Drug Discovery and Development Archives

Ori Biotech’s mission to democratize access to cell and gene therapies

London and New Jersey-based cell and gene therapy (CGT) manufacturing technology firm Ori Biotech aims to accelerate CGT manufacturing processes. The company was highlighted on our recent roundup of prominent cell and gene therapy vendors in 2023.

In January 2022, the company secured more than $100 million in a Series B funding round, enabling the move from pre-commercialization to market launch. According to CEO Jason C. Foster, the company has grown from “three of us in the biotech equivalent of a garage” to 72 employees and raised $140 million in venture funding to date.

From investor to CEO of Ori Biotech

Jason Foster

Foster met the Ori team in 2018, working with them as an investor. He later joined full time as CEO after the company closed its first institutional round in 2019.

Ori Biotech has developed a proprietary platform that automates and standardizes CGT manufact…

AlphaFold: Redefining drug discovery with digital biology and AI

[Christoph Burgstedt/Adobe Stock]

At the Nvidia GTC 2023, DeepMind’s Founder and CEO, Demis Hassabis, provided an in-depth look into the seismic potential of their protein folding AI system, AlphaFold. Hassabis said AlphaFold was a contender for the organization’s “biggest project to date.”

Hassabis noted that DeepMind’s AlphaFold has made strides in addressing the protein folding problem, a challenge that has stumped scientists for more than five decades. The protein folding problem involves predicting the 3D structure of proteins solely from the amino acid sequence. Until AlphaFold, determining a protein’s structure was a complex and time-consuming process, often taking years of experimental work.

At the end of 2022, the company announced that it updated AlphaFold with the structure predictions of more than 200 million proteins.

Hassabis explained the enormity of the challenge, …

This RNA sensor could trigger cells to turn on a synthetic gene

Caption:MIT researchers demonstrated that their RNA sensor could accurately identify cells expressing a mutated version of the p53 gene, which drives cancer development. [Image courtesy of MIT/iStock]

Engineers at MIT designed a new way to trigger cells that could lead to targeted therapies for cancer and other diseases.

Using an RNA sensor for the trigger, the researchers ensure that synthetic genes activate only in specific cells. They demonstrated that the sensor could accurately identify cells expressing a mutated version of the p53 gene, which drives cancer development. Additionally, they found they could turn on a gene encoding a fluorescent protein only within those cells.

With this approach, the engineers feel they can develop sensors to trigger production of cell-killing proteins in cancer cells, sparing healthy cells, too. They see the possibility of developing treatments for other diseases, too…

Non-hallucinogenic LSD derivative reduces depression symptoms in preclinical studies

Vancouver-based biotech firm BetterLife Pharma (OTCM:BETRF) is working on a non-hallucinogenic LSD derivative for treating neuropsychiatric and neurological disorders.

Known as BETR-001 (2-bromo-LSD), the compound is also not controlled. Conversely, LSD, like other serotonergic hallucinogens, is a Schedule I compound. That status makes it difficult and expensive to research for potential medical uses.

BetterLife announced recent positive results from preclinical studies for the LSD derivative in reducing depressive symptoms in rats.

The research has been published in Cell Reports.

In vitro studies found that 2-bromo-LSD promoted dendritogenesis and spinogenesis in rat cortical neurons. Additionally, in vivo studies found that it produced active coping behavior and reversed chronic stress deficits. Those effects are likely dependent on activation of the 5-HT2A serotonin receptor. The study concluded that 2-bromo-LSD appears to have potential as a t…

Nvidia launches BioNeMo Cloud as a breakthrough AI service for drug discovery research

[Image courtesy of Nvidia]

During Nvidia’s (Nasdaq:NVDA) GTC event, the company introduced BioNeMo Cloud, a new component to their AI Foundations suite. This service, designed to streamline life sciences research, drug discovery and protein engineering, provides researchers with access to pretrained AI models, allowing for customization with proprietary data. Offered as a cloud service, BioNeMo Cloud enables accelerated drug discovery workflows, with multinational biotech company Amgen (Nasdaq:AMGN) and several startups already utilizing the platform.

BioNeMo Cloud features pretrained AI models for molecular biology, chemistry and molecular dynamics. The platform allows researchers to fine-tune models with proprietary data, executing AI model inference via web browsers or new cloud APIs. This accelerates drug discovery, molecular identification and protein 3D structure prediction.

Featured models in…

Sandoz’s Hyrimoz to challenge AbbVie’s Humira dominance

A robotic arm transfers Humira pens from the conveyor to two-pack cartons. [Image from AbbVie]

The generic and biosimilar company Sandoz, a Novartis (NYSE:SIX) subsidiary, has received FDA approval for its Humira biosimilar Hyrimoz (adalimumab-adaz) injection. AbbVie’s (NYSE:ABBV) megablockbuster Humira is a biologic medication with indications for an array of autoimmune conditions, including rheumatoid arthritis, Crohn’s disease and psoriasis. Sandoz’s biosimilar will likely be priced significantly lower than Humira’s. This fact could dramatically impact the marketplace for AbbVie’s version of the drug, as it is priced lower and could lead to a decrease in market share for Humira.

