attempts to understand the etiology of these diseases are under way, but currently their underlying causes are still unclear. A difficulty presented by AMD is that no animal model is readily available, because it is a disease of the fovea, which mediates high-acuity vision. Except for primates, other mammals do not possess this small critical retinal area. Whereas large primates are not feasible for extensive cellular or molecular studies, small primates such as marmosets that have a fovea are potential models but have not been used much to date.

Other approaches for restoring vision have been suggested and have even yielded some progress. From both normal humans and those with an inherited retinal disease, skin biopsy cells can be induced to form tiny retinal eyecups called organoids (15). Containing all retinal cell types, these structures could be a source of retinal cells for studying retinal disease development and possible therapies, as well as for cell transplantation. A fovea has not been observed in any organoid so far, but this is not beyond the realm of possibility. Another suggested approach is to surgically transplant whole eyes into blind individuals. This appears feasible, but whether there is sufficient optic nerve regrowth remains an open question.

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Restoring vision to the blind - Science Magazine

Applied Genetic Reports Positive Preclinical Data for Eye Disorder Treatment

Applied Genetic Technologies Corporation (NASDAQ:AGTC) announced the results for its clinical trial related to X-Linked retinitis pigmentosa. The data provided the basis for determining the starting dose and the vector for its current Phase 1/2 clinical trial. The results demonstrated that the company's proprietary AAV vector and engineered RPGR constructs were well tolerated. It also had a positive impact on markers of disease in a canine model of XLRP.

Full length RPGR protein's DNA sequence may contain repetitive sequences which may cause instability during vector engineering and manufacturing. There are two main approaches in this regard. The first one involves removing the repetitive sequence, leading to a shortened RPGR protein while another approach is to cut down instability while producing the full length RPGR protein. Mark S. Shearman, Ph.D., Chief Scientific Officer, AGTC, said, "The results of this study identified an AAV-RPGR vector construct that has optimized safety and efficacy in a highly relevant animal model of human XLRP disease and that large-scale manufacturing that will be essential for making XLRP gene therapy available to the patients who may benefit from it." The company had earlier reported positive interim six-month data from Phase 1/2 XLRP clinical trial.

This dose ranging study used the company's proprietary rAAV2tYF capsid to deliver either a truncated RPGR DNA sequence (hRPGRstb) or an RPGR sequence encoding full-length RPGR protein optimized for stability (hRPGRco) in a canine model of XLRP. After subretinal injection, both transgenes showed similar levels of efficacy. The variables used for this purpose included fundus reflectivity, outer nuclear layer thickness, correction of opsin mislocalization and length of cone inner segments. However, in some cases, hRPGRco showed superior performance. Both the vectors were well tolerated. High dosage group experienced some cases of inflammation and retinal detachment.

Gilead Sciences, Inc. (NASDAQ:GILD) announced positive top-line data from its Phase 2b/3 trial designed to assess the efficacy and safety of filgotinib in biologic-nave or biologic-experienced adult patients with moderately to severely active ulcerative colitis. The drug candidate met all its primary endpoints at 200mg dosage. However, at 100mg dosage, it did not show any statistically significant clinical remission at Week 10.

The results were obtained from SELECTION, a Phase 2b/3, double-blind, randomized and placebo-controlled trial. It involved 1,348 patients being administered oral, once-daily, selective JAK1 inhibitor drug candidate. At week 10, the drug showed clinical remission, which is defined as an endoscopic subscore of 0 or 1, rectal bleeding subscore of 0, and 1 point decrease in stool frequency from baseline to achieve a subscore of 0 or 1. In the biologic-nave cohort, 52 percent of patients had nine or higher on a baseline Mayo Clinic Score (MCS) while the corresponding ratio for biologically-experienced cohort stood at 74 percent.

For biologic nave patients cohort, 26.1 percent of the patients showed clinical remission at Week 10 when treated with filgotinib 200 mg in comparison to 15.3 percent hitting the mark in placebo group. The corresponding data for biologic-experienced patients was at 11.5 percent and 4.2 percent, respectively. Both the cohorts showed statistically significant better performance. Patients who showed either a clinical response or remission after 10 weeks of treatment with filgotinib 100 mg or 200 mg were re-randomized to their induction dose of filgotinib or placebo in a 2:1 ratio and treated through Week 58. For the maintenance trial, both the doses met their primary endpoints.

