FDA Approval, Packaging and Virus - Drug Discovery Digest

A Visual Guide to Genome Editors

Codon

JUNE 5, 2025

The treatment, now known as Casgevy, became the first CRISPR-based therapy to gain FDA approval, in 2023. If the same virus invades the cell a second time, the gRNA’s spacer sequence will bind to the matching viral DNA sequence, then be cut by the Cas protein.

DNA

DNA RNA Clinical Trials Engineering

Levers for Biological Progress

Codon

NOVEMBER 1, 2024

They engineered genetically-encoded RNA exporters, based on viruses, that package and secrete RNA molecules in protective nanoparticles, allowing non-destructive monitoring of those RNA molecules in real-time. Today, a single injection of an FDA-approved gene therapy, called Hemgenix , cures this disease.

DNA

DNA RNA Molecular Biology Laboratories

New gene delivery vehicle shows promise for human brain gene therapy

Broad Institute

MAY 16, 2024

But FDA-approved forms of the most commonly used vehicle for packaging and delivering these therapies to target cells, adeno-associated viruses (AAVs), aren’t able to efficiently cross the blood-brain barrier at high levels and deliver therapeutic cargo.

Therapies

Therapies FDA Approval Engineering Disease

Researchers reprogram gene therapy viral vectors to bind specific protein targets

Broad Institute

JULY 19, 2023

By Allessandra DiCorato July 19, 2023 Credit: Kevin Middleton, Broad Communications A three-dimensional model of adeno-associated viruses (AAVs), which scientists have engineered to package and deliver gene therapies to cells in the body.

Therapies

Therapies Research Engineering Virus

What FDA’s Newest Gene Therapy Approval Tells Us About Durability: How Long is Long Enough?

FDA Law Blog: Drug Discovery

DECEMBER 7, 2022

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.

Therapies

Therapies FDA FDA Approval Treatment

Altasciences Chosen by Virpax to Support the Development of a New Drug to Prevent Spread of Flu-like Viruses

Alta Sciences

SEPTEMBER 20, 2023

AnQlar is a nanoparticle intended to help prevent the spread of negatively charged viruses such as COVID-19 and influenza via intranasal spray, and has been shown to inhibit the ability of the virus to replicate at non-toxic concentrations. We look forward to further collaboration with Virpax in the near-future.”

Virus

Virus Clinical Pharmacology Drugs FDA Approval

New insights into the role of viral capsids in gene therapy safety

Drug Target Review

DECEMBER 5, 2024

1 In 2017, the US Food and Drug Administration (FDA) approved the first AAV-based gene replacement therapy (Luxturna), for Leber congenital amaurosis type 2. 2 Since then, the FDA has approved four more AAV-based gene therapies—Zolgensma, Hemgenix, Elevidys and Rocktavian—for treating various diseases. Labcompare.

Therapies

Therapies Virus DNA Compliance

Regeneron’s COVID-19 Outpatient Trial Prospectively Demonstrates that REGN-COV2 Antibody Cocktail Significantly Reduced Virus Levels and Need for Further Medical Attention

The Pharma Data

OCTOBER 28, 2020

In the overall patient group with detectable virus at baseline, the average daily reduction in viral load through day 7 was a 0.36 About REGN-COV2 REGN-COV2 is a combination of two monoclonal antibodies (REGN10933 and REGN10987) and was designed specifically to block infectivity of SARS-CoV-2, the virus that causes COVID-19.

Virus

Virus Trials Clinical Trials Production

DNA Barcodes Could Streamline Search for New Drugs to Combat Cancer

NIH Director's Blog: Drug Discovery

MARCH 8, 2016

Instead of the black, printed stripes of the Universal Product Codes (UPCs) that we see on everything from package deliveries to clothing tags, they used short, unique snippets of DNA to label cells. A little more than a decade ago, researchers began adapting a familiar commercial concept to genomics: the barcode.

DNA

DNA Drugs Small Molecule FDA Approval

REGN-COV2 Independent Data Monitoring Committee Recommends Holding Enrollment in Hospitalized Patients with High Oxygen Requirements and Continuing Enrollment in Patients with Low or No Oxygen Requirements

The Pharma Data

OCTOBER 30, 2020

About REGN-COV2 REGN-COV2 is a combination of two monoclonal antibodies (REGN10933 and REGN10987) and was designed specifically to block infectivity of SARS-CoV-2, the virus that causes COVID-19.

Hospitals

Hospitals Virus Production Clinical Trials

New England Journal of Medicine Publishes Positive Initial Regeneron Antibody Cocktail Results in Non-hospitalized Patients with COVID-19

The Pharma Data

DECEMBER 17, 2020

About casirivimab and imdevimab Casirivimab and imdevimab (formerly known as REGN-COV2 or REGEN-COV2) is a cocktail of two monoclonal antibodies (also known as REGN10933 and REGN10987, respectively) and was designed specifically to block infectivity of SARS-CoV-2, the virus that causes COVID-19.