The FDA approved Sandoz’s Hyrimoz, a biosimilar of AbbVie’s Humira, to treat seven conditions: rheumatoid arthritis, juvenile idiopathic arthritis, psoriatic arthritis, ankylosing spondylitis,…

One-time gene therapy Zolgensma from Novartis shows lasting benefits for SMA patients

[Zolgensma image from Novartis]

A gene therapy for spinal muscular atrophy (SMA) patients is making waves. Novartis (NYSE:NVS) revealed new long-term data highlighting the durability of Zolgensma (onasemnogene abeparvovec) up to 7.5 years after a single treatment. The data comes from two long-term follow-up studies, LT-001 and LT-002, which examined a range of patient populations and demonstrated a positive overall benefit-risk profile. LT-001 is an ongoing 15-year long-term follow-up study of patients who completed the phase 1 START study. More than 3,000 children have been treated with Zolgensma in clinical trials, managed access programs and commercial settings.

Novartis expects Zolgensma to eventually generate $1.5–2 billion, according to BioPharma Dive. NVS shares were up 1 percent to $83.01 today after announcing the new data.

FDA approved Zolgensma (onasemnogene abeparvovec-xioi) in 2019 for chi…

QurAlis targets UNC13A RNA mis-splicing in ALS and FTD with novel FlexASO platform

QurAlis, a privately-held company specializing in precision medicines for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases, has debuted its latest program. This initiative targets UNC13A RNA mis-splicing, a critical gene alteration present in ALS and frontotemporal dementia (FTD). FTD has recently gained renewed attention following the disclosure of Bruce Willis’ diagnosis of the condition. UNC13A is a vital regulator of neurotransmitter release at synapses, and the genetic alteration resulting in mis-splicing is estimated to occur in 58% of ALS patients and up to half of FTD cases.

To tackle this gene alteration, QurAlis has developed its proprietary FlexASO splice modulator platform. This technology uses antisense oligonucleotides (ASOs) to correct mis-splicing, restore UNC13A protein production, and reduce cryptic exons that may contribute to disease progression. Kasper Roet, QurAlis CEO, explained that “FlexASOs operate on the s…

How EEG and machine learning are transforming epilepsy clinical trials

[Image courtesy of peterschreiber.media/Adobe Stock]

Epilepsy is a brain disorder that triggers recurring seizures. It is the fourth the most common neurological disorders in the world, according to the Epilepsy Foundation. The Centers for Disease Control and Prevention estimates that 65 million people worldwide have active epilepsy. In 2015, 1.2% of the total U.S. population — 3 million adults and nearly 500,000 children — had active epilepsy.

There are many different causes of epilepsy, including genetics, head trauma, brain abnormalities, infection, prenatal injury and developmental disorders, such as autism.

Seizure symptoms vary greatly and can manifest in a person as uncontrollable limb movements, staring, muscle stiffness, confusion and loss of consciousness or awareness.

These symptoms are not mutually exclusive. Some patients with epilepsy experience multiple types of seizures.

Epil…

The next wave: 10 promising investigational antihypertensive drugs to watch

[Image courtesy of Adobe Stock]

Cardiovascular diseases remain a leading cause of morbidity and mortality worldwide. This review highlights ten promising antihypertensive drug candidates with the potential to address pulmonary arterial hypertension (PAH), resistant hypertension and uncontrolled hypertension.

Aprocitentan, developed by Janssen NYSE:JNJ) and Idorsia (SIX:IDIA), is an experimental oral drug designed to treat resistant hypertension. As a dual endothelin receptor antagonist (ERA), it effectively blocks the binding of ET-1 to both ET A and ET B receptors. The drug has a low likelihood of interacting with other medications and its mechanism of action is well-suited for treating resistant hypertension. In clinical trials, aprocitentan demonstrated better blood pressure reduction than placebo, with the effects lasting up to week 40, and was generally well-tolerated by patients. (Text continues beneath th…

Harnessing the untapped potential of legacy data in pharma R&D

[Image courtesy of James Thew/Adobe Stock]

Clinical trials for a new therapy cost a median of $41,117 per patient. Costs like this are no surprise to pharma leaders. But during an age of increasing budgetary pressures, drug developers are under pressure to do more with less money and staff. While there are no “simple” answers to this challenge, there is one strategy that offers research and development (R&D) teams a very powerful approach: better leveraging existing legacy data.

Pharmaceutical companies own petabytes of imaging data, generated by in-house research, investigator-initiated studies or clinical trials. This data is valuable and can yield insights that can help researchers better understand disease mechanisms and inform therapeutic approaches. But in many cases, researchers cannot access this important data, as it remains in silos with CROs, investigator labs, or within a specific research group…

Vericel’s unique biopharma focus: Tackling unmet needs in sports medicine and burn treatment

Vericel (Nasdaq:VCEL), a biopharma focused on sports medicine and severe burn care therapies, has a portfolio including the cartilage repair treatment MACI and the burn treatments Epicel and NexoBrid.

Focal cartilage defects, which occur in approximately 60% of arthroscopies, can lead to debilitating osteoarthritis if left untreated. Vericel’s flagship product, MACI, tackles this medical challenge head-on by enabling younger, active patients to avoid knee replacements. Harnessing the power of the patient’s own cells to repair cartilage damage, MACI halts the progression to osteoarthritis.

In the realm of burn care, Vericel’s Epicel serves as a permanent skin replacement for patients with severe burns, while NexoBrid is an enzymatic treatment designed to remove dead tissue from burn wounds. In a recent interview, Vericel CEO Nick Colangelo highlighted the company’s unique position in the market, noting the limited competition and signific…