For biologic nave patients, in the induction trial, 1.2 percent patients showed serious adverse events for 200mg dosage, while 4.7 percent suffered these hazards at 100mg dosage. The corresponding data for placebo group stood at 2.9 percent. Merdad Parsey, MD, PhD, Chief Medical Officer, Gilead Sciences said, "Patients with moderate to severe ulcerative colitis can struggle to effectively manage their disease. These top-line data suggest that filgotinib could play a role in helping more patients achieve a meaningful and sustained improvement in treatment response with an oral therapy." Ulcerative colitis is a chronic and idiopathic inflammatory disease.

The SELECTION Phase 2b/3 trial is a multi-center, randomized, double-blind, placebo-controlled trial. It comprised two induction trails and a Maintenance Trial. The company is collaborating with Galapagos NV (NASDAQ:GLPG) for developing and commercializing filgotinib in various inflammatory indications. Some of the other clinical trials involving the drug candidate are the DIVERSITY Phase 3 trial in Crohn's disease, the FINCH Phase 3 program in rheumatoid arthritis and the Phase 3 PENGUIN trials in psoriatic arthritis. It is also being tested in Phase 2 studies in uveitis and in small bowel and fistulizing Crohn's disease.

Filgotinib is a lead drug candidate for Gilead and is an investigational agent. The drug candidate is currently under review by the FDA as treatment for rheumatoid arthritis. It is also being reviewed in other geographies including European Union. The drug is expected to face competition from the likes of Rinvoq from AbbVie (NYSE:ABBV) but is still likely to generate impressive revenue stream. Since the drug failed to meet its primary endpoint at 100mg dosage, the prospects for 200mg dosage seem better.

Apellis Pharmaceuticals Inc. (NASDAQ:APLS) announced that it is in the process of submitting a New Drug Application (NDA) for pegcetacoplan during the second half of 2020. The application will be accompanied by the data from the Phase 3 PEGASUS trial comparing pegcetacoplan to eculizumab in patients suffering from Paroxysmal Nocturnal Hemoglobinuria. The company is also in the process of carrying out discussions with authorities in the European Union.

The PEGASUS study is a randomized, multi-center, active comparator and open label controlled Phase 3 study. The trial involved 80 adult patients suffering from Paroxysmal Nocturnal Hemoglobinuria. The primary endpoint of the trial was to assess the efficacy and safety of pegcetacoplan compared to eculizumab. Participants were required to be on eculizumab (stable for at least three months) with a hemoglobin level of <10.5 g/dL at the screening visit. All participants completing the randomized controlled period proceeded to the open-label pegcetacoplan treatment period. They were administered pegcetacoplan in the randomized and controlled period.

The study met its primary endpoint as the data showed superior performance by pegcetacoplan to eculizumab. The drug candidate led to a statistically significant improvement in hemoglobin levels at 16 weeks. During the run-in period of four weeks, the participants were given 1080 mg of pegcetacoplan twice a week along with their current dose of eculizumab. For 16-week randomized, controlled period, the participants were randomized and they were either given their current dose of eculizumab or 1,080 mg of pegcetacoplan twice weekly. The data also showed that pegcetacoplan had the drug safety profile comparable to eculizumab.

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Applied Genetic Reports Positive Data, And Other News: The Good, Bad And Ugly Of Biopharma - Seeking Alpha

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Cutting-edge research on rewriting the genes responsible for Tay-Sachs disease, sickle-cell anemia, and other disorders will be presented at this weeks online annual meeting of the American Society for Gene and Cell Therapy. Originally planned as a Boston gathering, the scientific groups get-together became a virtual one because of the Covid-19 pandemic.

From Tuesday through Friday, academic researchers will be presenting their latest data online, along with updates from firms like Bluebird Bio (ticker: BLUE), Voyager Therapeutics (VYGR), Fate Therapeutics (FATE), Beam Therapeutics (BEAM), Axovant Gene Therapies (AXGT), and many others. Patients and their families have found their way to clinical trials through the societys website.

Bluebird plans presentations on its cell therapy against the blood cancer known as multiple myeloma. Using a technology known as CAR-T, the company creates supercharged versions of a patients immune cells that have halted disease progression in some of the 18 patients enrolled in a continuing Phase 1 trial.

Featured on Friday will be reports on the first babies treated with gene therapy for the debilitating neurodegenerative disorder Tay-Sachs. The treatment is being developed by the London-based Axovant under license from the University of Massachusetts Medical School.

Voyager will discuss its preclinical mouse studies on treating neurological disorders like amyotrophic lateral sclerosis and Huntingtons disease by using techniques that block the rogue signals generated by defective genes.

