Genetics

4BIO Capital’s review of AAV gene therapy clinical trials published in Nature Reviews Drug Discovery

Monday, January 25, 2021 - 12:00pm

LONDON & BOSTON 4BIO Capital (4BIO or the Group), an international venture capital firm focused solely on the advanced therapies sector, announces the publication of a systematic review paper entitled The Clinical Landscape for AAV Gene Therapies in Nature Reviews Drug Discovery.

Key Points: 
  • LONDON & BOSTON 4BIO Capital (4BIO or the Group), an international venture capital firm focused solely on the advanced therapies sector, announces the publication of a systematic review paper entitled The Clinical Landscape for AAV Gene Therapies in Nature Reviews Drug Discovery.
  • The review, led by Dr. Dima Kuzmin in collaboration with academics from the University of Oxford and Childrens Hospital of Philadelphia as well as several members of 4BIOs management and advisory teams, analyses 149 unique adeno-associated virus (AAV) gene therapy clinical trials, determining current key trends and depicting the impact of clinical trials on the gene therapy field.
  • This is the first systematic review and meta-analysis that establishes transition probabilities, cumulative safety, and efficacy data for AAV gene therapies.
  • The authors show that AAV gene therapy is generally safe and well tolerated, with no clinical trials failing to reach their primary safety endpoints prior to the cut-off date of the review.

Worldwide Tissue Banking Industry to 2025 - Technological Advances in Drug Discovery and Development are Driving Growth - ResearchAndMarkets.com

Friday, January 22, 2021 - 3:54pm

The key factor contributing to the growth of the segment is mainly driven by growing research activities and increasing the focus of market players on product development, along with the rising burden of chronic diseases.

Key Points: 
  • The key factor contributing to the growth of the segment is mainly driven by growing research activities and increasing the focus of market players on product development, along with the rising burden of chronic diseases.
  • Factors, such as the aging population, lifestyles changes, leads to long-term health problems, and their treatment can be expensive.
  • The United States market is driven by increasing R&D activities, along with rising burden of chronic diseases.
  • Different pharmaceutical/ biotech companies, academia, and research institutes are utilizing tissue banks for the storage of various samples.

Vaxess Technologies Awarded NSF and DARPA Funding for Development of mRNA-Based Single-Dose, Refrigeration-Free Vaccine Patch

Friday, January 22, 2021 - 3:00pm

The MIMIX platform patch technology provides a solution to challenges related to global vaccine distribution and delivery.

Key Points: 
  • The MIMIX platform patch technology provides a solution to challenges related to global vaccine distribution and delivery.
  • The patch is also compatible with a wide range of COVID-19 vaccine candidates, as demonstrated by Vaxess work towards a combination seasonal protection patch that includes both a COVID-19 vaccine and a broadly protective seasonal flu vaccine.
  • Funding from the NSF and DARPA represent a renewed commitment from the Federal Government to support the development of novel vaccines to combat the current pandemic and future threats, said Vaxess co-founder Livio Valenti.
  • Vaxess has validated performance and compatibility with a range of molecules including proteins, killed viruses, mRNA, hormones, and live viruses.

Announcing Marketing Approval for Hunterase ICV Injection 15 mg, the World’s First Enzyme Replacement Therapy for Mucopolysaccharidosis Type II (Hunter Syndrome) Administered by ICV Injection

Friday, January 22, 2021 - 2:00pm

(Clinigen), headquartered in Tokyo, has received on January 22, 2021, Japan manufacturing and marketing approval for Hunterase ICV (intracerebroventricular) Injection 15 mg (generic name: idursulfase-beta (recombinant)) as a treatment for mucopolysaccharidosis type II (Hunter syndrome).

Key Points: 
  • (Clinigen), headquartered in Tokyo, has received on January 22, 2021, Japan manufacturing and marketing approval for Hunterase ICV (intracerebroventricular) Injection 15 mg (generic name: idursulfase-beta (recombinant)) as a treatment for mucopolysaccharidosis type II (Hunter syndrome).
  • An enzyme-replacement therapy drug for intravenous injection is already in use in Japan and other countries as a treatment for systemic symptoms of mucopolysaccharidosis type II.
  • The approval in Japan is ahead of the world, being the first approval of Hunterase ICV Injection in any country worldwide.
  • Hunterase ICV Injection 15 mg is also the worlds first and only drug for the treatment of central nervous system symptoms of mucopolysaccharidosis type II.

Vertex to Announce Fourth-Quarter 2020 Financial Results on February 1

Friday, January 22, 2021 - 2:01pm

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) will report its fourth-quarter 2020 financial results on Monday, February 1, 2021 after the financial markets close.

Key Points: 
  • Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) will report its fourth-quarter 2020 financial results on Monday, February 1, 2021 after the financial markets close.
  • To access the call, please dial (866) 501-1537 (U.S.) or +1 (720) 545-0001 (International).
  • To ensure a timely connection, it is recommended that participants register at least 15 minutes prior to the scheduled webcast.
  • Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases.

