Genome Editing and CRISPR Market & IP Landscape

Genome editing (also called gene editing or genome engineering) is a class of technologies that allow specific changes to the DNA of a cell or organism. Genetic materials such as DNA are inserted, deleted, altered or replaced at site specific locations in the genome.

In October 2020, The Royal Swedish Academy of Sciences awarded the Nobel Prize in Chemistry to Emmanuelle Charpentier and Jennifer A. Doudna, who discovered “genetic scissors” for rewriting DNA, known as CRISPR/Cas9. This technology enables precise insertions along strands of DNA to make genetic adjustments on demand.

Precision genome editing technologies are diverse, and also include zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALEN), prime editing, antisense RNA, and adenine base editors (ADE). This alphabet soup of techniques can introduce genetic modifications that go beyond lab science, to cure inherited diseases and boost traditional therapies. Recently, a clinical trial from University of California tested the use of CRISPR gene editing to alter a person’s immune cells to recognize mutated proteins specific to cancer tumors.

For the forseeable future, the IP landscape will be full of clinical milestones that translate CRISPR therapeutics from bench to bedside - how to bet on who will win in the market?

Recently, Intellia Therapeutics updated its ongoing Phase I trial of NTLA-2001, a CRISPR-based gene editing therapy, for the treatment of transthyretin (ATTR) amyloidosis. Based on data from 12 patients, NTLA-2001 resulted in durable responses.

PACT Pharma reported using CRISPR gene editing technology to substitute a gene in patients' immune cells to treat cancer. The results demonstrated an early proof-of-concept of reprogramming a patient's immune system to recognize their own cancer.

In another milestone, Vertex Pharmaceuticals and CRISPR Therapeutics announced that FDA granted a rolling review for exagamglogene autotemcel (exa-cel) to treat sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). Exa-cel (also known as CTX001™) is an autologous CRISPR/Cas9 gene-edited therapy being evaluated for one-time use by patients with TDT or SCD.

Genome editing & CRISPR investments in startups – and academia/small/big pharma collaborations – are growing rapidly in tandem with clinical successes

In 2022, Ionis Pharmaceuticals announced payment of $80 million up front, and an unspecified total of downstream payments, to gene editing startup Metagenomi. The funding aids a collaboration to discover novel CRISPR-based enzymes capable of editing genes from microbial DNA. This funding occurred just a few months after Metagenomi had raised $175 million. Metagenomi also collaborated with COVID-19 vaccine leader Moderna to combine its next-generation CRISPR gene editing systems with Moderna’s mRNA technologies to accelerate the development of gene editing therapeutics.

SparingVision, a genomic medicine company developing vision-saving treatments for ocular diseases, raised €75 million to expand its portfolio of genomic medicines for ocular diseases. The funding will advance its first-in-human trials of their approach to gene therapy.

Tessera Therapeutics received more than $300M to bolster its new gene-editing platforms. 2022 appears to be the best year in the IP Landscape for Genome Editing & CRISPR funding, and is likely to grow further over the next several years.

Fights over Genome Editing & CRISPR inventorship and patentability have already occurred and complexity remains: nearly half of seminal patents and applications are owned by academic and research institutions

In 2022, the USPTO Patent Trial and Appeal Board (PTAB) issued a decision that the use of CRISPR-Cas9 in eukaryotic cells belongs to Broad Institute. This decision meant invalidation of multiple patents from the University of California (UC Berkeley) and stated UC Berkeley’s claims “are unpatentable.” The estimated loss in licensing revenues for UC Berkeley are expected to range from $100 million to $10 billion from former licensees CRISPR Therapeutics, Intellia Therapeutics, Caribou Biosciences and ERS Genomics. The value of understanding & anticipating IP landscape changes in this area is quite significant.

The top 5 Genome Editing and CRISPR IP Landscape leaders by publishing volume are MIT, University of California, Broad Institute, Harvard University, and the Chinese Academy of Sciences. How their portfolios empower and overlap industry is something best visualized, explored by category and captured in context of relationships, growth trends and overlaps. IP Landscape dashboards help to surface important emerging technologies, impactful patent portfolios and potential blockbusters. This visibility and the headstart it offers can be worth billions in the Genome Editing and CRISPR IP Landscape.

Objectives of Intellar Genome Editing and CRISPR IP Landscape Dashboards

Intellar Genome Editing and CRISPR Landscape Dashboards are designed to visualize who is doing what, when and where - and for how much?
Key concepts: genome editing, genome engineering, gene editing, meganucleases, epigenome editing, zinc finger nucleases, transcription activator-like effector nucleases, TALEN technology, clustered regularly interspaced short palindromic repeats (CRISPRs)

Data Coverage for Intellar Genome Editing and CRISPR IP Landscape Dashboards

More than 7,500 de-duplicated global simple patent families published in (machine translated) English since 2017
Technical literature, scientific, medical and engineering journal abstracts, full text (when available) & images published in English since 2017