Hospitals

Hospitals Production Clinical Trials Virus

Article FDA Thank You FDA finalizes guidance on electronic submissions for OTC products

Agency IQ

JULY 26, 2024

Prior to CARES Act reforms, FDA relied on a monograph process through which firms could bring OTC drugs to market without FDA approval so long as they adhered to pre-set terms under the monograph. On the other hand, data, information and comments to a proposed or final order should use the OTC Monographs@FDA portal.

FDA

FDA Production Regulations Packaging

Regeneron’s BCMAxCD3 Bispecific Antibody (REGN5458) Shows Deep and Durable Responses in Patients with Heavily-pretreated Multiple Myeloma in Phase 1

The Pharma Data

DECEMBER 5, 2020

Founded and led for 30 years by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to eight FDA-approved treatments and numerous product candidates in development, all of which were homegrown in our laboratories.

Production

Production Trials Treatment Disease

Janssen Announces U.S. FDA Approval of CABENUVA (rilpivirine and cabotegravir), the First Long-Acting Regimen for the Treatment of HIV

The Pharma Data

JANUARY 21, 2021

Food and Drug Administration (FDA) has approved CABENUVA (consisting of Janssen’s rilpivirine and ViiV Healthcare’s cabotegravir), the first and only once-monthly, long-acting regimen for the treatment of human immunodeficiency virus type 1 (HIV-1) infection in adults.

FDA Approval

FDA Approval Treatment RNA FDA

Regeneron Announces U.S. Government Agreement to Purchase Additional COVID-19 Antibody Cocktail Doses

The Pharma Data

JANUARY 12, 2021

. “Patients in our antibody cocktail outpatient clinical trial experienced significant reductions in virus levels and required fewer medical visits for COVID-19, suggesting the therapy can help reduce the current burden on hospitals and healthcare systems,” said George D. Yancopoulos , M.D.,

Government

Government Hospitals Virus Clinical Trials

Months of Work Hang in the Balance as Moderna’s COVID-19 Vaccine Awaits AdComm Vote

The Pharma Data

DECEMBER 16, 2020

To this point, Moderna has only submitted two months of follow-up safety data, and the FDA typically requires six months for a full approval. intends to ship just shy of six million doses of Moderna’s vaccine once the FDA approves EUA. Initial doses are expected to be limited as manufacturing ramps up.

Vaccine

Vaccine Immune Response FDA Virus

Serum Institute of India Obtains Emergency Use Authorisation in India for AstraZeneca’s COVID-19 Vaccine

The Pharma Data

JANUARY 6, 2021

We hope this effective, well-tolerated and simple-to-administer vaccine will now begin to have a real impact on this deadly virus. After vaccination, the surface spike protein is produced, priming the immune system to attack the SARS-CoV-2 virus if it later infects the body. AZD1222 (SARS-CoV-2 vaccine) FDA Approval History.

Vaccine

Vaccine Virus Clinical Trials Government

Regeneron’s Casirivimab and Imdevimab Antibody Cocktail for COVID-19 is First Combination Therapy to Receive FDA Emergency Use Authorization

The Pharma Data

NOVEMBER 20, 2020

“The casirivimab and imdevimab antibody cocktail is designed to mimic what a well-functioning immune system does by using very potent antibodies to neutralize the virus,” said George D. Casirivimab and imdevimab injection is an investigational combination therapy and has been authorized by FDA for the emergency use described above.

Therapies

Therapies FDA Clinical Trials Hospitals

Codon Digest: Ghana Approves A Better Malaria Vaccine

Codon

APRIL 23, 2023

Most people would take the two CRISPR gene-editing components (a Cas9 protein and guide RNA), package them up inside of a virus, and then inject the viruses into the skulls of mice. Promising new malaria vaccine for kids approved in Ghana. If you want to edit a gene inside of a neuron, what do you do? From Stahl E.C. in bioRxiv.

Vaccine

Vaccine DNA Virus Engineering

Codon Digest: Ghana Approves A Better Malaria Vaccine

Codon

APRIL 23, 2023

Most people would take the two CRISPR gene-editing components (a Cas9 protein and guide RNA), package them up inside of a virus, and then inject the viruses into the skulls of mice. Promising new malaria vaccine for kids approved in Ghana. If you want to edit a gene inside of a neuron, what do you do? From Stahl E.C. in bioRxiv.

Vaccine

Vaccine DNA Virus Engineering

Regeneron’s REGEN-COV2 is First Antibody Cocktail for COVID-19 to Receive FDA Emergency Use Authorization

The Pharma Data

NOVEMBER 21, 2020

.” “REGEN-COV2 is designed to mimic what a well-functioning immune system does by using very potent antibodies to neutralize the virus,” said George D. Casirivimab and imdevimab injection (REGEN-COV2) is an investigational combination therapy and has been authorized by FDA for the emergency use described above.