Fate Therapuetics is scheduled to show a new off-the-shelf CAR-T technology that it hopes will allow the immune system to target a broad range of solid tumors as well as multiple myeloma. The approach is licensed from Harvard Universitys Dana-Farber Cancer Institute.

Beam, meanwhile, will detail success it has shown in preclinical editing of the genetic defect that causes sickle-cell anemia. The company is developing a sharper-edged way of rewriting faulty genes than the widely used Crispr technology that Beam licensed from researchers at the Broad institute of Harvard and MIT. Beam founder and Crispr pioneer Feng Zhang will give a featured lecture as part of the online meeting on Thursday.

Write to Bill Alpert at william.alpert@barrons.com

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Gene-Therapy Treatments for Tay-Sachs, Sickle Cell to Be Featured in Online Gathering - Barron's

Trying to hack fitness is a multi-million-dollar industry; weve all seen at least one ad featuring a purported miracle product that claims it can make people lose weight and look greatwithout even trying. From low-effort exercise machines to strange-ingredient diets to fat-burning belts and bands, theres no shortage of attempts to make it easy to be fit.

A gene therapy trial performed on mice may foreshadow yet another way to hack fitness. In a study done by a team at Washington University in St. Louis medical school, mice quickly built muscle mass and reduced obesity after receiving the therapy, even while eating a diet high in fat and not exercising. The results were published last week in a paper in Science Advances.

Sound appealing? Heres how it worked.

The gene targeted was FST, which is responsible for making a protein called follistatin. In humans and most other mammals, follistatin helps grow muscle and control metabolism by blocking a protein called myostatin, which acts to restrain muscle growth and ensure muscles dont get too big.

The researchers injected eight-week-old mice with a virus carrying a healthy FST gene (gene therapy involves adding healthy copies of a gene to cells, usually using a virus as a deliveryman).

Over a period of 18 weeks, or about 4 months, the team observed that the muscle mass of the treated mice more than doubled, as did their strength level. They also experienced reduced damage related to osteoarthritis, less inflammation in their joints, and had healthier hearts and blood vessels than mice that didnt receive the gene therapyeven though all the mice ate the same high-fat diet and did the same amount of exercise.

Going into the study, the researchers worried the muscle growth catalyzed by the gene therapy could harm the heart, mainly through thickening of the hearts walls. Surprisingly, though, heart function and cardiovascular health of the treated mice actually improved. In subsequent studies, the team will continue to monitor the treatments effect on the heart, as complications could emerge over time.

Talk about a fitness hack; imagine being able to build muscle and maintain a healthy metabolism while lounging on the couch eating burgers and fries. There have been similar studies to replicate the effects of exercise by commandeering the genetic instructions that control the way cells interact with proteins; though various exercise pills have successfully mimicked the effects of regular cardiovascular activity in mice, scientists still dont fully understand how, at a molecular level, exercise has the effects it does on the human body.

This may change in the next couple years, though; a National Institutes of Health consortium called the Molecular Transducers of Physical Activity is in the midst of an in-depth study on the molecular effects of exercise on tissues and organs in 3,000 people.

If the muscle-building gene therapy eventually reaches a point where it can be used in humans, though, the research team isnt viewing it as a quick-fix health hack. Rather, it would be used to help get people with conditions like muscular dystrophy or severe obesity to a baseline from which they could adopt tried-and-true muscle-building practices like weight lifting or physical therapy.

In cases of severe obesity or muscle loss, it is extremely difficultif not impossibleto lose weight or improve muscle strength through normal exercise and diet, said Farshid Guilak, orthopedic surgery professor and director of research at Shriners Hospitals for Children in St. Louis. The goal of this study was to show the importance of muscle strength in overriding many of the harmful effects of obesity on the joint.

If every condition, process, and trait in our bodies is tightly linked to our genes, its conceivable that almost any aspect of our health could be manipulated using gene therapy and related tools. Maybe one day there will indeed be a pill we can take or a shot we can get to give us svelte, muscular bodies without any of the effort.

The fact that this would ruin the pleasure and satisfaction of a good workout is another conversationand one not everybody would be interested in having. But even if genetic or chemical exercise-replacement tools become safe to use in humans in the foreseeable future, theyll likely be limited, at least at first, to those who need them due to debilitating health conditions.

That saidfor the time being, keep hitting the treadmill, the weight room, or your other off-the-couch, effort-intensive workout of choice.

Image Credit: Aberro Creative from Pixabay

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Forget ExerciseThese Mice Got Ripped With Gene Therapy - Singularity Hub