Pharmaceutical Technology Editors

The formation of AION Labs, a new biotechnology innovation laboratory, was announced on Oct.13, 2021. This first-of-its-kind innovation lab is meant to spearhead the adoption of artificial intelligence (AI) technologies and computational science to solve therapeutic challenges.

AION Labs was formed from the alliance of four leading pharmaceutical companies—AstraZeneca, Merck KGaA, Pfizer, and Teva Pharmaceutical Industries—and a hi-tech and biotech investment firm—Amazon Web Services (AWS) and Israel Biotech Fund (IBF)— respectively. The alliance aims to build groundbreaking AI and computational ventures that will leverage the cloud to transform how new therapies are discovered and developed.

The purpose of AION Labs is to create and invest in early-stage start-up teams focused on AI and computational biology in drug discovery and development. The lab will offer each team resources and mentorship while working closely with them to develop new technologies that answer the most significant challenges in the pharmaceutical industry. Each start-up will use AI along with the scale and security of the cloud to find new treatments faster and more efficiently, avoid animal experiments, and advance healthcare towards patient-centric precision medicine.

AION Labs will include both a wet lab for biomedical research and a cloud-based computational lab environment that focuses on the development of new algorithms and computational methods to accelerate the discovery and development of potential new therapies. Both wet lab and computational lab will be under the guidance of top senior researchers and experts from AION Labs’ partners.

In addition, AION Labs has formed a strategic partnership with BioMed X, an independent biomedical research institute based in Heidelberg, Germany, giving AION Labs a vehicle with which to seed biomedical innovations at the interface between academic research and the pharmaceutical industry. BioMed X’s innovation model, based on global crowdsourcing and local incubation of research talents and ideas, will serve as the R&D engine to drive AION Labs’ venture creation model.

The lab launched following receipt of a government tender in December 2020 for establishing an innovation lab based on the identification of the life sciences sector as a vital area for growth and investment by the Israel Innovation Authority.

Mati Gill, a former senior executive at Teva Pharmaceuticals, has been appointed CEO of the lab. Yair Benita, former head of computational biology at Compugen and science operations at CytoReason and principal scientist at Merck & Co., has been appointed chief technology officer.

“We are excited to launch our new Innovation Lab for new drug and device discoveries,” said Gill in a company press release. “Our R&D needs-based approach, coupled with our outstanding alliance, is poised to transform the process of therapeutic discovery and development. With a strong talent pool, AI technology at our core, and a significant commitment of the Israeli government, we hope to contribute to the health and well-being of humankind. I’m honored to lead such a meaningful initiative, and to work closely with our consortium members to solve many of the world’s critical health issues.”

“We are excited to extend our successful innovation model to Israel,” said Christian Tidona, founder and managing director of BioMed X, in the press release. “In our view, Israel is currently the best place to jointly leverage the power of artificial intelligence for drug discovery and development together with leading global players in pharmaceuticals, tech, and venture capital.”

Articles

AION Labs will be a first-of-its-kind AI/pharma and computational biology innovation lab aimed at spearheading the adoption of AI technologies and computational science. 

AstraZeneca, Merck KGaA, Pfizer, and Teva Team up with Tech and Investment Leaders to Launch New Biotech Innovation Lab 

Bionano Genomics announced their $100 million acquisition of BioDiscovery Inc., a software company specializing in genomics data, on Oct. 12th, 2021, in a press release. This grants them access to BioDiscovery’s N

 Clinical software, which the company plans to integrate into their optical genome mapping (OGM) software.

 uses next-generation sequencing (NGS) and microarray data to provide data analysis and visualization of copy number variants (CNVs) and single-nucleotide variants (SNVs), providing a consolidated view of the genome. According to the company press release, adding OGM data to this platform would streamline the process of visualizing, interpreting, and reporting structural variant calls, potentially reducing processing time from hours to less than 20 minutes.

 that incorporates their OGM data (alongside existing data types) into the software. In the future, additional capabilities such as RNA expression profiling, epigenetics with methylation, and proteomics will be considered for addition.