FDA

FDA Clinical Trials Hospitals Production

Regeneron Reports Third Quarter 2020 Financial and Operating Results

The Pharma Data

NOVEMBER 5, 2020

FDA accepted for priority review Libtayo ® (cemiplimab-rwlc) for both advanced non-small cell lung cancer and basal cell carcinoma. FDA approved Inmazeb for Ebola ( Zaire ebolavirus). REGN-COV2 , a dual antibody therapy to SARS-CoV-2 virus. Regeneron Pharmaceuticals, Inc. Schleifer , M.D., In July 2020 , the U.S.

Production

Production FDA Trials FDA Approval

Codon Digest: Injected Gene Editors

Codon

APRIL 2, 2023

Read Inhalable nanoparticles, packaged with mRNA or CRISPR systems, efficiently edit lung cells. Therapeutic neutralizing monoclonal antibody administration protects against lethal yellow fever virus infection. Read A really simple way to isolate AAVs, which are a type of virus often used to deliver gene therapies into cells.

DNA

DNA RNA Engineering Licensing

Regeneron Announces Encouraging Initial Data from COVID-19 Antibody Cocktail Trial in Hospitalized Patients on Low-flow Oxygen

The Pharma Data

DECEMBER 29, 2020

About Regeneron Antibody Cocktail Casirivimab and imdevimab is a cocktail of two monoclonal antibodies (also known as REGN10933 and REGN10987, respectively) and was designed specifically to block infectivity of SARS-CoV-2, the virus that causes COVID-19. Casirivimab and imdevimab injection is not FDA approved for any use.

Hospitals

Hospitals Trials Clinical Trials Production

Fujifilm to Invest Over 200 Billion yen (2 Billion USD) to Establish New Large-Scale Cell Culture Manufacturing Site for Biopharmaceuticals in the U.S.A.

The Pharma Data

JANUARY 6, 2021

End-to-End Single-Site Solution from Drug Substances to Fill-Finish & Packaging. In addition to drug substance manufacture, the facility will also provide commercial scale, automated fill-finish and assembly, packaging and labeling services. Packaging line?. TOKYO , Jan. 8 x 20,000L bioreactors for mammalian cells.

Packaging

Packaging Small Molecule Production FDA Approval

Moderna Announces Primary Efficacy Analysis in Phase 3 COVE Study for Its COVID-19 Vaccine Candidate and Filing Today with U.S. FDA for Emergency Use Authorization

The Pharma Data

NOVEMBER 30, 2020

“I want to thank the thousands of participants in our Phase 1, Phase 2 and Phase 3 studies, as well as the staff at clinical trial sites who have been on the front lines of the fight against the virus. MRNA-1273 (SARS-CoV-2 vaccine) FDA Approval History. The Company expects that the U.S. Source: Moderna, Inc. . Source link.

Vaccine

Vaccine FDA Clinical Trials Disease

Article EMA Thank You EMA’s 2023 new medicines report suggests return to pre-pandemic volume

Agency IQ

JANUARY 19, 2024

The FDA CRL cited inspection findings that arose from a “multi-sponsor inspection of a third-party, contract manufacturing organization” which involved the monoclonal antibody drug substance for lebrikizumab. The clinical package, safety and label were not affected. However, BeiGene announced that it is seeking approval in the U.S.

Biosimilars

Biosimilars FDA Production Treatment

Analysis Life Sciences Thank You What We Expect the FDA to do in November 2023

Agency IQ

OCTOBER 27, 2023

Title Type Date Cleared by OIRA Legal Deadline None Notable FDA Comment Periods Closing in November and December FDA comment periods are typically open for 30-60 days, unless they are extended. FDA has proposed to amend the 503A Bulks List by placing additional bulk drug substances on the list.

FDA

FDA Science Regulations Production

Analysis Life Sciences Thank You The U.S. regulatory landscape for probiotics and other microbiome-based therapies

Agency IQ

NOVEMBER 3, 2023

The prebiotic may either feed the probiotic that it is packaged with, or it may feed another beneficial microbe that is intended to complement the activity of the probiotic. According to the FDA, a recombinant LBP is distinctive from a standard LBP. As the name suggests, a synbiotic is a product that seeks “synergy” from its contents.

Therapies

Therapies Science FDA Production

FDA Approves Inmazeb (atoltivimab, maftivimab and odesivimab-ebgn) Antibody Cocktail for Ebola (Zaire Ebolavirus)

The Pharma Data

OCTOBER 14, 2020

Food and Drug Administration (FDA) approved Inmazeb (atoltivimab, maftivimab and odesivimab-ebgn) for the treatment of infection caused by Zaire ebolavirus in adult and pediatric patients, including newborns of mothers who have tested positive for the infection. A Randomized, Controlled Trial of Ebola Virus Disease Therapeutics.

