Drug Discovery and Development Archives - Page 68 of 158

Precision BioSciences says gene-editing technique could potentially cure hepatitis B infection

Gene editing company Precision BioSciences (Nasdaq:DTIL) has announced that preclinical research involving in vivo gene editing targeting hepatitis B virus (HBV) has been published in Molecular Therapy.

In the research, the company used an engineered ARCUS nuclease (ARCUS-POL) targeting the Hepatitis B virus (HBV) genome.

In this preclinical study, ARCUS efficiently targeted and degraded HBV covalently closed circular DNA (cccDNA) by 85%.

Gilead Sciences (Nasdaq:GILD) and Acuitas Therapeutics (Vancouver) collaborated with Precision BioSciences on the research.

Derek Jantz

“We’re very excited to see this study published in Molecular Therapy and to showcase the compelling reductions in cccDNA and surface antigen obtained with our ARCUS gene-editing platform in two novel animal models of HBV infection,” said Derek Jantz, chief scientific officer of Precision BioSciences, in a news release. …

How PolarityTE aims to help heal chronic wounds

Founded in 2016, PolarityTE (Nasdaq:PTE), a company focused on developing regenerative tissue products to address unmet medical needs in chronic wounds, has recently commenced a Phase 3 pivotal study involving patients with diabetic foot ulcers (DFUs).

In addition, the FDA granted its investigational SkinTE product Regenerative Medicine Advanced Therapy (RMAT) designation.

PTE shares, however, skidded 30% to $2.97 today after the Salt Lake City-based company announced a 1-for-25 reverse stock split. Yesterday, the company reported quarterly earnings that missed analysts’ expectations.

The company went public in 2017 after entering a reverse merger with Majesco Entertainment Company, a video game company.

Richard Hague

PolarityTE CEO Richard Hague is optimistic about its prospects given the unmet need for non-healing chronic wounds, the ongoing Phase 3 study of SkinTE and the FD…

FDA allows booster dose Pfizer-BioNTech COVID-19 vaccine in children 5 to 11

The Pfizer-BioNTech vaccine booster had been previously limited in the U.S. to individuals at least 12 years old, but now children as young as 5 are eligible, thanks to an expanded emergency use authorization related to the vaccine.

The agency agreed to expand the vaccine’s use in children to “provide continued protection against COVID-19,” said FDA Commissioner Robert M. Califf in a news release.

While “COVID-19 tends to be less severe in children than adults,” Califf noted that the omicron wave has resulted in more children getting infected and hospitalized with COVID-19 infections. “Children may also experience longer-term effects, even following initially mild disease,” he added.

The FDA has determined that vaccine effectiveness in all authorized populations wanes in the weeks and months following administration of a second dose.

“The FDA has determined that the known and potential benefits of a single booster dose of the Pfizer-BioNTech COVID…

Massachusetts is competing for ARPA-H biomedical research center

[Image courtesy of Pixabay]

The U.S. government is planning to establish the Advanced Research Projects Agency for Health (ARPA-H) to accelerate biomedical research.

The new federal research agency will not be based alongside its NIH parent in Bethesda, Maryland.

After winning authorization to create the agency, Sen. Edward J. Markey (D-Mass.) and several colleagues in Massachusetts are asking the Biden administration to locate the agency there.

The ARPA-H headquarters would also likely serve as a magnet for the broader life sciences industry.

In a letter to U.S. Secretary of Health and Human Services Xavier Becerra, Markey argues that Massachusetts is a premier biomedical research hub, given its talented workforce and network of elite universities, hospitals and research institutions.

“As ARPA-H seeks to accelerate medical breakthroughs and invest in high-risk, high-reward proj…

AstraZeneca paying up to $157M for broad-spectrum monoclonal antibodies against COVID

RQ Biotechnology (London) emerged from stealth mode today, saying that AstraZeneca will pay it up to $157 million plus royalties for its existing mAbs against SARS-CoV-2.

“Our vision is to build on our successful debut with neutralizing antibody therapy for SARS-CoV-2 and develop innovative medicines to address current and evolving unmet needs in other viral infectious diseases,” said Hugo Fry, a former Sanofi executive who is CEO of RQ Bio.

“By combining our expertise and innovative excellence in core areas, we have created a smarter approach to antibody generation, making us uniquely positioned to deliver fast patient impact,” Fry said in a news release.

The agreement gives AstraZeneca an exclusive worldwide license to develop, manufacture and commercialize mAbs against SARS-CoV-2.

“The COVID-19 pandemic has changed the landscape for immune therapies, including the use of monoclonal antibodies to protect vulnerable patien…

GenScript debuts single- and double-strand DNA service for cell and gene therapy development

The popularity of CRISPR-based non-viral gene insertion techniques is fueling interest in CRISPR/Cas genome-editing technology that uses the homology-directed repair (HDR) pathway to insert DNA sequences.

To respond to that demand, biotech services firm GenScript has introduced GMP-compliant single-stranded DNA (ssDNA) and closed-end linear double-stranded DNA (dsDNA) services.

The company’s GenExact ssDNA services support up to 5000 nt in length and 100mg per batch production scale. GenWand services offer closed-end, linear dsDNA up to 10,000 nt in length and gram level/batch production scale.

Launching the services at the American Society of Gene & Cell Therapy annual meeting, Piscataway, New Jersey-based GenScript now boasts a range of non-viral HDR payload materials for early discovery research, process development and clinical trials.

The company said the offerings would also help facilitate CAR-T drug development.