“This acquisition accelerates our efforts to make OGM ubiquitous by enabling us to simplify the assessment of clinically-relevant variants in cytogenomics applications, potentially reducing interpretation time per sample and expanding our reach into the discovery and translational research markets where the combination of NGS and OGM can reveal more answers in genetic disease and cancer research,” said Erik Holmlin, CEO of Bionano Genomics, in the press release.

“I believe Bionano Genomics is a natural fit for BioDiscovery, with a shared passion for delivering an exceptional customer experience and revealing all answers across the genome,” said Soheil Shams, founder and CEO of BioDiscover, in the press release. “Bionano’s Saphyr system, which delivers OGM data today, enables Bionano to offer the most comprehensive genome analysis by combining NGS with OGM data in one integrated workflow so all variants across the genome, from single base to full chromosomes, can be assessed for better insights towards elevating human health.”

The deal is expected to close before Oct. 22, 2021.

This acquisition grants Bionano access to BioDiscovery’s clinical software solution for variant analysis.

Bionano Genomics Acquires BioDiscovery Inc. for $100 Million

Intellia Therapeutics, a genome editing company developing curative therapeutics using clustered regularly interspaced short palindromic repeats (CRISPR)-associated protein-9 (CRISPR/Cas9), announced a strategic collaboration with SparingVision, a genomic company developing treatments for ocular diseases, on Oct. 13th, 2021, in a press release. The goal of this partnership is to develop novel genomic medicines for the treatment of ocular diseases using CRISPR/Cas9 technology.

Intellia will grant SparingVision exclusive rights to Intellia’s proprietary 

 CRISPR/Cas9-based genome editing technology for up to three ocular targets that address significant unmet medical need. SparingVision will lead and fund the preclinical and clinical development for the genome-editing product candidates. Both parties will research and develop novel self-inactivating adeno-associated viruses (AAV) vector and lipid-nanoparticle (LNP)-based approaches to address delivery of CRISPR/Cas9 genome editing reagents to the retina.

Per the terms of the agreement, Intellia will receive a 10% equity ownership in SparingVision. Additionally, the company will be eligible to receive certain milestone payments of approximately $200 million per product, in addition to royalties on products arising from this collaboration. The transaction is expected to close by the end of the fourth quarter of 2021.

“Intellia is the first company in history to present clinical data supporting precision editing of a disease-causing gene within the body following a single systemic dose of CRISPR/Cas9, and we are honored to have been selected as a strategic partner,” said Stéphane Boissel, president and CEO, SparingVision, in the press release. “We look forward to working together with the shared goal of radically changing the treatment of blinding ocular diseases.”

“We have been thoroughly impressed with the team at SparingVision, particularly regarding their unparalleled understanding of retinal diseases and track-record for developing novel therapies for patients with ocular diseases,” said John Leonard, president and CEO, Intellia, in a press release. “We believe SparingVision will be an excellent partner to expand our genome editing capabilities into the field of ophthalmology and we look forward to our new partnership.”

The collaboration between Intellia and SparingVision will work to develop genomic medicines for ocular diseases.

Intellia Therapeutics and SparingVision Announce Strategic Collaboration to Develop Novel Ocular Therapies Using CRISPR/Cas9 Technology

Enzyvant, a subsidiary of Sumitovant Biopharma, announced that FDA has approved Rethymic (allogeneic processed thymus tissue-agdc) for the treatment of pediatric patients with congenital athymia on Oct. 8th, 2021. According to a company press release, Rethymic is the first and only FDA-approved treatment for immune reconstitution of congenital athymia. Enzyvant has obtained a priority review voucher for Rethymic under the Rare Pediatric Disease Program.

Congenital athymia is a rare condition, with approximately 17–24 new cases occurring each year. Children with congenital athymia are born without a thymus, resulting in depleted T-cells, severe immunodeficiency, and immune dysregulation. Without supportive care, patients typically die by age two or three.

Rethymic is engineered human thymus tissue that is designed to regenerate the thymic function in children with the condition. This is intended to induce a robust immune response that patient’s bodies cannot naturally produce. Notably, Rethymic does not require donor-recipient matching.

