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Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.
Genome engineering and gene therapies that manipulate DNA sequences in cells have driven a biotechnological revolution over the past decade. 1 Adeno-associated virus (AAV) vectors are the leading platform for in vivo gene delivery for the treatment of various human diseases. Nature 578 , 229-236 (2020). References Doudna JA.
HIV drug resistance is caused by changes in the genetic structure of HIV that affects the ability of drugs to block the replication of the virus. Over the past decade, the world has witnessed unprecedented scale-up of antiretroviral therapy (ART), which has saved the lives of tens of millions of people. Department information note.
The FDA’s January 2020 guidance, Chemistry, Manufacturing and Control (CMC) [1] Information for Human Gene Therapy Investigational New Drug Applications (INDs), outlines the analytical methods that define the quality, safety and efficacy of gene therapy therapeutics.
There are a few approved therapies for DMD including four exon-skipping drugs and one AAV-microdystrophin drug, which uses a shortened version of dystrophin. Because mRNA is not a virus, it doesn’t have to meet biosafety requirements for manufacturing and administration that an AAV approach would need. 2020 Jul 3;10(1):10967.
Strong R&D commitment to developing therapeutics against SARS-CoV-2 virus. billion EUR in 2020 (18.9% Boehringer Ingelheim stepped up its investments in R&D significantly in 2020 in pursuit of innovative medicines and therapies for diseases for which no satisfactory treatments are available. of net sales).
5, 2020 — A pair of new gene therapies promise a potentially lasting cure for sickle cell disease by subtly altering the genetic information in patients’ bone marrow cells, researchers report. Both of the new gene therapy studies were published online Dec. SATURDAY, Dec. 5 in the New England Journal of Medicine.
The year 2020 will certainly go down in history as the year of COVID-19. Gilead Sciences Makes Multiple Bets : When Gilead Sciences wasn’t working on remdesivir in COVID-19, the company seemed to be constantly flexing its M&A muscle over the course of 2020. Nestlé Health Science has a history of advancing nutritional therapies.
If approved by the FDA, this maternal immunization has the potential to be the first vaccine candidate to help protect infants in their vulnerable first months of life from disease caused by this highly-contagious virus. Burden of RSV RSV is a contagious virus and a common cause of respiratory illness. Pfizer Inc.
Monthly News Roundup – December 2020. The messenger RNA (mRNA) vaccines encode a form of the spike (S) protein of SARS-CoV-2 virus. The vaccine teaches the cells to make a piece of the spike protein, which triggers an immune response to help prevent illness if later exposed to the virus.
Posted: December 2020.
27, 2020 06:30 UTC. The agreement covers the gene therapy program targeting OTOF (OTOF-GT), the gene encoding the otoferlin protein. We are delighted and proud to contribute to the progress of Sensorion’s innovative gene therapy programs targeting OTOF. Mutations in OTOF lead to severe or profound congenital hearing loss.
Parkinson’s disease is most common neurodegenerative movement disorder / It impacts more than 10 million people worldwide/ No function-restoring therapy is currently available / Bayer is pursuing a two-pronged approach to deliver transformative therapies with one cell and one gene therapy candidate in clinical trials.
30, 2020 12:30 UTC. Innovative, novel approach to treat and protect against SARS-CoV-2 using thermoresponsive virus-targeting nanoparticles as an inhalation medicine. ImmunoPrecise is a global technology platform company with end-to-end solutions empowering companies to discover and develop therapies against any disease.
27, 2020 /PRNewswire/ — Aridis Pharmaceuticals, Inc. Dr. Hasan Jafri , Chief Medical Officer of Aridis Pharmaceuticals, will be a speaker on a panel entitled “Direct Antivirals and Other Agents Against SARS-CoV2 Virus.” SAN JOSE, Calif. , AR-711 (COVID-19). AR-201 (RSV infection). AR-201 (RSV infection).
Most targeted cancer therapies used today operate by inhibiting targets along well-known oncogenic signalling cascades. The reactivation of oncogenic signalling upstream or downstream of the driving oncogene is a well-studied source of resistance to targeted cancer therapies.
2, 2020 /PRNewswire/ — Corlieve Therapeutics today announced the closing of its seed financing led by Kurma Partners and IDinvest Partners, together with Pureos Bioventures. Maryland , US), a leading gene therapy company. PARIS , Nov. Corlieve is focused on developing novel therapeutics for severe neurological disorders.
27, 2020 10:45 UTC. –( BUSINESS WIRE )– Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced financial results for the third quarter of 2020.
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for the third quarter of 2020. .
for the third quarter of 2020. KENILWORTH, N.J.–(
21 October 2020 — ViiV Healthcare, the global specialist HIV company majority owned by GSK, with Pfizer Inc. In the midst of the global pandemic, the analysis found no antiretroviral therapy interruptions across the entirety of the ongoing clinical development programme for long-acting cabotegravir and rilpivirine.
November 21, 2020 – Regeneron Pharmaceuticals, Inc. NASDAQ: REGN) today announced that the antibody cocktail casirivimab and imdevimab administered together (formerly known as REGN-COV2 or REGEN-COV2), a therapy currently being investigated for use in COVID-19 , has received Emergency Use Authorization (EUA) from the U.S.
Nirsevimab demonstrate d protect ion against respiratory syncytial virus disease in healthy infants in Phase 3 trial. Respiratory Syncytial Virus (RSV) is the leading cause of hospitalization in all infants. 1 – 5. RSV is the most common cause of LRTI and the leading cause of hospitalizations in all infants.
30, 2020 /PRNewswire/ — Eli Lilly and Company (NYSE: LLY) and Incyte (NASDAQ: INCY) announced today new data for baricitinib (marketed as OLUMIANT ® ) will be presented at the annual Fall Clinical Dermatology meeting taking place virtually October 29-November 1, 2020. INDIANAPOLIS , Oct.