Xalud Therapeutics Appoints Diem Nguyen, Ph.D., as Chief Executive Officer

Friday, January 22, 2021 - 1:00pm

Xalud Therapeutics, a clinical-stage biotechnology company that has developed a non-viral gene therapy platform to treat pathologic inflammation through immune modulation, today announced the appointment of Diem Nguyen, Ph.D., as its chief executive officer.

Key Points: 
  • Xalud Therapeutics, a clinical-stage biotechnology company that has developed a non-viral gene therapy platform to treat pathologic inflammation through immune modulation, today announced the appointment of Diem Nguyen, Ph.D., as its chief executive officer.
  • Diems vision and leadership will enable Xalud to realize the full potential of its non-viral gene therapy platform.
  • Diem has a tremendous track record of building highly successful biopharma business units across therapeutic areas, functionalities and geographies.
  • Xalud Therapeutics is a clinical-stage biotechnology company developing a non-viral gene therapy platform to treat pathologic inflammation through immune modulation.

AVROBIO to Present at the Cowen 2021 Gene Therapy: CMC & Regulatory Summit

Friday, January 22, 2021 - 12:00pm

AVROBIO, Inc. (Nasdaq:AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced Azadeh Golipour, VP of Manufacturing Operations at AVROBIO, will present virtually at the Cowen 2021 Gene Therapy: CMC & Regulatory Summit at 11:30 a.m.

Key Points: 
  • AVROBIO, Inc. (Nasdaq:AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced Azadeh Golipour, VP of Manufacturing Operations at AVROBIO, will present virtually at the Cowen 2021 Gene Therapy: CMC & Regulatory Summit at 11:30 a.m.
  • Our vision is to bring personalized gene therapy to the world.
  • AVROBIO is powered by our industry leading plato gene therapy platform, our foundation designed to deliver gene therapy worldwide.
  • AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.

Gel Electrophoresis Market 2020-2024 | Increasing Incidence of Chronic and Infectious Diseases to be a Key Driver | Technavio

Thursday, January 21, 2021 - 6:11pm
Key Points: 
  • https://www.technavio.com/report/report/gel-electrophoresis-market-indus...
    The gel electrophoresis market is driven by the increasing incidence of chronic and infectious diseases.
  • In addition, rapid growth potential in emerging economies is anticipated to boost the growth of the gel electrophoresis market.
  • The prevalence of various chronic diseases such as cancer, neurological diseases, and cardiovascular diseases is increasing across the world.
  • Gel Electrophoresis Market Product Outlook (Revenue, USD Million, 2020-2024)
    Gel Electrophoresis Market End-user Outlook (Revenue, USD Million, 2020-2024)
    Gel Electrophoresis Market Geography Outlook (Revenue, USD Million, 2020-2024)
    Are you a start-up willing to make it big in the business?

Global Cancer Gene Therapy Market Research during 2021-2025 | Industry Planning Analysis for the New Normal | Technavio

Thursday, January 21, 2021 - 3:26pm
Key Points: 
  • View the full release here: https://www.businesswire.com/news/home/20210121005646/en/
    Technavio has announced its latest market research report titled Global Cancer Gene Therapy Market 2021-2025 (Graphic: Business Wire)
    Worried about the impact of COVID-19 on your business?
  • The report on the cancer gene therapy market provides a holistic update, market size and forecast, trends, growth drivers, and challenges, as well as vendor analysis.
  • The cancer gene therapy market analysis includes the application and geography landscape.
  • The cancer gene therapy market covers the following areas:

Meissa Announces First Dosing in Phase 2 Study of Intranasal Live Attenuated Vaccine Candidate for RSV

Thursday, January 21, 2021 - 2:00pm

Meissa Vaccines (Meissa), a biotechnology company developing vaccines to prevent viral respiratory infections, announced today that the first adult participants have been dosed in a Phase 2 study of MV-012-968, the companys intranasal live attenuated vaccine candidate against respiratory syncytial virus (RSV).

Key Points: 
  • Meissa Vaccines (Meissa), a biotechnology company developing vaccines to prevent viral respiratory infections, announced today that the first adult participants have been dosed in a Phase 2 study of MV-012-968, the companys intranasal live attenuated vaccine candidate against respiratory syncytial virus (RSV).
  • Meissa is developing MV-012-968, an intranasal (needle-free), live attenuated vaccine candidate, to protect infants and at-risk, older adults from RSV.
  • Meissa plans to initiate a Phase 1c study in sero-negative infants in the first half of this year.
  • Meissa is advancing live attenuated vaccine candidates against life threatening respiratory viruses, including respiratory syncytial virus (RSV), SARS-CoV-2 (COVID-19), and human metapneumovirus (hMPV).