Ten studies, involving 105 patients who were surgically implanted with Rethymic, occurred from 1993 to 2020. Of these patients, 29 died, with 23 deaths occurring within a year of implantation. For patients who were alive at one year post implantation, the estimated long-term survival rate was 94% at a median follow-up time of 10.7 years.

Researchers found that T-cells began to reconstitute over the first year, with a durable increase through year two. There was also a statistically significant reduction in the number of infections over the first two years after treatment.

“For too long, families have faced a reality that the brutal journey for pediatric congenital athymia patients receiving supportive care only would end tragically. The FDA approval of Rethymic will help patients access this desperately needed therapy beyond clinical study,” said Rachelle Jacques, CEO of Enzyvant, in the press release. “We are deeply grateful to the 105 patients who participated in clinical trials, their families, and all of the stakeholders who contributed to this pioneering regenerative medicine research program.”

Enzyvant’s Rethymic is the first FDA approved treatment for congenital athymia.

FDA Approves Rethymic for Pediatric Congenital Athymia

Element Materials Technology announced the acquisition of Nanosyn, a pharmaceuticals contract development and manufacturing organization, on Oct. 11, 2021. This acquisition gives Element access to Nanosyn’s three 100,000 ft² facilities as well as over 75 scientists and experts.

According to a company press release, the acquisition of Nanosyn will greatly increase Element’s offering for customers in North America. Nanosyn’s facilities are all located in the San Francisco Bay area, whereas Element’s headquarters are in London.

The acquisition of Nanosyn is the latest in a series of moves made by Element to strengthen their life sciences services in 2021. Nanosyn is the fifth life sciences company Element acquired this year. Most recently, they acquired another North America-based company, Impact Analytical, 

“Nanosyn complements Element’s life sciences offering by extending our services to include drug discovery and small-scale manufacturing for drug development, making us a source of support and true partner for the full value chain,” said Jo Wetz, Element’s CEO, in the press release. “Nanosyn has a deep customer base that includes several of the top pharmaceutical companies in the world, and it supports the development of Element’s position as a full-service solutions provider.”

“Nanosyn has a 23-year track record of supporting numerous drug discovery and development programs which have led to over 50 clinical candidates and several marketed drugs,” said Nikolai Sepetov, Nanosyn’s CEO, in the press release. “Joining Element as it expands its life sciences offering will create the opportunity for us to be at the forefront of progress and development in the pharmaceutical industry.”

Element’s acquisition of Nanosyn is the latest in a series of moves made to strengthen the company’s North American presence.

Element Strengthens US Presence with Nanosyn Acquisition

According to an Oct. 11, 2021 press release, Roche has submitted a marketing authorization application for the monoclonal antibody combination therapy, Ronapreve (casirivimab/imdevimab), as a treatment for COVID-19 to the European Medicines Agency (EMA).

Ronapreve has been co-developed by Roche Registration and Regeneron Pharmaceuticals for the treatment of COVID-19 in adults and adolescents (12 years and older), who do not need supplemental oxygen therapy and who are at an increased risk of progressing to severe disease. Additionally, the combination monoclonal therapy has been developed for the prevention of COVID-19 in adults and adolescents aged 12 years and older.

Thanks to a rolling review of data that has been performed by EMA’s Committee for Medicinal Products for Human Use (CHMP), the agency is now able to assess the benefits-risk balance of Ronapreve in a shortened timeframe, with a potential opinion released in two months. The brevity of EMA’s assessment will be dependent upon the robustness of the data submitted and whether or not further information will be required.

Furthermore, the Pharmacovigilance Risk Assessment Committee of the EMA has already complete the preliminary assessment of the risk management plan and the agency’s Paediatric Committee has provided its opinion on the pediatric investigation plans for the combination drugs.

As soon as CHMP has provided its assessment on the benefit-risk balance of the therapy, EMA will provide further details. If the outcome from CHMP is positive, EMA will work closely with the European Commission to expedite the decision regarding marketing authorization for the product in the European Union.

Roche has submitted a marketing authorization application to EMA for Ronapreve (casirivimab/imdevimab) as a treatment for COVID-19.