FDA Approval

FDA Approval FDA Production Treatment

The Lancet Publishes Results from Pivotal Global Phase 3 Program Evaluating RINVOQ® (upadacitinib) in Atopic Dermatitis

The Pharma Data

JUNE 18, 2021

FDA approval for adult patients with moderately to severely active rheumatoid arthritis who have had an inadequate response or intolerance to methotrexate. RINVOQ ® (upadacitinib) [Package Insert]. 14 In August 2019 , RINVOQ received U.S. Some people have died from these infections. ClinicalTrials.gov. North Chicago, Ill.

Treatment

Treatment Disease FDA Therapies

Making a “Miracle” HIV Medicine

Codon

FEBRUARY 23, 2025

Experimenting with a retrovirus called murine leukemia virus , he found that zidovudine did, in fact, obstruct retrovirus replication by inhibiting reverse transcriptase. Zidovudine showed promise against multiple HIV strains in cultured cells, and the Food and Drug Administration (FDA) approved it for human studies within five months.

Clinical Trials

Clinical Trials Virus Vaccine DNA

Drug Discovery Digest

A Visual Guide to Genome Editors

Levers for Biological Progress

Trending Sources

New gene delivery vehicle shows promise for human brain gene therapy

Researchers reprogram gene therapy viral vectors to bind specific protein targets

What FDA’s Newest Gene Therapy Approval Tells Us About Durability: How Long is Long Enough?

Altasciences Chosen by Virpax to Support the Development of a New Drug to Prevent Spread of Flu-like Viruses

New insights into the role of viral capsids in gene therapy safety

Regeneron’s COVID-19 Outpatient Trial Prospectively Demonstrates that REGN-COV2 Antibody Cocktail Significantly Reduced Virus Levels and Need for Further Medical Attention

DNA Barcodes Could Streamline Search for New Drugs to Combat Cancer

REGN-COV2 Independent Data Monitoring Committee Recommends Holding Enrollment in Hospitalized Patients with High Oxygen Requirements and Continuing Enrollment in Patients with Low or No Oxygen Requirements

New England Journal of Medicine Publishes Positive Initial Regeneron Antibody Cocktail Results in Non-hospitalized Patients with COVID-19

Article FDA Thank You FDA finalizes guidance on electronic submissions for OTC products

Regeneron’s BCMAxCD3 Bispecific Antibody (REGN5458) Shows Deep and Durable Responses in Patients with Heavily-pretreated Multiple Myeloma in Phase 1

Janssen Announces U.S. FDA Approval of CABENUVA (rilpivirine and cabotegravir), the First Long-Acting Regimen for the Treatment of HIV

Regeneron Announces U.S. Government Agreement to Purchase Additional COVID-19 Antibody Cocktail Doses

Months of Work Hang in the Balance as Moderna’s COVID-19 Vaccine Awaits AdComm Vote

Serum Institute of India Obtains Emergency Use Authorisation in India for AstraZeneca’s COVID-19 Vaccine

Regeneron’s Casirivimab and Imdevimab Antibody Cocktail for COVID-19 is First Combination Therapy to Receive FDA Emergency Use Authorization

Codon Digest: Ghana Approves A Better Malaria Vaccine

Codon Digest: Ghana Approves A Better Malaria Vaccine

Regeneron’s REGEN-COV2 is First Antibody Cocktail for COVID-19 to Receive FDA Emergency Use Authorization

Regeneron Reports Third Quarter 2020 Financial and Operating Results

Codon Digest: Injected Gene Editors

Regeneron Announces Encouraging Initial Data from COVID-19 Antibody Cocktail Trial in Hospitalized Patients on Low-flow Oxygen

Fujifilm to Invest Over 200 Billion yen (2 Billion USD) to Establish New Large-Scale Cell Culture Manufacturing Site for Biopharmaceuticals in the U.S.A.

Moderna Announces Primary Efficacy Analysis in Phase 3 COVE Study for Its COVID-19 Vaccine Candidate and Filing Today with U.S. FDA for Emergency Use Authorization

Article EMA Thank You EMA’s 2023 new medicines report suggests return to pre-pandemic volume

Analysis Life Sciences Thank You What We Expect the FDA to do in November 2023

Analysis Life Sciences Thank You The U.S. regulatory landscape for probiotics and other microbiome-based therapies

FDA Approves Inmazeb (atoltivimab, maftivimab and odesivimab-ebgn) Antibody Cocktail for Ebola (Zaire Ebolavirus)

The Lancet Publishes Results from Pivotal Global Phase 3 Program Evaluating RINVOQ® (upadacitinib) in Atopic Dermatitis

Making a “Miracle” HIV Medicine

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