“Th…

Startup vies to get blockchain adoption in pharma off the ground

[Image courtesy of Pixabay]

Blockchain may be arguably one of the most hyped technologies in recent memory, but the distributed ledger technology better known for its role in cryptocurrency may have significant potential in the pharma supply chain, clinical trials and beyond.

A blockchain-based initiative known as PharmaLedger has won support from prominent companies like Pfizer, Novartis, Merck & Co., Bayer and AstraZeneca.

In 2019, the FDA launched a pilot program supporting the Drug Supply Chain Security Act with the support of IBM, KPMG, Merck & Co. and Walmart.

The pandemic’s disruption of the pharmaceutical supply chain further spurred industry interest in exploring blockchain to improve logistics networks, as the Harvard Business Review observed.

Overall adoption of blockchain in pharma or the enterprise at large, however, remains at a nascent stage.

Blockchain in…

Lilly’s tirzepatide wins FDA approval for type 2 diabetes

FDA has approved Mounjaro (tirzepatide), the first glucose-dependent insulinotropic polypeptide (GIP) and glucagon-like peptide-1 (GLP-1) for treating adults with type 2 diabetes.

Developed by Eli and Co., tirzepatide could quickly become a mega-blockbuster. According to Mizuho Securities, annual sales of the drug could approach $14 billion by 2030.

To win approval, Lilly provided data from the Phase 3 SURPASS clinical studies showing that it supported superior A1C reductions than all comparators, including insulin glargine and insulin degludec. The SURPASS trials also pitted tirzepatide against metformin, SGLT2 inhibitors and sulfonylureas.

The drug also has the side benefit of supporting weight loss, although it is not indicated for obesity. Volunteers in the SURPASS trials lost an average of 12 and 25 lb., depending on dose level.

In the SURMOUNT-1 study, the drug yielded up to 22.5% in adults with obesity or overweight.

Lilly will marke…

Black Diamond analysis highlights 22 new oncogenic HER2 driver mutations

On the heels of announcing a restructuring plan that reduces its workforce by 30%, Black Diamond Therapeutics (Nasdaq:BDTX) has announced that its discovery engine was featured in the peer-reviewed Cancer Research journal.

The journal is published by American Association for Cancer Research (AACR).

A paper highlighted in the journal explained how the company’s Mutation-Allostery-Pharmacology (MAP) discovery engine identified and experimentally validated 22 new oncogenic HER2 driver mutations.

The paper is titled “Computational and Functional Analyses of HER2 Mutations Revealing Allosteric Activation Mechanisms and Altered Pharmacologic Effects.”

The analysis highlights work from researchers at Black Diamond Therapeutics and Foundation Medicine (Cambridge, Massachusetts), who used the MAP discovery engine to produce a list of 222 known and potential driver mutations from 820 single-nucleotide variants.

The scientists screened 111 using Ba/F3…

Axogen announces positive topline data from Phase 3 nerve graft study

Focused on developing peripheral nerve regeneration and repair products, Axogen (Nasdaq:AXGN) announced that the Phase 3 RECON study met its primary endpoint in comparing its Avance nerve graft to conduits in digital nerve injuries.

The primary endpoint was the return of sensory function based on static two-point discrimination. This test measures the ability to detect that two close objects touching the skin are distinct points.

“The study data confirmed that as gap lengths increased, Avance returned superior levels of sensation, and this was achieved at earlier time points than those observed for conduits,” said co-lead Investigator Dr. Jonathan Isaacs, professor and chair, division of hand surgery at Virginia Commonwealth University.

The Avance nerve graft also had a good safety profile in the study.

Axogen plans to submit a Biologics License Application (BLA) for the product in the second half of 2023.

NIH licenses COVID-19 research technologies to WHO initiative

The Biden-Harris administration has announced that the NIH has licensed COVID-19 research technologies and vaccine candidates to the Medicines Patent Pool (MPP).

Working with the WHO’s COVID-19 Technology Access Pool (C-TAP), the NIH will provide licenses to enable manufacturers to use the technologies to develop COVID-19 vaccines, treatments and diagnostics.

In total, NIH is providing 11 COVID-19 technologies under two licenses, bringing the number of technologies licenses to MPP to the following 14 items:

A VSV-EBOV-based vaccine candidate. This SARS-CoV-2 vaccine candidate is based on a modified approved Ebola vaccine known as Ervebo from Merck & Co. ACE2 Dimer construct (Research tool for drug development). This technology is a plasmid encoding human ACE2 dimers, which refers to two conjoined copies of ACE2. ACE2 is a protein involved in COVID-19 infections. NIH notes that the research tool can help isolate antibodies generated against SARS-CoV-2. …

Nine out of ten U.S. neurologists have Aduhelm efficacy doubts

A recent physician survey found that 89% of neurologists in the U.S. have concerns about the efficacy of the controversial Alzheimer’s drug Aduhelm (aducanumab) from Biogen (Nasdaq:BIIB) and Eisai Co. (TYO:4523). Nearly as many — 87% — were concerned about the drug’s safety profile.

The survey from the physician social media network Sermo (New York City) asked more than 50 neurologists about their view of the drug, which has failed to find widespread use, generating just $3 million in 2021. Only 11% of the neurologists surveyed reported prescribing the drug.

Despite low uptake, many patients are aware of the drug. A total of 62% of the surveyed neurologists reported that at least one patient had asked them about Aduhelm.

The drug has faced an array of challenges since it was FDA-approved in June 2021. Recently, Medicare decided to limit Aduhelm’s coverage of the drug to clinical trial participants, and other payers, including the Department of Veteran Af…