He has also emerged in recent years as a skilled and relatable science communicator, renowned for his efforts at Broad and beyond during the COVID-19 pandemic to unpack the fast-moving research on the new virus in an accessible way and give practical advice about masking, vaccines, and other public health measures.
10, 2020 04:32 UTC. NASDAQ: RCKT) (“Rocket”), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces the pricing of an upsized underwritten public offering of 4,642,858 shares of its common stock at a public offering price of $56.00
This donation of the vaccine is another tool in our toolbox of measures to help make the Olympic and Paralympic Games Tokyo 2020 safe and secure for all participants and to show solidarity with our gracious Japanese hosts,” said IOC President Thomas Bach. “We This press release features multimedia. View the full release here: [link].
The announcement, following a $98 million IPO in October 2020, comes as the company advances several programs through its pipeline. In addition to its lead program, which began Phase I testing in 2020, the company expects to nominate three clinical candidates in 2021.
22, 2020 (GLOBE NEWSWIRE) — Replimune Group, Inc. Nasdaq: REPL), a biotechnology company developing oncolytic immuno-gene therapies derived from its Immulytic platform, today announced the pricing of its public offering of 4,687,500 shares of its common stock at a public offering price of $40.00 BOSTON, Oct.
BT-001 is a best-in-class oncolytic Vaccinia virus. platform and its patented large-capacity VV cop TK-RR- oncolytic virus, which has been engineered to encode both a Treg-depleting human recombinant anti-CTLA4 antibody generated by BioInvent’s proprietary n-CoDeR ® /F.I.R.S.T LUND, Sweden and STRASBOURG, France , Dec.
This announcement follows the approval received in December 2020 from the Belgian health authorities. This announcement follows the approval received in December 2020 from the Belgian health authorities. BT-001 IS AN ONCOLYTIC VIRUS GENERATED WITH TRANSGENE’S INNOVATIVE INVIR.IO BT-001 is based on the patented Invir.IO
Investigators at Cedars-Sinai have identified a potential new therapy for COVID-19: a biologic substance created by reengineered human skin cells. Scientists found the substance stopped SARS-CoV-2, the virus that causes COVID-19, from reproducing itself and also protected infected cells when tested in human lung cells.
29, 2020– Alexion Pharmaceuticals, Inc. NASDAQ:ALXN) today announced financial results for the third quarter of 2020. Non-GAAP diluted EPS for the third quarter of 2020 was $3.24 , a 16 percent increase versus the third quarter of 2019. ” Third Quarter 2020 Financial Highlights. in the prior year.
5, 2020 /PRNewswire/ — Third quarter 2020 revenues increased 32% to $2.29 Third quarter 2020 EYLEA ® U.S. Third quarter 2020 Dupixent ® global net sales (2) , which are recorded by Sanofi, increased 69% to $1.07 Third quarter 2020 Dupixent ® global net sales (2) , which are recorded by Sanofi, increased 69% to $1.07
a San Diego-based biotechnology company with an array of technology platforms for antibody discovery and optimization, and novel NK and T cell engager generation, today announced licensing of a panel of its anti-SARS-CoV-2 antibody clones to IGM Biosciences for COVID-19 therapy development.
(Nasdaq: PASG), a genetic medicines company focused on developing transformative therapies for rare monogenic central nervous system (CNS) disorders, today announced that U.S. GM1 is a rare and often life-threatening CNS disorder with no approved disease-modifying therapies available. About PBGM01. About Passage Bio.
B38 blocks SARS-CoV-2 from binding to the ACE2 receptor (light pink) of a human cell, ACE2 is what the virus uses to infect cells. Just one infusion of this combination antibody therapy lowered the amount of viral genetic material in the animals’ lungs by as much as 30 percent compared to the amount in untreated animals.
The COVID-causing virus SARS-CoV-2 harbors a vulnerable site at the base of its spike protein that is found also on closely related coronaviruses, according to a new study from Scripps Research. That prospect has spurred efforts towards the development of a pan-beta-coronaviral vaccine or antibody therapy.
3, 2020 17:00 UTC. Under his leadership, the Company has demonstrated the potential of virus-based immunotherapies and developed world class innovative therapies that could be game-changers in the field of cancer treatment. Sandrine has been Chief Financial Officer of Institut Mérieux since March 2020.
The outcome confirms that only one single case was attributable to the original SARS-CoV-2 virus. “In addition, the variant-rich environment underlines the importance of developing next-generation vaccines as new virus variants continue to emerge.” More than half of the cases (57%) were caused by Variants of Concern.
In terms of people being willing to take a vaccine, 35% of Americans say they have no plans to be inoculated against the virus, according to new polling. AstraZeneca seals manufacturing deal to deliver COVID-19 vaccine in China – Published on 07/08/2020. New Phase 1/2 on Novavax’s COVID-19 vaccine candidate NVX?CoV2373
3, 2020 09:00 UTC. MTX-COVAB was also found to have virus neutralizing potency (IC50= 40pM), which is comparable or better to that of the leading antibody therapies in development, and cross-neutralizes all clinically relevant virus mutants. About Memo Therapeutics AG.
23, 2020 — Fresh takes on the efficacy of fish oil and vitamin D, new treatments for heart failure patients, the cardiovascular effects of COVID-19 , and structural racism as a driver of health disparity were just a few of the major developments that emerged from the American Heart Association Scientific Sessions 2020, held virtually from Nov.
The acquisition provides Gilead with Hepcludex ® (bulevirtide), which was conditionally approved by the European Medicines Agency for the treatment of chronic hepatitis delta virus (HDV) in adults with compensated liver disease in July 2020. Hepcludex is an important new addition to the Gilead portfolio.
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